This study will make use of the existing NorthStar network to collect a wider range of clinically relevant data from paediatric and adult DMD patients, to describe more completely the natural history of the disease in different domains and across different life stages and disease phases. Data will be collected using assessment techniques and outcome measures appropriate to the major phases of the disease, and which are in some cases not currently part of the standard of care for DMD in the UK. The National Neuromuscular Database will continue to be used to collect and store these data. This study will allow the network to continue to describe the current natural history in DMD prospectively, with more robust subject ascertainment and completeness. This will help meet the current need for contemporary natural history data for the evaluation of new therapies, and further research in DMD.
Explore Clinical Trials
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Showing 39 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:
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
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
Clinical Trial NCT ID:
NCT06839469
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:
Molecular Analysis of Patients With Neuromuscular Disease
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
Clinical Trial NCT ID:
NCT00390104
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:
Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD
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
Clinical Trial NCT ID:
NCT02972580
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:
