This is a multicenter, observational study of participants receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 360 participants across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include participants who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program) and who provide consent. Participants will be followed for at least 5 years from their date of enrollment. Safety and efficacy data will be collected in conjunction with routine visits conducted as per usual care. Although there are no protocol-mandated procedures, it is expected that physicians and other caregivers will follow published treatment guidelines and standards of care.
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Showing 36 clinical trials
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
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
Clinical Trial NCT ID:
NCT06597656
Location:
United States
Next Steps:
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
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
Clinical Trial NCT ID:
NCT06138639
Location:
United States, Canada, Italy
Next Steps:
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy
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
Clinical Trial NCT ID:
NCT06450639
Location:
United States, Italy, Poland, Spain
Next Steps:
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
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
Clinical Trial NCT ID:
NCT06817382
Location:
United States
Next Steps:
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD
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
Clinical Trial NCT ID:
NCT05683379
Location:
United States
Next Steps:
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
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
Clinical Trial NCT ID:
NCT05693142
Location:
United States, Canada
Next Steps:
An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
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
Clinical Trial NCT ID:
NCT06270719
Location:
United States
Next Steps:
Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
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
Clinical Trial NCT ID:
NCT05280730
Location:
United States
Next Steps:
Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
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
Clinical Trial NCT ID:
NCT06224660
Location:
United States
Next Steps:
NS-050/NCNP-03 in Boys With DMD (Meteor50)
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
Clinical Trial NCT ID:
NCT06053814
Location:
United States, Japan
Next Steps:
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
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
Clinical Trial NCT ID:
NCT05996003
Location:
United States
Next Steps:
Once Weekly Infant Corticosteroid Trial for DMD
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
Clinical Trial NCT ID:
NCT05412394
Location:
United States
Next Steps:
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.
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
Clinical Trial NCT ID:
NCT06564974
Location:
United States, Puerto Rico
Next Steps:
Trial of Cell Based Therapy for DMD
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
Clinical Trial NCT ID:
NCT06692426
Location:
United States
Next Steps:
Vasodilator and Exercise Study for DMD (VASO-REx)
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
Clinical Trial NCT ID:
NCT06290713
Location:
United States
Next Steps:
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
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
Clinical Trial NCT ID:
NCT05881408
Next Steps:
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
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
Clinical Trial NCT ID:
NCT04626674
Location:
United States
Next Steps:
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
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
Clinical Trial NCT ID:
NCT05126758
Location:
United States
Next Steps:
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
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
Clinical Trial NCT ID:
NCT05540860
Location:
United States
Next Steps:
A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
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
Clinical Trial NCT ID:
NCT05166109
Location:
United States, Italy
Next Steps:
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (MIS51ON)
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
Clinical Trial NCT ID:
NCT03992430
Next Steps:
AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
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
Clinical Trial NCT ID:
NCT04240314
Location:
United States
Next Steps:
Defining Endpoints in Becker Muscular Dystrophy
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
Clinical Trial NCT ID:
NCT05257473
Next Steps:
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
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
Clinical Trial NCT ID:
NCT03373968
Next Steps:
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
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
Clinical Trial NCT ID:
NCT04687020
Location:
United States, Canada
Next Steps:
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
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
Clinical Trial NCT ID:
NCT03368742
Location:
United States
Next Steps:
Open-label Extension of the HOPE-2 Trial
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
Clinical Trial NCT ID:
NCT04428476
Location:
United States
Next Steps:
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
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
Clinical Trial NCT ID:
NCT03340675
Location:
United States
Next Steps:
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping (EXPLORE44OLE)
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
Clinical Trial NCT ID:
NCT06244082
Location:
United States
Next Steps:
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
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
Clinical Trial NCT ID:
NCT05291091
Next Steps:
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
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
Clinical Trial NCT ID:
NCT06100887
Location:
United States
Next Steps:
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
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
Clinical Trial NCT ID:
NCT05524883
Location:
United States, Australia, Belgium, Canada, Ireland, Italy, Republic of Korea, Spain, United Kingdom
Next Steps:
Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
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
Clinical Trial NCT ID:
NCT03936894
Location:
United States
Next Steps:
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
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
Clinical Trial NCT ID:
NCT02500381
Next Steps:
Tadalafil as Adjuvant Therapy for DMD
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
Clinical Trial NCT ID:
NCT05195775
Location:
United States
Next Steps:
Twice Weekly Steroids and Exercise as Therapy for DMD
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
Clinical Trial NCT ID:
NCT04322357
Location:
United States
Next Steps:
