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Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (ACT DMD)

Primary Purpose

Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Ataluren

Placebo

About this trial

This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Duchenne muscular dystrophy, Dystrophinopathy, Nonsense mutation, Premature stop codon, Becker muscular dystrophy, DMD/BMD, PTC124, Ataluren

Eligibility Criteria

7 Years - 16 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Ability to provide written informed consent (parental/guardian consent if applicable)/assent per local requirements.
Phenotypic evidence of dystrophinopathy based on the onset of characteristic clinical symptoms or signs (for example; proximal muscle weakness, waddling gait, and Gowers' maneuver) by 6 years of age, an elevated serum creatinine kinase level, and ongoing difficulty with walking.
Documentation of the presence of a nonsense point mutation in the dystrophin gene as determined by gene sequencing from a laboratory certified by the College of American Pathologists (CAP), the Clinical Laboratory Improvement Act/Amendment (CLIA) or an equivalent organization.
Documentation that a blood sample has been drawn for confirmation of the presence of a nonsense mutation in the dystrophin gene.
Use of systemic corticosteroids (prednisone, prednisolone, or deflazacort) for a minimum of 6 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen (not related to body weight change) for a minimum of 3 months immediately prior to start of study treatment and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
Valid Screening 6-minute walk distance (6MWD) greater than or equal to (≥) 150 meters. Valid Screening 6MWD must have been less than or equal to (≤) 80% of predicted for age and height.
Results of the 2 Baseline 6MWD results must be determined as valid and results of the Day 2 Baseline 6MWD must be within 20% of the Day 1 Baseline 6MWD.
Baseline 6MWD (mean of valid Day 1 and Day 2 values) must be no more than a 20% change from the valid Screening 6MWD.
Confirmed screening laboratory values within the central laboratory ranges (hepatic, renal, and serum electrolyte parameters).
Willingness to abstain from sexual intercourse or employ an approved method of contraception during the period of study drug administration and 6-week follow-up period.
Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

Exclusion Criteria:

Treatment with systemic aminoglycoside antibiotics within 3 months prior to start of study treatment.
Initiation of systemic corticosteroids therapy within 6 months prior to start of study treatment.
Change in systemic corticosteroid therapy (for example; change in type of drug, dose modification not related to body weight change, schedule modification, interruption, or reinitiation) within 3 months prior to start of study treatment.
Any change (initiation, change in type of drug, dose modification, schedule modification,interruption, discontinuation, or reinitiation) in prophylaxis/treatment for congestive heart failure (CHF) within 3 months prior to start of study treatment.
Ongoing use of coumarin-based anticoagulants (for example; warfarin), phenytoin, tolbutamide, or paclitaxel.
Prior therapy with ataluren.
Known hypersensitivity to any of the ingredients or excipients of the study drug.
Exposure to another investigational drug within 3 months prior to start of study treatment.
History of major surgical procedure within 6 weeks prior to start of study treatment.
Ongoing immunosuppressive therapy (other than corticosteroids).
Ongoing participation in any clinical trial (except for studies specifically approved by PTC Therapeutics).
Expectation of major surgical procedure (for example; scoliosis surgery) during the 12-month treatment period of the study.
Requirement for daytime ventilator assistance.
Uncontrolled clinical symptoms and signs of CHF (American College of Cardiology/American Heart Association Stage C or Stage D).
Prior or ongoing medical condition (for example; concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings (for example; lower limb injury that may affect 6MWT performance), electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.

