Back to results

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

Primary Purpose

Duchenne Muscular Dystrophy

Status

Terminated

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

PF-06252616

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne muscular dystrophy, myostatin, open-label

Eligibility Criteria

6 Years - 18 Years (Child, Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Subjects with Duchenne muscular dystrophy who enrolled and completed study B5161002.
Signed and dated informed consent document (ICD) indicating that the subject's parent or legal guardian/caregiver has been informed of all pertinent aspects of the study.
Subjects and their legal guardians/caregivers who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
Subject have;
1. Adequate hepatic function on screening laboratory assessments
2. GLDH less than 20 units/liter (2 x upper limit of normal [ULN])
3. Iron content estimate on the liver MRI within the normal range.

Exclusion Criteria:

Unwilling or unable (eg, metal implants) to undergo examination with closed MRI.
All male subjects who are able to father children and are sexually active and at risk for impregnating a female partner, who are unwilling or unable to use a highly effective method of contraception. In addition, all sexually active male subjects who are unwilling or unable to prevent potential transfer of and exposure to drug through semen to their partners by using a condom consistently and correctly. .
Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are related to Pfizer employees directly involved in the conduct of the study.
Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation.
Participation in other studies involving investigational drug(s), with the exception of B5161002.
History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of this investigational product.

Sites / Locations

David Geffen School of Medicine at UCLA/UCLA Neurology
Ronald Reagan UCLA Medical Center
Ronald Reagan UCLA Pharmacy
UCLA (David Geffen School of Medicine), Department of Orthopedic Surgery
UCLA Clinical & Translational Research Center
University of California, Davis Medical Center
University of Iowa ICTS, Clinical Research Unit
Kennedy Krieger Institute Out-patient Center
Kennedy Krieger Institute
Johns Hopkins Hospital
Johns Hopkins Investigational Drug Service
Massachusetts General Hospital
University of Minnesota Masonic Children's Hospital
(For Drug deliveries) IDS Pharmacy
St. Louis Childrens's Hospital
Duke University Medical Center, Lenox Baker Children's Hospital
Duke University, Investigational Drug Pharmacy
Cincinnati Children's Hospital Medical Center
Children's Hospital of Philadelphia
Children's Hospital of Pittsburgh of UPMC
Center for Clinical and Translational Sciences
Utah Center for Advanced Imaging and Research (UCAIR)
Investigational Drug Services
Utah Program for Inherited Neuromuscular Disorder
University of Utah Hospital
University of Utah School of Medicine
Alberta Children's Hospital
UBC Children's and Women's Health Centre of British Columbia
Children's Hospital- London Health Sciences Centre
Centre de Readaptation Marie Enfant (CRME)
CHU Sainte-Justine
UOC Farmacia - Istituto Gianna Gaslini,
UOC Medicina Fisica Riabilitativa - Istituto G. Gaslini
UOC Neurologia Pediatrica e Malattie Muscolari Istituto Gianna Gaslini
UOC Radiologia - Istituto Gianna Gaslini,
UOS Dipartimentale Endocrinologia Clinica Sperimentale - Ist.
Dipartimento Pediatrico Universitario-Ospedaliero Endocrinologia
Farmacia Ospedaliera, IRCCS Ospedale Pediatrico Bambino Gesu
IRCCS Ospedale Pediatrico Bambino Gesu - Centro Trials
U.O. Malattie Neuromuscolari e Neurodegenerative, IRCCS Ospedale Pediatrico Bambino Gesu
Hyogo college of medicine hospital
National Center of Neurology and Psychiatry
Dubowitz Neuromuscular Centre, Institute of Child Health
Great Ormond Street Hospital
Institute of Genetic Medicine,Muscle Team
Clinical Research Facility
Royal Victoria Infirmary Research Pharmacy

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PF-06252616

Arm Description

Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002

Outcomes

Primary Outcome Measures

Number of Participants With Dose Reduced or Temporary Discontinuation Due to AEs

An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment or usage. Treatment-related AEs were determined by the investigator. The number of participants with dose reduced or temporary discontinuation due to both all-causality and treatment-related AEs are presented below.

Number of Participants With Severe Treatment-Emergent Adverse Events (TEAEs)

An AE was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment or usage. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Severe TEAEs were TEAEs that interfered significantly with participants' usual function. Treatment-related TEAEs were determined by the investigator. The number of participants with severe all-causality and treatment-related TEAEs are presented below.

Number of Participants Who Discontinued From the Study Due to TEAEs

An AE was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment or usage. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Treatment-related TEAEs were determined by the investigator. The number of participants who discontinued from the study due to both all-causality and treatment-related TEAEs are presented below.

