Home ❯ Clinical Trials ❯ Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
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 15 years after treatment with gene therapy. Participants who received fordadistrogene movaparvovec in Pfizer studies C3391001 and C3391008 or are currently enrolled in Pfizer study C3391011 will be allowed to roll over into the long-term safety follow-up period of this study and will be considered Cohort 3.
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.
Study Status
Actively Recruiting
Active, Not Recruiting
Therapeutic Approach
Any Restoring or Replacing Dystrophin Reducing Inflammation Regulating Calcium Balance Improving Muscle Growth & Protection Improving Heart Function Enhancing Bone Health Observational
Type
Any Observational Investigational Open Label Extension
Eligible Sexes
Any Male Female Other
Diagnosis
Any Becker Duchenne Female carrier
Applied Filters:
4 Years to <9 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Excluding any deletion of exon 8 and/or 9
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
0 Years to <18 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Any deletion in exons 1 to 11 or 42 to 45 are excluded
Industry/Sponsor:
Solid Biosciences Inc.
Ambulation:
Varies by cohort
Steroid Use:
Cohorts 1,2,4,&5 - Stable course of steroids; Cohort 3 - N/A
Eligible Sexes:
Male
8 Years to <18 Years
Enhancing Bone Health
Actively Recruiting
Therapeutic Approach:
Enhancing Bone Health
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Hoffmann-La Roche
Ambulation:
Group 1 - No specific requirement, Group 2 - Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
2 Years to <5 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - variants in exons 18 to 58
Industry/Sponsor:
Insmed Gene Therapy LLC
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
0 Years to <12 Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
REGENXBIO Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
1 to <12 years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Part 1: Excluding variants in exons 1-17, Part 2 and 3: Excluding deletions or point mutations in exons 8, 9, and/or 10
Industry/Sponsor:
REGENXBIO Inc.
Ambulation:
Ambulatory
Steroid Use:
Varies by cohort and age
Eligible Sexes:
Male
4+ Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Excluding any deletion of exon 8 and/or 9
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
No specific requirement
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
3+ Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Virginia Commonwealth University
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
All
Next Steps:
10+ Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Red Nucleus Enterprise Solutions, LLC
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
5+ Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Columbia University
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
All
Next Steps:
5 Years to <63 Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
No specific requirement
Industry/Sponsor:
University of Florida
Ambulation:
Varies by cohort
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
18+ Years
Regulating Calcium Balance
Actively Recruiting
Therapeutic Approach:
Regulating Calcium Balance
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Sardocor Corp.
Ambulation:
No specific requirement
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
All ages
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Diagnosis of a neuromuscular disorder for self or family member
Industry/Sponsor:
Boston Children's Hospital
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
All
Next Steps:
4 years to <15 years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - amenable to exon 50 skipping
Industry/Sponsor:
NS Pharma, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
4 Years to <15 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 44 skipping
Industry/Sponsor:
NS Pharma, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
1 Month to 30 Months
Reducing Inflammation
Actively Recruiting
Therapeutic Approach:
Reducing Inflammation
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Nationwide Children's Hospital
Ambulation:
No specific requirement
Steroid Use:
Steroid-naive
Eligible Sexes:
Male
2+ Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Catalyst Pharmaceuticals, Inc.
Ambulation:
No specific requirement
Steroid Use:
Currently on treatment with Vamorolone (AGAMREE)
Eligible Sexes:
Male
All ages
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
No specific requirement
Industry/Sponsor:
Parent Project Muscular Dystrophy
Ambulation:
Either
Steroid Use:
No specific requirement
Eligible Sexes:
All
18+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
University of Minnesota
Ambulation:
Non-ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
All
6+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
University of Florida
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
10+ Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Vanderbilt University Medical Center
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
8 Years to 18+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Excluding any deletion of exon 8 and/or 9
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Cohort 1 - Non-ambulatory; Cohort 2 - Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
Location:
United States, Australia, Belgium, Germany, Hong Kong, Israel, Italy, Japan, Republic of Korea, Spain, Sweden, Taiwan, United Kingdom
2+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - variant criteria varies by cohort
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Varies by cohort
Steroid Use:
Varies by cohort
Eligible Sexes:
Male
10+ Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Capricor Inc.