Sites / Locations

University of California, Los Angeles
UC Davis Medical Center
Children's Hospital Colorado - Center for Cancer and Blood Disorders
Child Neurology Center of Northwest Florida
Rush University Medical Center
University of Iowa
University of Kansas Medical Center
Boston Children's Hospital
Children's Hospital Boston
University of Minnesota
Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
Columbia University College of Physicians & Surgeons
Cincinnati Children's Hospital Medical Center
Nationwide Children's Hospital
Oregon Health & Science University
The Children's Hospital of Philadelphia
Children's Hospital of Pittsburgh
Childrens Medical Center Dallas, Texas
Texas Children's Hospital
University of Utah
Seattle Children's Hospital - Childhood Cancer and Blood Disorders
The Children's Hospital at Westmead
The Royal Children's Hospital
UZ Leuven
Universidade Federal do Rio de Janeiro - Instituto de Puericultura e Pediatria Martagao Gesteira
Sao Paulo University -HC/FMUSP
Alberta Children's Hospital
British Columbia Children's Hospital
Children's Hospital of Western Ontario
Hospital Luis Calvo Mackenna
Hospital Clinico Universidad Catolica
University Hospital Brno
Motol University Hospital
Hospital de la Timone
CHU de Nantes
Hopital Necker - Enfants Malades
Groupe Hospitalier La Pitie-Salpetriere
University of Essen-Duisburg
University Hospital Medical Center Freiburg
Hadassah University Hospital
Policlinico Universitario G. Martino
Fondazione IRCS Ca Granda Ospedale Maggiore Policlinico di Milano
Bambino Gesu Hospital
U.O. Complessa di Neuropsichiatria Infantile
Seoul National University Hospital
Medical University of Warsaw
Hospital Sant Joan de Deu
Hospital Universitari i Politecnic la Fe
Queen Silvia Children's Hospital
Astrid Lindgren Childrens Hospital
CHUV Lausanne
Hacettepe Childrens Hospital
University College London Institute of Child Health
Royal Manchester Children's Hospital
The Newcastle upon Tyne Hospitals, NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

Ataluren

Arm Description

Participants will receive placebo matching to ataluren orally 3 times a day (TID) at morning, midday, and evening for 48 weeks.

Participants will receive ataluren suspension orally TID, 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.

Outcomes

Primary Outcome Measures

Change From Baseline in 6MWD at Week 48

The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline and Week 48 6MWD values are each the average of two valid 6MWD values, or a single available value if one was missing.

Secondary Outcome Measures

Time to 10 Percent (%) Persistent Worsening in 6MWD

The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Time to 10% persistent worsening in 6MWD <300 meters, >=300 to 400 meters, and >=400 meters was evaluated. For participants who did not have 10% 6MWD worsening or who were removed from study, time to 10% 6MWD worsening was censored at the time of the last 6MWD test. Participants who became non-ambulatory were considered to have 10% worsening.

Change From Baseline in Time to Walk/Run 10 Meters at Week 48

During the test for walking/running 10 meters, the method of walk/run used by the participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support from a person or with assistive device (full leg calipers [knee-ankle-foot orthoses ] or walker); 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression, a value of 30 seconds was used. A cumulative change from baseline data has been reported.

Change From Baseline in Time to Climb 4 Stairs at Week 48

During the test for stair-climbing, the method of climbing used by the participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. A cumulative change from baseline data has been reported.

Change From Baseline in Time to Descend 4 Stairs at Week 48

During the test for stair-descending, the method of descending used by the participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. A cumulative change from baseline data has been reported.

Percentage of Participants With Treatment-Emergent Adverse Events (AEs)

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. Treatment-emergent adverse event (TEAE) was defined as an adverse event that occurred or worsened in the period extending from first dose of study drug to 6 weeks after the last dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.

Full Information

NCT ID

NCT01826487

First Posted

March 26, 2013

Last Updated

July 17, 2020

Sponsor

PTC Therapeutics

1. Study Identification

Unique Protocol Identification Number

NCT01826487

Brief Title

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Acronym

ACT DMD

Official Title

A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Dystrophinopathy

Study Type

Interventional

2. Study Status

Record Verification Date

July 2020

Overall Recruitment Status

Completed

Study Start Date

March 26, 2013 (Actual)

Primary Completion Date

August 20, 2015 (Actual)

Study Completion Date

August 20, 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

PTC Therapeutics

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy in approximately 10-15 percent (%) of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability. The effect of ataluren on physical function, quality of life, and activities of daily living will be evaluated. This study will also provide additional information on the long-term safety of ataluren.

Detailed Description

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to determine the efficacy and safety of ataluren in participants with nmDMD.

6. Conditions and Keywords

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

Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn

Keywords

Duchenne muscular dystrophy, Dystrophinopathy, Nonsense mutation, Premature stop codon, Becker muscular dystrophy, DMD/BMD, PTC124, Ataluren

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

230 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Participants will receive placebo matching to ataluren orally 3 times a day (TID) at morning, midday, and evening for 48 weeks.

Arm Title

Ataluren

Arm Type

Experimental

Arm Description

Participants will receive ataluren suspension orally TID, 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.

Intervention Type

Drug

Intervention Name(s)

Ataluren

Other Intervention Name(s)

PTC124

Intervention Description

Ataluren will be administered as per the dose and schedule specified in the arm.

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo will be administered as per the schedule specified in the arm.

Primary Outcome Measure Information:

Title

Change From Baseline in 6MWD at Week 48

Description

Time Frame

Baseline, Week 48

Secondary Outcome Measure Information:

Title

Time to 10 Percent (%) Persistent Worsening in 6MWD

Description

The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Time to 10% persistent worsening in 6MWD <300 meters, >=300 to 400 meters, and >=400 meters was evaluated. For participants who did not have 10% 6MWD worsening or who were removed from study, time to 10% 6MWD worsening was censored at the time of the last 6MWD test. Participants who became non-ambulatory were considered to have 10% worsening.

Time Frame

Baseline to Week 48

Title

Change From Baseline in Time to Walk/Run 10 Meters at Week 48

Description

Time Frame

Baseline, Week 48

Title

Change From Baseline in Time to Climb 4 Stairs at Week 48

Description

Time Frame

Baseline, Week 48

Title

Change From Baseline in Time to Descend 4 Stairs at Week 48

Description

Time Frame

Baseline, Week 48

Title

Percentage of Participants With Treatment-Emergent Adverse Events (AEs)

Description

Time Frame

Baseline up to Week 54

Other Pre-specified Outcome Measures:

Title

Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 48

Description

Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one's head, stand on one's heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." The sum of these scores (except for 'raise one's head' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0.

Time Frame

Baseline, Week 48

Title

Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 48, as Assessed by a Standardized Survey Administered by Site Personnel

Description

Changes in activities of daily living and disease symptoms were captured via a DMD-specific survey administered by Site personnel. At screening or baseline, the participant and/or parent/caregiver were asked to identify any activities of daily living (for example, ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using the bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by the participant's DMD. At post-baseline visit (Week 48), the same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within the following categories: physical functioning; general energy level; cognition/school function; emotional/social functioning; and sleep. Changes from baseline were reported on a 5-point Likert scale: 1 (much worse), 2 (slightly worse), 3 (unchanged), 4 (slightly better), or 5 (much better).

Time Frame

Baseline, Week 48

Title

Change From Baseline in PODCI Transfers/Basic Mobility and Sports/Physical Functioning Scores at Week 48

Description

Changes in health-related quality of life (HRQL) were measured via the PODCI questionnaire that has been shown to correlate with disease progression and clinical outcome measures in DMD. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. The following PODCI domains were prespecified in the protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Sports/Physical Functioning domain assesses difficulty encountered in participating in more active recreational activities. Each domain was scored from 0 to 100, with 0 representing a poor outcome/worse health, while 100 representing the highest level of functioning and least pain.

Time Frame

Baseline, Week 48

Title

Ataluren Plasma Concentration

Description

Plasma samples for the determination of ataluren concentrations were analyzed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method with a lower limit of quantitation of 0.5 micrograms/milliliter (mcg/mL).

Time Frame

Weeks 8, 16, 24, 32, 40, and 48

Title

Study Drug Compliance

Description

Study drug compliance was assessed by quantification of used and unused study drug. Compliance was assessed in terms of the percentage of drug actually taken relative to the amount that should have been taken during the study.

Time Frame

Baseline to Week 48

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

7 Years

Maximum Age & Unit of Time

16 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Ability to provide written informed consent (parental/guardian consent if applicable)/assent per local requirements. Phenotypic evidence of dystrophinopathy based on the onset of characteristic clinical symptoms or signs (for example; proximal muscle weakness, waddling gait, and Gowers' maneuver) by 6 years of age, an elevated serum creatinine kinase level, and ongoing difficulty with walking. Documentation of the presence of a nonsense point mutation in the dystrophin gene as determined by gene sequencing from a laboratory certified by the College of American Pathologists (CAP), the Clinical Laboratory Improvement Act/Amendment (CLIA) or an equivalent organization. Documentation that a blood sample has been drawn for confirmation of the presence of a nonsense mutation in the dystrophin gene. Use of systemic corticosteroids (prednisone, prednisolone, or deflazacort) for a minimum of 6 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen (not related to body weight change) for a minimum of 3 months immediately prior to start of study treatment and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study. Valid Screening 6-minute walk distance (6MWD) greater than or equal to (≥) 150 meters. Valid Screening 6MWD must have been less than or equal to (≤) 80% of predicted for age and height. Results of the 2 Baseline 6MWD results must be determined as valid and results of the Day 2 Baseline 6MWD must be within 20% of the Day 1 Baseline 6MWD. Baseline 6MWD (mean of valid Day 1 and Day 2 values) must be no more than a 20% change from the valid Screening 6MWD. Confirmed screening laboratory values within the central laboratory ranges (hepatic, renal, and serum electrolyte parameters). Willingness to abstain from sexual intercourse or employ an approved method of contraception during the period of study drug administration and 6-week follow-up period. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions. Exclusion Criteria: Treatment with systemic aminoglycoside antibiotics within 3 months prior to start of study treatment. Initiation of systemic corticosteroids therapy within 6 months prior to start of study treatment. Change in systemic corticosteroid therapy (for example; change in type of drug, dose modification not related to body weight change, schedule modification, interruption, or reinitiation) within 3 months prior to start of study treatment. Any change (initiation, change in type of drug, dose modification, schedule modification,interruption, discontinuation, or reinitiation) in prophylaxis/treatment for congestive heart failure (CHF) within 3 months prior to start of study treatment. Ongoing use of coumarin-based anticoagulants (for example; warfarin), phenytoin, tolbutamide, or paclitaxel. Prior therapy with ataluren. Known hypersensitivity to any of the ingredients or excipients of the study drug. Exposure to another investigational drug within 3 months prior to start of study treatment. History of major surgical procedure within 6 weeks prior to start of study treatment. Ongoing immunosuppressive therapy (other than corticosteroids). Ongoing participation in any clinical trial (except for studies specifically approved by PTC Therapeutics). Expectation of major surgical procedure (for example; scoliosis surgery) during the 12-month treatment period of the study. Requirement for daytime ventilator assistance. Uncontrolled clinical symptoms and signs of CHF (American College of Cardiology/American Heart Association Stage C or Stage D). Prior or ongoing medical condition (for example; concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings (for example; lower limb injury that may affect 6MWT performance), electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Robert Spiegel, M.D.

Organizational Affiliation

PTC Therapeutics

Official's Role

Study Director

Facility Information:

Facility Name

University of California, Los Angeles

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

UC Davis Medical Center

City

Sacramento

State/Province

California

ZIP/Postal Code

95817

Country

United States

Facility Name

Children's Hospital Colorado - Center for Cancer and Blood Disorders

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

Child Neurology Center of Northwest Florida

City

Gulf Breeze

State/Province

Florida

ZIP/Postal Code

32561

Country

United States

Facility Name

Rush University Medical Center

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60612

Country

United States

Facility Name

University of Iowa

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

University of Kansas Medical Center

City

Kansas City

State/Province

Kansas

ZIP/Postal Code

66160

Country

United States

Facility Name

Boston Children's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Children's Hospital Boston