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Hematology

Hematology evaluation included: hemoglobin, hematocrit, red blood cell (RBC) count, platelets, RBC morphology, white blood cell (WBC) count, absolute lymphocytes, absolute atypical lymphocytes, absolute total neutrophils, absolute total neutrophils count, absolute band cells, absolute basophils, absolute eosinophils and absolute monocytes. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (ULN=Upper Limit of Normal; LLN=Lower Limit of Normal).

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Coagulation

Coagulation evaluation included activated partial thromboplastin time (aPTT) and prothrombin time (PT). (ULN=Upper Limit of Normal). Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004.

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Liver Function

Liver function evaluation included: total bilirubin, aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT), alkaline phosphatase, total protein, albumin and glutamate dehydrogenase. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (LLN=Lower Limit of Normal; ULN=Upper Limit of Normal).

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Renal Function

Renal function evaluation included: blood urea nitrogen (BUN), creatinine and uric acid. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (ULN=Upper Limit of Normal).

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Electrolytes

Electrolytes evaluation included: sodium, potassium, chloride, calcium, phosphate and bicarbonate. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (LLN=Lower Limit of Normal, ULN=Upper Limit of Normal).

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Hormones

Hormone evaluations included free thyroxine (T4), thyroid stimulating hormone (TSH), lutenizing hormone (LH), follicle stimulating hormone (FSH), and androstenedione. Numbers of participants with abnormalities of LH, FSH and androstenedione were reported in different age groups. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (LLN=Lower Limit of Normal, ULN=Upper Limit of Normal).

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Clinical Chemistry

Clinical chemistry evaluation included glucose, creatine kinase (CK), troponin I, amylase, iron binding capacity, unsaturated iron binding capacity, transferrin saturation, iron and ferritin. Number of participants with iron abnormalities was reported in different age groups. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (LLN=Lower Limit of Normal, ULN=Upper Limit of Normal).

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Urinalysis

Urinalysis Microscopy included: urine red blood cell (RBC), urine white blood cell (WBC), urine uric acid crystals, urine calcium oxalate crystals, urine amorphous crystals, urine bacteria, urine microscopic exam. Urinalysis Dipstick included: urine pH, urine glucose, urine ketones, urine protein, urine blood/hemoglobin, urine nitrite, urine leukocyte esterase. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004.

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Fecal Blood

Number of participants with blood detected in fecal samples is presented. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (ULN=Upper Limit of Normal).

Number of Participants With Data of Serum Ferritin, Serum Iron and % Transferrin Saturation Meeting Categorical Summarization Criteria - B5161004 Baseline

Participants were asked to fast for at least 8 hours prior to collection of blood to evaluate serum iron, serum ferritin and % transferrin saturation. The unit of iron was mcg/dL; the unit of ferritin was ng/mL; the unit of %transferrin saturation was %. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Number of Participants With Significant Results of Physical Examinations Including Nose and Throat Mucosal Examinations

Physical examinations were conducted by a physician, trained physician's assistant, or nurse practitioner as acceptable according to local regulation. A targeted nose and throat mucosal exam was performed to monitor for any signs of mucosal telangiectasias. The clinically significant physical examination results were determined by the investigator.

Summary of Pubertal Development by Tanner Stage

Tanner staging was performed before the first dose of this study to monitor for signs of accelerated sexual development. The physical changes in pubertal development (pubic hair, penis and testes) were assessed using the system described by Marshall and Tanner. Stage 1 is preadolescent, Stages 2, 3, and 4 are initiation of puberty and Stage 5 is mature adult. Details about the system can be referred to Tanner JM. Growth at Adolescence. Blackwell Scientific Publications 1962; 2nd edition. Participant's Week 97 visit within study B5161002 (parent study) was collected as screening data. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Summary of Testicular Volume

Testicular volume was used to monitor pubertal development. Participant's Week 97 visit within Study B5161002 (parent study) was collected as screening data in current study. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Number of Participants With Post-Baseline Vital Signs Data Meeting Categorical Summarization Criteria - B5161004 Baseline

The number of participants pre-dose supine blood pressure and pulse rate meeting categorical summarization criteria are recorded in this table. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (DBP=diastolic blood pressure, SBP=systolic blood pressure; The unit for blood pressure is: mmHg, the unit for pulse rate is: beats per minute [BPM])

Number of Participants With Post-Baseline Vital Signs Data Meeting Categorical Summarization Criteria - Overall Baseline

The number of participants with data of pre-dose supine blood pressure meeting categorical summarization were recorded in this table. Overall Baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. (DBP=diastolic blood pressure, SBP=systolic blood pressure; The unit for blood pressure is: mmHg).

Number of Participants With Post-Baseline ECG Data Meeting Categorical Summarization Criteria - B5161004 Baseline

QTcF=QT/(60/Hour)**(1/3). Means of replicates were used in the calculations. QT=time between the start of the Q wave and the end of the T wave in the heart's electrical cycle; QTcF=corrected QT (Fridericia correction). All scheduled ECGs were performed after the participant had rested quietly for at least 10 minutes in a supine position. Baseline was defined as the average of the last triplicate pre-dose measurements prior to Day 1 in B5161004.

