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Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Primary Purpose

Duchenne Muscular Dystrophy

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
PF-06939926
Placebo
Placebo
PF-06939926
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Clinical trial, Gene therapy, Duchenne muscular dystrophy, fordadistrogene movaparvovec

Eligibility Criteria

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

Key inclusion criteria:

  1. Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing
  2. Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening
  3. Ambulatory, as assessed by protocol-specified criteria

Key exclusion criteria:

  1. Positive test performed by Pfizer for neutralizing antibodies to AAV9
  2. Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna™, EXONDYS 51™, VYONDYS 53™)
  3. Any prior treatment with gene therapy
  4. Any non-healed injury that may impact functional testing (eg NSAA)
  5. Abnormality in specified laboratory tests, including blood counts, liver and kidney function

  6. Any of the following genetic abnormalities in the dystrophin gene:

    1. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR
    2. A deletion that affects both exon 29 and exon 30;OR
    3. A deletion that affects any exons between 56-71, inclusive.

Sites / Locations

  • Arkansas Children's Hospital
  • Arkansas Children's
  • MRI Research Center
  • Reed Neurological Research Center
  • Ronald Reagan UCLA Medical Center (Investigational Drug Section)
  • Ronald Reagan UCLA Medical Center - Drug Information Center
  • UCLA (David Geffen School of Medicine)
  • UCLA Children's Heart Center
  • UCLA Clinical Lab Services
  • UCLA Mattel Children's Hospital
  • UCLA Medical Center
  • UCLA Outpatient Surgery Center
  • University of Iowa Hospitals and Clinics
  • KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway
  • KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow
  • University of Kansas Hospital - Investigational Pharmacy
  • University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room
  • University of Kansas Medical Center
  • Pediatric Cardiology
  • The Children's Hospital of Philadelphia
  • UPMC Children's Hospital of Pittsburgh
  • University of Utah Center for Clinical & Tranlational Science
  • University of Utah Imaging and Neurosciences Center
  • University of Utah Hospital & Clinics Investigational Drug Services
  • Primary Children's Hospital
  • University of Utah Clinical Neurosciences Center
  • University of Utah Hospital
  • Children's Specialty Group, PLLC Division of Child & Adolescent Neurology
  • Seattle Children's
  • The Children's Hospital at Westmead
  • The Royal Children's Hospital Melbourne
  • Perth Children's Hospital
  • UZ Gent
  • UZ leuven
  • Alberta Children's Hospital
  • Children's Hospital - London Health Sciences Centre
  • Children's Hospital - London Health Sciences Centre
  • Childrens Hospital of Eastern Ontario
  • The Hospital For Sick Children
  • CHU de Nantes- Hotel Dieu
  • Hopital Necker
  • Universitaetsklinikum Essen
  • Charité - Universitätsmedizin Berlin
  • Charité Universitaetsmedizin Berlin
  • Universitaetsklinikum Essen
  • Universitatsklinikum Essen
  • Hadassah University Medical Center, Ein Kerem
  • Schneider Children's Medical Center of Israel
  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
  • IRCCS Ospedale Pediatrico Bambino Gesù
  • Nagoya City University Hospital
  • Hyogo College of Medicine College Hospital
  • National Center of Neurology and Psychiatry
  • Pusan National University Yangsan Hospital
  • Seoul National University Hospital
  • Samsung Medical Center
  • Saint Petersburg State Paediatric Medical University
  • State Autonomous Healthcare Institution of Sverdlovsk Region Children's City Clinical Hospital No 9
  • Hospital Sant Joan de Déu
  • Hospital Universitario Vall d'Hebron
  • Hospital Universitari i Politecnic La Fe de Valencia
  • Inselspital, University Children's Hospital Berne
  • Universitaets-Kinderspital Zuerich
  • Kaohsiung Medical University Chung-Ho Memorial Hospital
  • National Taiwan University Hospital
  • Taipei Veterans General Hospital
  • The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria Infirmary
  • Alder Hey Children's NHS Foundation Trust
  • Great Ormond Street Institute of Child Health

Arms of the Study

Arm 1

Arm 2

Arm Type

Other

Other

Arm Label

Cohort 1

Cohort 2

Arm Description

Approximately two thirds of participants will be randomized to Cohort 1.

Approximately one third of participants will be randomized to Cohort 2.

Outcomes

Primary Outcome Measures

Change from Baseline in North Star Ambulatory Assessment (NSAA)
The NSAA is a 17-item test that measures gross motor function in children with Duchenne.

Secondary Outcome Measures

Change from Baseline in mini-dystrophin expression level in muscle
Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry (LC-MS).
Change from Baseline in distribution of mini-dystrophin expression in the muscle
Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence.
Change from Baseline in serum creatine kinase (CK)
Changes in the circulating levels of CK.
Number of skills gained based on the individual items of the NSAA.
To count the skills that each child gained, based on the individual items of the NSAA.
Number of skills improved or maintained based on the individual items of the NSAA
To count the skills that each child improved or maintained, based on the individual items of the NSAA.
Change from Baseline in the 10-meter run/walk test velocity
Velocity is calculated based on the time that it takes to complete the 10-meter run/walk test.
Change from Baseline in the rise from floor velocity
Velocity is calculated based on the time that it takes to the rise from floor.
Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale
The PODCI contains a list of questions to assess how each caregiver/child evaluates the child´s ability to to walk, stand, and perform activities of daily living.
Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrucment (PODCI): Sports and Physical Functioning Core Scale
The PODCI contains a list of questions to assess how each caregiver/child evaluates the child´s ability to perform recreational activities.

Full Information

First Posted
February 11, 2020
Last Updated
August 29, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT04281485
Brief Title
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Official Title
A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 5, 2020 (Actual)
Primary Completion Date
April 18, 2024 (Anticipated)
Study Completion Date
April 18, 2029 (Anticipated)

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
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
Detailed Description
The study will assess the efficacy of PF-06939926 gene therapy on ambulatory function while also monitoring its safety. Approximately 99 boys with DMD will be enrolled and randomly assigned to one of two groups: approximately two thirds will be in Cohort 1 and receive gene therapy at the start of the study; approximately one third will be in Cohort 2 and receive placebo at the start of the study and receive gene therapy after one year, as long as it remains safe to do so. The treatment (PF-06939926 gene therapy or placebo) will be given as an intravenous infusion lasting up to 2 hours. The study includes boys who are at least 4 years old and less than 8 years old (including 7 year olds up until their 8th birthday). All boys will need to be on a daily dose of glucocorticoids (prednisone, prednisolone, or deflazacort) for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening. The primary outcome of the study will be assessed at 52 weeks. All participants will be followed in the study for 5 years after treatment with gene therapy. The study medication, all medical tests associated with the study, and the visits to the study sites are free of charge. Participants will also be supported for travel costs associated with study visits.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Clinical trial, Gene therapy, Duchenne muscular dystrophy, fordadistrogene movaparvovec

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Model Description
Parallel up to the measurement of the primary outcome at Week 52. At the beginning of study Year 2 participants who were originally assigned to placebo will have the opportunity to receive PF-06939926. All participants will be followed for 5 years following treatment with PF-06939926.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
The study will be quadruple blind.
Allocation
Randomized
Enrollment
99 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1
Arm Type
Other
Arm Description
Approximately two thirds of participants will be randomized to Cohort 1.
Arm Title
Cohort 2
Arm Type
Other
Arm Description
Approximately one third of participants will be randomized to Cohort 2.
Intervention Type
Genetic
Intervention Name(s)
PF-06939926
Intervention Description
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
Intervention Type
Genetic
Intervention Name(s)
PF-06939926
Intervention Description
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2
Primary Outcome Measure Information:
Title
Change from Baseline in North Star Ambulatory Assessment (NSAA)
Description
The NSAA is a 17-item test that measures gross motor function in children with Duchenne.
Time Frame
Week 52
Secondary Outcome Measure Information:
Title
Change from Baseline in mini-dystrophin expression level in muscle
Description
Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry (LC-MS).
Time Frame
Week 52
Title
Change from Baseline in distribution of mini-dystrophin expression in the muscle
Description
Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence.
Time Frame
Week 52
Title
Change from Baseline in serum creatine kinase (CK)
Description
Changes in the circulating levels of CK.
Time Frame
Week 52
Title
Number of skills gained based on the individual items of the NSAA.
Description
To count the skills that each child gained, based on the individual items of the NSAA.
Time Frame
Week 52
Title
Number of skills improved or maintained based on the individual items of the NSAA
Description
To count the skills that each child improved or maintained, based on the individual items of the NSAA.
Time Frame
Week 52
Title
Change from Baseline in the 10-meter run/walk test velocity
Description
Velocity is calculated based on the time that it takes to complete the 10-meter run/walk test.
Time Frame
Week 52
Title
Change from Baseline in the rise from floor velocity
Description
Velocity is calculated based on the time that it takes to the rise from floor.
Time Frame
Week 52
Title
Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale
Description
The PODCI contains a list of questions to assess how each caregiver/child evaluates the child´s ability to to walk, stand, and perform activities of daily living.
Time Frame
Week 52
Title
Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrucment (PODCI): Sports and Physical Functioning Core Scale
Description
The PODCI contains a list of questions to assess how each caregiver/child evaluates the child´s ability to perform recreational activities.
Time Frame
Week 52

