AOC 1044-CS2 (EXPLORE44-OLE) is an open label, extension study to Part B of AOC 1044-CS1 (EXPLORE44). AOC 1044-CS2 is designed to evaluate the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044.

All participants who enroll in AOC 1044-CS2 will receive active treatment. The treatment period is 2 years with IV dosing every 6 weeks.

Once participants have completed active treatment, they will be followed through a 3-month safety follow-up period. The sponsor may extend active treatment beyond 2 years at a future timepoint.

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Showing 33 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

Actively Recruiting
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)

Actively Recruiting
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
Next Steps:

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy

Actively Recruiting
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
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)

Actively Recruiting
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

Actively Recruiting
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)

Actively Recruiting
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
Next Steps:

An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy

Actively Recruiting
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:

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

Actively Recruiting
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)

Actively Recruiting
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
Next Steps:

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Actively Recruiting
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

Actively Recruiting
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

Trial of Cell Based Therapy for DMD

Actively Recruiting
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)

Actively Recruiting
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

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)

Active, Not Recruiting
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)

Active, Not Recruiting
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

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Active, Not Recruiting
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)

Active, Not Recruiting
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)

Active, Not Recruiting
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
Next Steps:

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (MIS51ON)

Active, Not Recruiting
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.

Active, Not Recruiting
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:

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Active, Not Recruiting
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)

Active, Not Recruiting
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
Next Steps:

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Active, Not Recruiting
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

Active, Not Recruiting
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

Active, Not Recruiting
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)

Active, Not Recruiting
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)

Active, Not Recruiting
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

Active, Not Recruiting
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

Active, Not Recruiting
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
Next Steps:

Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy

Active, Not Recruiting
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)

Active, Not Recruiting
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

Active, Not Recruiting
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

Active, Not Recruiting
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