Number of Participants With Post-Baseline ECG Data Meeting Categorical Summarization Criteria - Overall Baseline

QT=time between the start of the Q wave and the end of the T wave in the heart's electrical cycle; QTcF=corrected QT (Fridericia correction). All scheduled ECGs were performed after the participant had rested quietly for at least 10 minutes in a supine position. Overall baseline was defined as the average of the last triplicate pre-dose measurements prior to the first day of dosing in study B5161002.

Number of Participants With Iron Accumulation Data Meeting Categorical Summarization Criteria

Liver Magnetic Resonance Imaging (MRIs) were sent to an independent central radiology imaging facility for calculation of the average R2* value which was used to monitor for iron accumulation in the liver. Mean R2* values had been used in the calculations. Normal: R2* <= 75 Hz at 1.5 T or <=139 Hz at 3.0 T; Above Normal: R2* > 75 Hz and <= 190 Hz at 1.5 T or R2* > 139 Hz and <= 369 Hz at 3.0 T Mild overload: R2* > 190 Hz at 1.5 T or R2* > 369 Hz at 3.0 T Data from participant's Week 93 visit in Study B5161002 (parent study) were used for screening in the current study.

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Cardiac MRI - B5161004 Baseline

The LVEF was the ratio of blood ejected during systole to blood in the ventricle at the end of diastole. LVEF was measured by cardiac magnetic resonance imaging (MRI) or echocardiography. The same method of cardiac imaging was used consistently for each participant. Cardiac MRIs were read by a central imaging vendor, while echocardiograms were read locally (at each site). The table presents the results from cardiac MRIs. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Cardiac MRI - Overall Baseline

The LVEF was the ratio of blood ejected during systole to blood in the ventricle at the end of diastole. LVEF was measured by cardiac magnetic resonance imaging (MRI) or echocardiography. The same method of cardiac imaging was used consistently for each participant. Cardiac MRIs were read by a central imaging vendor, while echocardiograms were read locally (at each site). The table presents the results from cardiac MRIs. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Echocardiogram - B5161004 Baseline

The LVEF was the ratio of blood ejected during systole to blood in the ventricle at the end of diastole. LVEF was measured by cardiac magnetic resonance imaging (MRI) or echocardiography. The same method of cardiac imaging was used consistently for each participant. Cardiac MRIs were read by a central imaging vendor, while echocardiograms were read locally (at each site). The table presents the results from echocardiograms. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Echocardiogram - Overall Baseline

The LVEF was the ratio of blood ejected during systole to blood in the ventricle at the end of diastole. LVEF was measured by cardiac magnetic resonance imaging (MRI) or echocardiography. The same method of cardiac imaging was used consistently for each participant. Cardiac MRIs were read by a central imaging vendor, while echocardiograms were read locally (at each site). The table presents the results from echocardiograms. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Bone Age to Chronological Age Ratio

Bone age assessment was evaluated by the ratio of the bone age to the chronological age using the X rays of the hand and wrist. Ratio of bone age to chronological age was calculated by bone age/chronological age at scan date. Chronological age at scan date was calculated by (scan date - date of birth + 1)/365.25. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Whole Body and Spine DXA: Bone Mineral Density Z-Score, Height Adjusted Over Time

Bone mineral density (BMD) was monitored by dual energy x-ray absorptiometry (DXA). DXA scans were obtained to evaluate bone mineral density of the spine and whole body without head. The height adjusted Z-score presented below is the number of standard deviations which compares the BMD of the participant to the average BMD matched for their age, sex and ethnicity. If the Z-score was -2 standard deviations or lower, the result was "below the expected range for age". If the Z-score was above -2 standard deviations, the result was "within the expected range for age".

Number of Participants With Suicidal Ideation or Suicidal Behavior

The Columbia Suicide Severity Rating Scale (C-SSRS) was performed to identify the risk of suicide ideation or behavior. C-SSRS was conducted with the participant's caregiver/legal guardian on the participant's behalf throughout the study, rather than administering this evaluation directly with the study participants. If at any visit the participant endorsed a 4 or 5 on the C-SSRS ideation section or reported any suicidality behavior, then an evaluation of suicide risk (risk assessment) had to be completed and the participant must have been discontinued. The significant result of C-SSRS was determined by the investigator.