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

University of Minnesota

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

Facility Name

Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

Facility Name

Columbia University College of Physicians & Surgeons

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

Facility Name

Cincinnati Children's Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229-3039

Country

United States

Facility Name

Nationwide Children's Hospital

City

Columbus

State/Province

Ohio

ZIP/Postal Code

43209

Country

United States

Facility Name

Oregon Health & Science University

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

The Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Children's Hospital of Pittsburgh

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15224

Country

United States

Facility Name

Childrens Medical Center Dallas, Texas

City

Dallas

State/Province

Texas

ZIP/Postal Code

75207

Country

United States

Facility Name

Texas Children's Hospital

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

University of Utah

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84112

Country

United States

Facility Name

Seattle Children's Hospital - Childhood Cancer and Blood Disorders

City

Seattle

State/Province

Washington

ZIP/Postal Code

98105

Country

United States

Facility Name

The Children's Hospital at Westmead

City

Westmead

State/Province

New South Wales

ZIP/Postal Code

2145

Country

Australia

Facility Name

The Royal Children's Hospital

City

Parkville

State/Province

Victoria

ZIP/Postal Code

3052

Country

Australia

Facility Name

UZ Leuven

City

Leuven

ZIP/Postal Code

3000

Country

Belgium

Facility Name

Universidade Federal do Rio de Janeiro - Instituto de Puericultura e Pediatria Martagao Gesteira