Ambulation:
No specific requirement
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
4 Years to <10 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Edgewise Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Cohorts 1,2,3,4,5 - Stable course of steroids; Cohort 2NS - No steroids within 6 months
Eligible Sexes:
Male
18 Years to <65 Years
Reducing Inflammation
Active, Not Recruiting
Therapeutic Approach:
Reducing Inflammation
Variant Requirement:
Becker - in-frame mutation
Industry/Sponsor:
ReveraGen BioPharma, Inc.
Ambulation:
Ambulatory
Steroid Use:
Not currently using
Eligible Sexes:
Male
4 Years to <14 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - amenable to exon 51 skipping
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
Location:
United States, Colombia, Czechia, Denmark, France, Germany, Greece, Hungary, India, Ireland, Italy, Jordan, Republic of Korea, Mexico, Netherlands, New Zealand, Norway, Poland, Romania, Serbia, Slovenia, Spain, Switzerland, Taiwan, Turkey, United Kingdom
6 Months to <14 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Duplication exon 2
Industry/Sponsor:
Nationwide Children's Hospital
Ambulation:
Pre-ambulant (not yet walking) or ambulant
Steroid Use:
Age 4 years+: Stable course of steroids
Eligible Sexes:
Male
18+ Years
Observational
Active, Not Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Known female carrier and/or mother of a child with Duchenne or Becker
Industry/Sponsor:
Nationwide Children's Hospital
Ambulation:
No specific requirement
Steroid Use:
No current steroid treatment
Eligible Sexes:
Female
Next Steps:
6+ Years
Observational
Active, Not Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Becker - Variant requirement varies by cohort
Industry/Sponsor:
Virginia Commonwealth University
Ambulation:
Varies by cohort
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
2+ Years
Observational
Active, Not Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
No specific requirement
Industry/Sponsor:
The Emmes Company, LLC
Ambulation:
Either
Steroid Use:
No specific requirement
Eligible Sexes:
All
Next Steps:
6+ Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Italfarmaco
Ambulation:
Ambulatory or non-ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Location:
United States, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Serbia, Spain, United Kingdom
4+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 53 skipping
Industry/Sponsor:
NS Pharma, Inc.
Ambulation:
No specific requirement
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
4 Years to <18 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Solid Biosciences Inc.
Ambulation:
Ambulatory for children, non-ambulatory for adolescents
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
Next Steps:
10+ years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - excludes exon 44 skip-amenable or deletion of exons 3-7
Industry/Sponsor:
Capricor Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
All
7+ Years
Improving Heart Function
Active, Not Recruiting
Therapeutic Approach:
Improving Heart Function
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Cumberland Pharmaceuticals
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
7 Years to <28 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 44 skipping
Industry/Sponsor:
Avidity Biosciences, Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
12 Years to <51 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Becker - No specific requirement
Industry/Sponsor:
Edgewise Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Not currently using
Eligible Sexes:
Male
Location:
United States, Australia, Belgium, Denmark, France, Germany, Israel, Italy, Netherlands, New Zealand, Spain, United Kingdom
6 Years to <18 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Edgewise Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
4 Years to <17 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - amenable to exon 51 skipping
Industry/Sponsor:
Dyne Therapeutics
Ambulation:
Ambulatory or non-ambulatory <2 years
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
2+ Years
Reducing Inflammation
Active, Not Recruiting
Therapeutic Approach:
Reducing Inflammation
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Children's National Research Institute
Ambulation:
Ambulatory
Steroid Use:
Steroid-naive
Eligible Sexes:
Male
6 Years to <14 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 45 or 53 skipping
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
Location:
United States, Argentina, Australia, Belgium, Bulgaria, Canada, Czechia, Denmark, France, Germany, Greece, Hungary, India, Ireland, Israel, Italy, Republic of Korea, Mexico, Poland, Russian Federation, Serbia, Spain, Sweden, United Kingdom
7 Years to <13 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
University of Florida
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
5 Years to <10 Years
Reducing Inflammation
Active, Not Recruiting
Therapeutic Approach:
Reducing Inflammation
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
University of Florida
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male