Secondary Outcome Measures

Change From Baseline on the 4 Stair Climb (4SC) - B5161004 Baseline

The 4SC quantified the time required for a participant to ascend 4 standard steps. The functional assessment of 4SC was conducted by a physiotherapist (or exercise physiologist). In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessments were completed at approximately the same time of day. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the 4SC - Overall Baseline

The 4SC quantified the time required for a participant to ascend 4 standard steps. The functional assessment of 4SC was conducted by a physiotherapist (or exercise physiologist). In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessments were completed at approximately the same time of day. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Change From Baseline on the Forced Vital Capacity (FVC) - B5161004 Baseline

FVC was measured using the FVC maneuver by spirometry to evaluate respiratory muscle function. The best (largest) FVC measurement from a set of 3 was captured on the database. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the FVC - Overall Baseline

Forced vital capacity (FVC) was measured using the FVC maneuver by spirometry to evaluate respiratory muscle function. The best (largest) FVC measurement from a set of 3 was captured on the database. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Change From Baseline on the Northstar Ambulatory Assessment (NSAA) Score - B5161004 Baseline

The NSAA was a 17-item test that measured gross motor function. Each individual item was evaluated with either 0-unable to perform independently, 1-able to perform with assistance, 2-able to perform without assistance. A total score was achieved by summing all the individual items. The total score could range from 0 to 34 (fully-independent function). Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the NSAA Score - Overall Baseline

The NSAA was a 17-item test that measured gross motor function. Each individual item was evaluated with either 0-unable to perform independently, 1-able to perform with assistance, 2-able to perform without assistance. A total score was achieved by summing all the individual items. The total score could range from 0 to 34 (fully-independent function). This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Change From Baseline on the NSAA - Time to Stand From Supine - B5161004 Baseline

Rise from supine was a timed functional test within NSAA. This test of time-to-stand from supine was analyzed separately for summary tabulation along with the total NSAA score. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the NSAA - Time to Stand From Supine - Overall Baseline

Rise from supine was a timed functional test within NSAA. This test of time-to-stand from supine was analyzed separately for summary tabulation along with the total NSAA score. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Change From Baseline on the NSAA - Time to Complete 10 m Run/Walk - B5161004 Baseline

A time to event analysis was performed for loss of ambulation. Loss of ambulation was defined as the inability to walk unassisted and without braces for at least 10 m, as assessed and reported by the investigator at each study visit, and confirmed by the inability to walk/run 10 m (as 1 component of the NSAA) evaluated at the next visit at which timed function tests were performed. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the NSAA - Time to Complete 10 m Run/Walk - Overall Baseline

A time to event analysis was performed for loss of ambulation. Loss of ambulation was defined as the inability to walk unassisted and without braces for at least 10 m, as assessed and reported by the investigator at each study visit, and confirmed by the inability to walk/run 10 m (as 1 component of the NSAA) evaluated at the next visit at which timed function tests were performed. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Change From Baseline on the Ankle Range of Motion (ROM) - B5161004 Baseline

ROM of the ankle was evaluated by goniometry and any occurrences of ankle contractures were recorded. In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessment of ankle ROM was completed at approximately the same time of day. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the Ankle ROM - Overall Baseline

ROM of the ankle was evaluated by goniometry and any occurrences of ankle contractures were recorded. In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessment of ankle ROM was completed at approximately the same time of day. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Change From Baseline on the Performance of Upper Limb (PUL) Overall Score - B5161004 Baseline

The PUL scale was used to assess motor performance of the upper limb for individuals with DMD. The PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into 3 levels; shoulder (4 items), middle (9 items) and distal (8 items). Scoring options per item may not be uniform and may vary from 0-1 to 0-6, according to the performance, with higher values corresponding to better performance. A total maximum score of 74 is achieved by adding the individual level scores; shoulder maximum 16, middle level maximum score 34 and distal level maximum score 24. In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessment of PUL was completed at approximately the same time of day. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the PUL Overall Score - Overall Baseline

The PUL scale was used to assess motor performance of the upper limb for individuals with DMD. The PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into 3 levels; shoulder (4 items), middle (9 items) and distal (8 items). Scoring options per item may not be uniform and may vary from 0-1 to 0-6, according to the performance, with higher values corresponding to better performance. A total maximum score of 74 is achieved by adding the individual level scores; shoulder maximum 16, middle level maximum score 34 and distal level maximum score 24. This is the overall change from baseline which included the change since enrolling in parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Change From Baseline on the Six Minute Walk Distance (6MWD) - B5161004 Baseline

The 6MWD evaluated ambulation ability by measuring the distance walked in 6 minutes. In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessment of 6MWD was completed at approximately the same time of day. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the 6MWD - Overall Baseline

The 6MWD evaluated ambulation ability by measuring the distance walked in 6 minutes. In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessment of 6MWD was completed at approximately the same time of day. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was start of the study treatment in parent study B5161002.