City

Rio de Janeiro

ZIP/Postal Code

21.941-912

Country

Brazil

Facility Name

Sao Paulo University -HC/FMUSP

City

São Paulo

ZIP/Postal Code

05403-900

Country

Brazil

Facility Name

Alberta Children's Hospital

City

Calgary

State/Province

Alberta

ZIP/Postal Code

T3B 6A8

Country

Canada

Facility Name

British Columbia Children's Hospital

City

Vancouver

State/Province

British Columbia

ZIP/Postal Code

V6H 3V4

Country

Canada

Facility Name

Children's Hospital of Western Ontario

City

London

State/Province

Ontario

ZIP/Postal Code

N6A 2E3

Country

Canada

Facility Name

Hospital Luis Calvo Mackenna

City

Santiago

State/Province

Región Metropolitana

Country

Chile

Facility Name

Hospital Clinico Universidad Catolica

City

Santiago

ZIP/Postal Code

8330073

Country

Chile

Facility Name

University Hospital Brno

City

Brno

ZIP/Postal Code

635 00

Country

Czechia

Facility Name

Motol University Hospital

City

Praha

ZIP/Postal Code

150 06

Country

Czechia

Facility Name

Hospital de la Timone

City

Marseille

ZIP/Postal Code

13385

Country

France

Facility Name

CHU de Nantes

City

Nantes Cedex

ZIP/Postal Code

44093

Country

France

Facility Name

Hopital Necker - Enfants Malades

City

Paris

ZIP/Postal Code

75015

Country

France

Facility Name

Groupe Hospitalier La Pitie-Salpetriere

City

Paris

ZIP/Postal Code

75651

Country

France

Facility Name

University of Essen-Duisburg

City

Essen

ZIP/Postal Code

45122

Country

Germany

Facility Name

University Hospital Medical Center Freiburg

City

Freiburg

ZIP/Postal Code

79106

Country

Germany

Facility Name

Hadassah University Hospital

City

Jerusalem

ZIP/Postal Code

91240

Country

Israel

Facility Name

Policlinico Universitario G. Martino

City

Messina

State/Province

Sicily

ZIP/Postal Code

98125

Country

Italy

Facility Name

Fondazione IRCS Ca Granda Ospedale Maggiore Policlinico di Milano

City

Milan

ZIP/Postal Code

20122

Country

Italy

Facility Name

Bambino Gesu Hospital

City

Rome

ZIP/Postal Code

00165

Country

Italy

Facility Name

U.O. Complessa di Neuropsichiatria Infantile

City

Rome

ZIP/Postal Code

00168

Country

Italy

Facility Name

Seoul National University Hospital

City

Seoul

ZIP/Postal Code

110-744

Country

Korea, Republic of

Facility Name

Medical University of Warsaw

City

Warsaw

ZIP/Postal Code

85- 822

Country

Poland

Facility Name

Hospital Sant Joan de Deu

City

Barcelona

ZIP/Postal Code

08950

Country

Spain

Facility Name

Hospital Universitari i Politecnic la Fe

City

Valencia

ZIP/Postal Code

46026

Country

Spain

Facility Name

Queen Silvia Children's Hospital

City

Goteburg

ZIP/Postal Code

SE-416 85

Country

Sweden

Facility Name

Astrid Lindgren Childrens Hospital

City

Stockholm

ZIP/Postal Code

17176

Country

Sweden

Facility Name

CHUV Lausanne

City

Lausanne

ZIP/Postal Code

1011

Country

Switzerland

Facility Name

Hacettepe Childrens Hospital

City

Ankara

ZIP/Postal Code

06100

Country

Turkey

Facility Name

University College London Institute of Child Health

City

London

ZIP/Postal Code

WC1N 1EH

Country

United Kingdom

Facility Name

Royal Manchester Children's Hospital

City

Manchester

ZIP/Postal Code

M13 9WL

Country

United Kingdom

Facility Name

The Newcastle upon Tyne Hospitals, NHS Foundation Trust

City

Newcastle upon Tyne

ZIP/Postal Code

NE1 3BZ

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

34693725

Citation

Shieh PB, Elfring G, Trifillis P, Santos C, Peltz SW, Parsons JA, Apkon S, Darras BT, Campbell C, McDonald CM; Members of the Ataluren Phase IIb Study Group; Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group; Members of the ACT DMD Study Group; Members of the ACT DMD Clinical Evaluator Training Group. Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy. J Comp Eff Res. 2021 Dec;10(18):1337-1347. doi: 10.2217/cer-2021-0018. Epub 2021 Oct 25.

Results Reference

derived

PubMed Identifier

34383312

Citation

McDonald CM, Wei LJ, Flanigan KM, Elfring G, Trifillis P, Muntoni F; ACT DMD Clinical Evaluator Training Group; ACT DMD Study Group. Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment. Muscle Nerve. 2021 Nov;64(5):614-619. doi: 10.1002/mus.27396. Epub 2021 Sep 2.

Results Reference

derived

PubMed Identifier

32851872

Citation

Campbell C, Barohn RJ, Bertini E, Chabrol B, Comi GP, Darras BT, Finkel RS, Flanigan KM, Goemans N, Iannaccone ST, Jones KJ, Kirschner J, Mah JK, Mathews KD, McDonald CM, Mercuri E, Nevo Y, Pereon Y, Renfroe JB, Ryan MM, Sampson JB, Schara U, Sejersen T, Selby K, Tulinius M, Vilchez JJ, Voit T, Wei LJ, Wong BL, Elfring G, Souza M, McIntosh J, Trifillis P, Peltz SW, Muntoni F; PTC124-GD-007-DMD Study Group; ACT DMD Study Group; Clinical Evaluator Training Groups. Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy. J Comp Eff Res. 2020 Oct;9(14):973-984. doi: 10.2217/cer-2020-0095. Epub 2020 Aug 27.

Results Reference

derived

PubMed Identifier

28728956

Citation

McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E; Clinical Evaluator Training Group; ACT DMD Study Group. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2017 Sep 23;390(10101):1489-1498. doi: 10.1016/S0140-6736(17)31611-2. Epub 2017 Jul 17.

Results Reference

derived

Links:

URL

http://www.ptcbio.com

Description

Related Info

Learn more about this trial

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

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