10. Eligibility

Sex
Male
Gender Based
Yes
Gender Eligibility Description
Male
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key inclusion criteria: Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening Ambulatory, as assessed by protocol-specified criteria Key exclusion criteria: Positive test performed by Pfizer for neutralizing antibodies to AAV9 Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna™, EXONDYS 51™, VYONDYS 53™) Any prior treatment with gene therapy Any non-healed injury that may impact functional testing (eg NSAA) Abnormality in specified laboratory tests, including blood counts, liver and kidney function Any of the following genetic abnormalities in the dystrophin gene: Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR A deletion that affects both exon 29 and exon 30;OR A deletion that affects any exons between 56-71, inclusive.
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
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Arkansas Children's
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
MRI Research Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Reed Neurological Research Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Ronald Reagan UCLA Medical Center (Investigational Drug Section)
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Ronald Reagan UCLA Medical Center - Drug Information Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
UCLA (David Geffen School of Medicine)
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
UCLA Children's Heart Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
UCLA Clinical Lab Services
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
UCLA Mattel Children's Hospital
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
UCLA Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
UCLA Outpatient Surgery Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway
City
Fairway
State/Province
Kansas
ZIP/Postal Code
66205
Country
United States
Facility Name
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Kansas Hospital - Investigational Pharmacy
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
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
Pediatric Cardiology
City
Prairie Village
State/Province
Kansas
ZIP/Postal Code
66208
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
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
University of Utah Center for Clinical & Tranlational Science
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
University of Utah Imaging and Neurosciences Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
University of Utah Hospital & Clinics Investigational Drug Services
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
Primary Children's Hospital
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Facility Name
University of Utah Clinical Neurosciences Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
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
Children's Specialty Group, PLLC Division of Child & Adolescent Neurology
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23510
Country
United States
Facility Name
Seattle Children's
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 Melbourne
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Perth Children's Hospital
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
UZ Gent
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
UZ leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Alberta Children's Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3B 6A8
Country
Canada
Facility Name
Children's Hospital - London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 4G5
Country
Canada
Facility Name
Children's Hospital - London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Facility Name
Childrens Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H8L1
Country
Canada
Facility Name
The Hospital For Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
CHU de Nantes- Hotel Dieu
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Hopital Necker
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Universitaetsklinikum Essen
City
Essen
State/Province
North Rhine-westphalia
ZIP/Postal Code
45147
Country
Germany
Facility Name
Charité - Universitätsmedizin Berlin
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Charité Universitaetsmedizin Berlin
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Universitaetsklinikum Essen
City
Essen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Universitatsklinikum Essen
City
Essen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Hadassah University Medical Center, Ein Kerem
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Facility Name
Schneider Children's Medical Center of Israel
City
Petach Tikvah
ZIP/Postal Code
4920235
Country
Israel
Facility Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
City
Roma
ZIP/Postal Code
00168
Country
Italy
Facility Name
IRCCS Ospedale Pediatrico Bambino Gesù
City
Rome
ZIP/Postal Code
00165
Country
Italy
Facility Name
Nagoya City University Hospital
City
Nagoya
State/Province
Aichi
ZIP/Postal Code
467-8602
Country
Japan
Facility Name
Hyogo College of Medicine College Hospital
City
Nishinomiya
State/Province
Hyogo
ZIP/Postal Code
663-8501
Country
Japan
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
ZIP/Postal Code
187-8551
Country
Japan
Facility Name
Pusan National University Yangsan Hospital
City
Yangsan-si
State/Province
Gyeongsangnam-do
ZIP/Postal Code
50612
Country
Korea, Republic of
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Samsung Medical Center
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
Saint Petersburg State Paediatric Medical University
City
Saint Petersburg
ZIP/Postal Code
194100
Country
Russian Federation
Facility Name
State Autonomous Healthcare Institution of Sverdlovsk Region Children's City Clinical Hospital No 9
City
Yekaterinburg
ZIP/Postal Code
620134
Country
Russian Federation
Facility Name
Hospital Sant Joan de Déu
City
Esplugues de Llobregat
State/Province
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Hospital Universitario Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital Universitari i Politecnic La Fe de Valencia
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Inselspital, University Children's Hospital Berne
City
Bern
ZIP/Postal Code
3010
Country
Switzerland
Facility Name
Universitaets-Kinderspital Zuerich
City
Zurich
ZIP/Postal Code
8032
Country
Switzerland
Facility Name
Kaohsiung Medical University Chung-Ho Memorial Hospital
City
Kaohsiung
ZIP/Postal Code
807
Country
Taiwan
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
Taipei Veterans General Hospital
City
Taipei
ZIP/Postal Code
11217
Country
Taiwan
Facility Name
The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria Infirmary
City
Newcastle upon Tyne
State/Province
England
ZIP/Postal Code
NE1 4LP
Country
United Kingdom
Facility Name
Alder Hey Children's NHS Foundation Trust
City
Liverpool
State/Province
Merseyside
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Facility Name
Great Ormond Street Institute of Child Health
City
London
ZIP/Postal Code
WCIN 1EH
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
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=C3391003
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

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