Change From Baseline on the Forced Expiratory Volume in One Second (FEV1) - B5161004 Baseline

The FEV1 was recorded as an absolute volume in litres and in terms of predicted values according to age, height, race and gender. The best single FEV1 measurement from a set of 3 was recorded in the database. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the Peak Expiratory Flow Rate (PEFR)- B5161004 Baseline

PEFR was one of the Pulmonary Function Tests (PFTs). Three technically adequate peak expiratory flow rate (PEFR) maneuvers were performed and reported in Litres/Minute (L/min), and the highest single PEFR was reported in the database. In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessment of PEFR was completed at approximately the same time of day. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the Myometry Based Muscle Strength - B5161004 Baseline

Muscle strength was quantified by means of a handheld dynamometer. The following muscle groups were evaluated: knee extension, elbow flexion, elbow extension, hip abduction and shoulder abduction. 95% Confidence Interval was not calculated when less than or equal to 3 participants' data were available. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Change From Baseline on the Myometry Based Muscle Strength - Overall Baseline

Muscle strength was quantified by means of a handheld dynamometer. The following muscle groups were evaluated: knee extension, elbow flexion, elbow extension, hip abduction and shoulder abduction. 95% Confidence Interval was not calculated when less than or equal to 3 participants' data were available. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was start of the study treatment in parent study B5161002.

Serum PF-06252616 (Domagrozumab) Concentration Versus Time Summary

Number of Participants With Anti-drug Antibodies (ADA) Development

The criterion for positive result of ADA samples was ADA titer >=1.88. The criterion for negative result of ADA samples was ADA titer <1.88.

Full Information

NCT ID

NCT02907619

First Posted

September 14, 2016

Last Updated

October 28, 2020

Sponsor

Pfizer

1. Study Identification

Unique Protocol Identification Number

NCT02907619

Brief Title

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

Official Title

A MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYSTROPHY

Study Type

Interventional

2. Study Status

Record Verification Date

October 2020

Overall Recruitment Status

Terminated

Why Stopped

Termination Date: 30Aug2018; Reason for termination: Lack of Efficacy

Study Start Date

October 13, 2016 (Actual)

Primary Completion Date

November 22, 2018 (Actual)

Study Completion Date

November 22, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose based on their tolerability profile/data from B5161002. This study will not contain a placebo comparator. Subjects will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional capacity evaluations (4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing, six minute walk test and pulmonary function tests) and pharmacokinetic testing.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophy, myostatin, open-label

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Masking

None (Open Label)

Allocation

N/A

Enrollment

59 (Actual)

8. Arms, Groups, and Interventions

Arm Title

PF-06252616

Arm Type

Experimental

Arm Description

Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002

Intervention Type

Biological

Intervention Name(s)

PF-06252616

Intervention Description

Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002

Primary Outcome Measure Information:

Title

Number of Participants With Dose Reduced or Temporary Discontinuation Due to AEs

Description

Time Frame

2 Years

Title

Number of Participants With Severe Treatment-Emergent Adverse Events (TEAEs)

Description

Time Frame

2 Years

Title

Number of Participants Who Discontinued From the Study Due to TEAEs

Description

An AE was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment or usage. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Treatment-related TEAEs were determined by the investigator. The number of participants who discontinued from the study due to both all-causality and treatment-related TEAEs are presented below.

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Hematology

Description

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Coagulation

Description

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Liver Function

Description

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Renal Function

Description

Renal function evaluation included: blood urea nitrogen (BUN), creatinine and uric acid. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (ULN=Upper Limit of Normal).

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Electrolytes

Description

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Hormones

Description

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Clinical Chemistry

Description

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Urinalysis

Description

Time Frame

2 Years

Title

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Fecal Blood

Description

Number of participants with blood detected in fecal samples is presented. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. (ULN=Upper Limit of Normal).

Time Frame

2 Years

Title

Number of Participants With Data of Serum Ferritin, Serum Iron and % Transferrin Saturation Meeting Categorical Summarization Criteria - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 37, 49, 61, 73 and 85.

Title

Number of Participants With Significant Results of Physical Examinations Including Nose and Throat Mucosal Examinations

Description

Time Frame

2 Years

Title

Summary of Pubertal Development by Tanner Stage

Description

Time Frame

Screening, Baseline, Week 49.

Title

Summary of Testicular Volume

Description

Time Frame

Screening, Baseline, Week 49.

Title

Number of Participants With Post-Baseline Vital Signs Data Meeting Categorical Summarization Criteria - B5161004 Baseline

Description

Time Frame

2 Years

Title

Number of Participants With Post-Baseline Vital Signs Data Meeting Categorical Summarization Criteria - Overall Baseline

Description

Time Frame

2 Years

Title

Number of Participants With Post-Baseline ECG Data Meeting Categorical Summarization Criteria - B5161004 Baseline

Description

Time Frame

2 Years

Title

Number of Participants With Post-Baseline ECG Data Meeting Categorical Summarization Criteria - Overall Baseline

Description

Time Frame

2 Years

Title

Number of Participants With Iron Accumulation Data Meeting Categorical Summarization Criteria

Description

Time Frame

Screening and Week 49.

Title

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Cardiac MRI - B5161004 Baseline

Description

Time Frame

Baseline and Week 49.

Title

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Cardiac MRI - Overall Baseline

Description

The LVEF was the ratio of blood ejected during systole to blood in the ventricle at the end of diastole. LVEF was measured by cardiac magnetic resonance imaging (MRI) or echocardiography. The same method of cardiac imaging was used consistently for each participant. Cardiac MRIs were read by a central imaging vendor, while echocardiograms were read locally (at each site). The table presents the results from cardiac MRIs. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Time Frame

Baseline, Weeks 49, 97, 146.

Title

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Echocardiogram - B5161004 Baseline

Description

The LVEF was the ratio of blood ejected during systole to blood in the ventricle at the end of diastole. LVEF was measured by cardiac magnetic resonance imaging (MRI) or echocardiography. The same method of cardiac imaging was used consistently for each participant. Cardiac MRIs were read by a central imaging vendor, while echocardiograms were read locally (at each site). The table presents the results from echocardiograms. Baseline was defined as the last assessment prior to dosing on Day 1 in B5161004. Week 1 was counted starting from the study treatment in study B5161004.

Time Frame

Baseline, Week 49.

Title

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Echocardiogram - Overall Baseline

Description

The LVEF was the ratio of blood ejected during systole to blood in the ventricle at the end of diastole. LVEF was measured by cardiac magnetic resonance imaging (MRI) or echocardiography. The same method of cardiac imaging was used consistently for each participant. Cardiac MRIs were read by a central imaging vendor, while echocardiograms were read locally (at each site). The table presents the results from echocardiograms. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Time Frame

Baseline, Weeks 49, 97, 146.

Title

Bone Age to Chronological Age Ratio

Description

Time Frame

Baseline and Week 49.

Title

Whole Body and Spine DXA: Bone Mineral Density Z-Score, Height Adjusted Over Time

Description

Time Frame

Screening (Week 97 visit within parent study B5161002) and Week 49

Title

Number of Participants With Suicidal Ideation or Suicidal Behavior

Description

Time Frame

2 Years

Secondary Outcome Measure Information:

Title

Change From Baseline on the 4 Stair Climb (4SC) - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49, 73.

Title

Change From Baseline on the 4SC - Overall Baseline

Description

The 4SC quantified the time required for a participant to ascend 4 standard steps. The functional assessment of 4SC was conducted by a physiotherapist (or exercise physiologist). In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessments were completed at approximately the same time of day. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Time Frame

Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Change From Baseline on the Forced Vital Capacity (FVC) - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49 and 73.

Title

Change From Baseline on the FVC - Overall Baseline

Description

Time Frame

Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Change From Baseline on the Northstar Ambulatory Assessment (NSAA) Score - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49, 73.

Title

Change From Baseline on the NSAA Score - Overall Baseline

Description

The NSAA was a 17-item test that measured gross motor function. Each individual item was evaluated with either 0-unable to perform independently, 1-able to perform with assistance, 2-able to perform without assistance. A total score was achieved by summing all the individual items. The total score could range from 0 to 34 (fully-independent function). This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Time Frame

Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Change From Baseline on the NSAA - Time to Stand From Supine - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49, 73.

Title

Change From Baseline on the NSAA - Time to Stand From Supine - Overall Baseline

Description

Time Frame

Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Change From Baseline on the NSAA - Time to Complete 10 m Run/Walk - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49, 73.

Title

Change From Baseline on the NSAA - Time to Complete 10 m Run/Walk - Overall Baseline

Description

A time to event analysis was performed for loss of ambulation. Loss of ambulation was defined as the inability to walk unassisted and without braces for at least 10 m, as assessed and reported by the investigator at each study visit, and confirmed by the inability to walk/run 10 m (as 1 component of the NSAA) evaluated at the next visit at which timed function tests were performed. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Time Frame

Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Change From Baseline on the Ankle Range of Motion (ROM) - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49 and 73.

Title

Change From Baseline on the Ankle ROM - Overall Baseline

Description

ROM of the ankle was evaluated by goniometry and any occurrences of ankle contractures were recorded. In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessment of ankle ROM was completed at approximately the same time of day. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Time Frame

Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Change From Baseline on the Performance of Upper Limb (PUL) Overall Score - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49 and 73.

Title

Change From Baseline on the PUL Overall Score - Overall Baseline

Description

The PUL scale was used to assess motor performance of the upper limb for individuals with DMD. The PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into 3 levels; shoulder (4 items), middle (9 items) and distal (8 items). Scoring options per item may not be uniform and may vary from 0-1 to 0-6, according to the performance, with higher values corresponding to better performance. A total maximum score of 74 is achieved by adding the individual level scores; shoulder maximum 16, middle level maximum score 34 and distal level maximum score 24. This is the overall change from baseline which included the change since enrolling in parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was counted starting from the study treatment in study B5161002.

Time Frame

Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Change From Baseline on the Six Minute Walk Distance (6MWD) - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49 and 73.

Title

Change From Baseline on the 6MWD - Overall Baseline

Description

The 6MWD evaluated ambulation ability by measuring the distance walked in 6 minutes. In order to provide optimal testing conditions and consistency in endpoint measurements, the functional assessment of 6MWD was completed at approximately the same time of day. This is the overall change from baseline which included the change since enrolling in the parent study B5161002. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was start of the study treatment in parent study B5161002.

Time Frame

Baseline,Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Change From Baseline on the Forced Expiratory Volume in One Second (FEV1) - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49 and 73.

Title

Change From Baseline on the Peak Expiratory Flow Rate (PEFR)- B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49 and 73.

Title

Change From Baseline on the Myometry Based Muscle Strength - B5161004 Baseline

Description

Time Frame

Baseline, Weeks 13, 25, 49, 73.

Title

Change From Baseline on the Myometry Based Muscle Strength - Overall Baseline

Description

Muscle strength was quantified by means of a handheld dynamometer. The following muscle groups were evaluated: knee extension, elbow flexion, elbow extension, hip abduction and shoulder abduction. 95% Confidence Interval was not calculated when less than or equal to 3 participants' data were available. Overall baseline was defined as the last pre-dose assessment prior to the first day of dosing in study B5161002. Week 1 was start of the study treatment in parent study B5161002.

Time Frame

Baseline,Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.

Title

Serum PF-06252616 (Domagrozumab) Concentration Versus Time Summary

Time Frame

Weeks 1, 25, 49 and 73

Title

Number of Participants With Anti-drug Antibodies (ADA) Development

Description

The criterion for positive result of ADA samples was ADA titer >=1.88. The criterion for negative result of ADA samples was ADA titer <1.88.

Time Frame

Weeks 1, 25, 49, 73 and Early Termination

10. Eligibility

Sex

Male

Gender Based

Yes

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Subjects with Duchenne muscular dystrophy who enrolled and completed study B5161002. Signed and dated informed consent document (ICD) indicating that the subject's parent or legal guardian/caregiver has been informed of all pertinent aspects of the study. Subjects and their legal guardians/caregivers who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. Subject have; Adequate hepatic function on screening laboratory assessments GLDH less than 20 units/liter (2 x upper limit of normal [ULN]) Iron content estimate on the liver MRI within the normal range. Exclusion Criteria: Unwilling or unable (eg, metal implants) to undergo examination with closed MRI. All male subjects who are able to father children and are sexually active and at risk for impregnating a female partner, who are unwilling or unable to use a highly effective method of contraception. In addition, all sexually active male subjects who are unwilling or unable to prevent potential transfer of and exposure to drug through semen to their partners by using a condom consistently and correctly. . Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are related to Pfizer employees directly involved in the conduct of the study. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation. Participation in other studies involving investigational drug(s), with the exception of B5161002. History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of this investigational product.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Pfizer CT.gov Call Center

Organizational Affiliation

Pfizer

Official's Role

Study Director

Facility Information:

Facility Name

David Geffen School of Medicine at UCLA/UCLA Neurology

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

Ronald Reagan UCLA Medical Center

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

Ronald Reagan UCLA Pharmacy

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

UCLA (David Geffen School of Medicine), Department of Orthopedic Surgery

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

UCLA Clinical & Translational Research Center

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

University of California, Davis Medical Center

City

Sacramento

State/Province

California

ZIP/Postal Code

95817

Country

United States

Facility Name

University of Iowa ICTS, Clinical Research Unit

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

Kennedy Krieger Institute Out-patient Center

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

Facility Name

Kennedy Krieger Institute

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

Facility Name

Johns Hopkins Hospital

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21287

Country

United States

Facility Name

Johns Hopkins Investigational Drug Service

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21287

Country

United States

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114

Country

United States

Facility Name

University of Minnesota Masonic Children's Hospital

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55454

Country

United States

Facility Name

(For Drug deliveries) IDS Pharmacy

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

Facility Name

St. Louis Childrens's Hospital

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

Facility Name

Duke University Medical Center, Lenox Baker Children's Hospital

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27705

Country

United States

Facility Name

Duke University, Investigational Drug Pharmacy

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27710

Country

United States

Facility Name

Cincinnati Children's Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

Facility Name

Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Children's Hospital of Pittsburgh of UPMC

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15224

Country

United States

Facility Name

Center for Clinical and Translational Sciences

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84108

Country

United States

Facility Name

Utah Center for Advanced Imaging and Research (UCAIR)

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84108

Country

United States

Facility Name

Investigational Drug Services

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84112

Country

United States

Facility Name

Utah Program for Inherited Neuromuscular Disorder

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84112

Country

United States

Facility Name

University of Utah Hospital

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84132

Country

United States

Facility Name

University of Utah School of Medicine

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84132

Country

United States

Facility Name

Alberta Children's Hospital

City

Calgary

State/Province

Alberta

ZIP/Postal Code

T3B 6A8

Country

Canada

Facility Name

UBC Children's and Women's Health Centre of British Columbia

City

Vancouver

State/Province

British Columbia

ZIP/Postal Code

V6H 3V4

Country

Canada

Facility Name

Children's Hospital- London Health Sciences Centre

City

London

State/Province

Ontario

ZIP/Postal Code

N6A5W9

Country

Canada

Facility Name

Centre de Readaptation Marie Enfant (CRME)

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H1T 1C9

Country

Canada

Facility Name

CHU Sainte-Justine

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H3T 1C5

Country

Canada

Facility Name

UOC Farmacia - Istituto Gianna Gaslini,

City

Genova

ZIP/Postal Code

16147

Country

Italy

Facility Name

UOC Medicina Fisica Riabilitativa - Istituto G. Gaslini

City

Genova

ZIP/Postal Code

16147

Country

Italy

Facility Name

UOC Neurologia Pediatrica e Malattie Muscolari Istituto Gianna Gaslini

City

Genova

ZIP/Postal Code

16147

Country

Italy

Facility Name

UOC Radiologia - Istituto Gianna Gaslini,

City

Genova

ZIP/Postal Code

16147

Country

Italy

Facility Name

UOS Dipartimentale Endocrinologia Clinica Sperimentale - Ist.

City

Genova

ZIP/Postal Code

16147

Country

Italy

Facility Name

Dipartimento Pediatrico Universitario-Ospedaliero Endocrinologia

City

Rome

ZIP/Postal Code

00150

Country

Italy

Facility Name

Farmacia Ospedaliera, IRCCS Ospedale Pediatrico Bambino Gesu

City

Rome

ZIP/Postal Code

00165

Country

Italy

Facility Name

IRCCS Ospedale Pediatrico Bambino Gesu - Centro Trials

City

Rome

ZIP/Postal Code

00165

Country

Italy

Facility Name

U.O. Malattie Neuromuscolari e Neurodegenerative, IRCCS Ospedale Pediatrico Bambino Gesu

City

Rome

ZIP/Postal Code

00165

Country

Italy

Facility Name

Hyogo college of medicine hospital

City

Nishinomiya-shi

State/Province

Hyogo

ZIP/Postal Code

663-8501

Country

Japan

Facility Name

National Center of Neurology and Psychiatry

City

Kodaira

State/Province

Tokyo

ZIP/Postal Code

187-8551

Country

Japan

Facility Name

Dubowitz Neuromuscular Centre, Institute of Child Health

City

London

ZIP/Postal Code

WC1N 3JH

Country

United Kingdom

Facility Name

Great Ormond Street Hospital

City

London

ZIP/Postal Code

WC1N 3JH

Country

United Kingdom

Facility Name

Institute of Genetic Medicine,Muscle Team

City

Newcastle-upon-Tyne

ZIP/Postal Code

NE1 3BZ

Country

United Kingdom

Facility Name

Clinical Research Facility

City

Newcastle-upon-Tyne

ZIP/Postal Code

NE1 4LP

Country

United Kingdom

Facility Name

Royal Victoria Infirmary Research Pharmacy

City

Newcastle-upon-Tyne

ZIP/Postal Code

NE1 4LP

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

IPD Sharing URL

https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

Citations:

PubMed Identifier

32522498

Citation

Wagner KR, Abdel-Hamid HZ, Mah JK, Campbell C, Guglieri M, Muntoni F, Takeshima Y, McDonald CM, Kostera-Pruszczyk A, Karachunski P, Butterfield RJ, Mercuri E, Fiorillo C, Bertini ES, Tian C, Statland J, Sadosky AB, Purohit VS, Sherlock SP, Palmer JP, Binks M, Charnas L, Marraffino S, Wong BL. Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy. Neuromuscul Disord. 2020 Jun;30(6):492-502. doi: 10.1016/j.nmd.2020.05.002. Epub 2020 May 19. Erratum In: Neuromuscul Disord. 2021 Feb;31(2):167-168.

Results Reference

derived

Links:

URL

https://pmiform.com/clinical-trial-info-request?StudyID=B5161004

Description

To obtain contact information for a study center near you, click here.

Learn more about this trial

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

We'll reach out to this number within 24 hrs