Duchenne Muscular Dystrophy

Condition Description

An inherited disorder of progressive muscular weakness, typically in boys.

Many people with muscular dystrophy have Duchenne syndrome. Girls can be carriers and mildly affected, but the disease typically affects boys.

Symptoms include frequent falls, trouble getting up or running, waddling gait, big calves, and learning disabilities.

Active Trials

  • TRIAL TITLE: A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants with DMD Mutations Amenable to Exon 44 Skipping

    DESCRIPTION: AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping.

    Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers.

    Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.

    SPONSOR: Avidity Biosciences

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: Explore 44 AOC 1044-CS1

    PHASE: 1/2

    STATUS: Active

    RECRUITING PATIENTS: No

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  • TRIAL TITLE: A Phase 2, Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.

    DESCRIPTION: 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 pharmacodynamic effect of AOC 1044 on dystrophin protein production in skeletal muscle as well as the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044.

    Participants from AOC 1044-CS1 Part B are eligible to enroll in AOC 1044-CS2 if they have satisfactorily completed AOC 1044-CS1.

    SPONSOR: Avidity Biosciences

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: AOC 1044-CS2

    PHASE: 2

    STATUS: Active

    RECRUITING PATIENTS: Yes

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  • TRIAL TITLE: A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3) (HOPE-3)

    DESCRIPTION: HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.

    SPONSOR: Capricor Inc.

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: CAP-1002-DMD-04

    PHASE: 3

    STATUS: Active

    RECRUITING PATIENTS: No
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  • TRIAL TITLE: CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD).

    DESCRIPTION: CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone over 4 weeks old who has been diagnosed with DMD or BMD or who is a carrier of DMD or BMD can join. Parents or legal guardians can sign up their child(ren).

    SPONSOR: CureDuchenne

    INDICATION: Duchenne Muscular Dystrophy/ Becker Muscular Dystrophy

    STUDY PROTOCOL: CD-2021-01

    PHASE: Observation

    STATUS: Active

    RECRUITING PATIENTS: Yes

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  • TRIAL TITLE: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)

    DESCRIPTION: The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

    The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).

    SPONSOR: Dyne Therapeutics

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: DYNE251-DMD-201

    PHASE: 1 & 2

    STATUS: Active

    RECRUITING PATIENTS: No

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  • TRIAL TITLE: Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

    DESCRIPTION: This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

    SPONSOR: Italfarmaco

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: DSC 14/2357/51

    PHASE: 3 LT OL

    STATUS: Active

    RECRUITING PATIENTS: No


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  • TRIAL TITLE: A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

    DESCRIPTION: This is a 2-part, multi-center, Phase 2 study to evaluate the effect of sevasemten (EDG-5506) on safety, pharmacokinetics and biomarkers of muscle damage in approximately 72 children with DMD treated with oral, once-daily sevasemten for 24 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), a 92-week open-label extension period (Part B), and a 2-week follow up period.

    SPONSOR: Edgewise Therapeutics, Inc.

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: EDG-5506-210

    PHASE: 2

    STATUS: Active

    RECRUITING PATIENTS: Yes

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  • TRIAL TITLE: A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys with Duchenne Muscular Dystrophy (DMD)

    DESCRIPTION: This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly.

    The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.

    SPONSOR: NS Pharma, Inc.

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: NS-089/NCNP-02-201

    PHASE: 2

    STATUS: Active

    RECRUITING PATIENTS: Yes

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  • TRIAL TITLE: Anti-AAV8 Antibody Assessment Study of Boys with Duchenne Muscular Dystrophy Aged 0 to <12 Years

    DESCRIPTION: This is a noninterventional screening study; no investigational product is administered in this study. Information collected in this study may be used to identify potential participants for DMD investigational gene therapy clinical trials. This study consists of a single phone/video interview, a single home health visit, and a single phone/video call for communication of AAV8 antibody test results.

    SPONSOR: REGENXBIO Inc.

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: RGX-202-0101

    PHASE: N/A

    STATUS: Active

    RECRUITING PATIENTS: Yes

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  • TRIAL TITLE: Two-Part Study for Dose Determination of SRP-5051 (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)

    DESCRIPTION: This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of SRP-5051 at MAD levels to determine doses to be administered in Part B, and 2) Part B will be conducted to further evaluate the SRP-5051 doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.

    SPONSOR: Sarepta Therapeutics, Inc.

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: Momentum 5051-201

    PHASE: 2

    STATUS: Active

    RECRUITING PATIENTS: No
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  • TRIAL TITLE: A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

    DESCRIPTION: This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be conducted for the selection of a high dose (high dose level 1 versus high dose level 2) and its comparison with the 30 milligrams/kilogram (mg/kg) dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

    SPONSOR: Sarepta Therapeutics, Inc.

    INDICATION: Duchenne Muscular Dystrophy

    STUDY PROTOCOL: MIS51ON 4658-402

    PHASE: 3b

    STATUS: Active

    RECRUITING PATIENTS: No
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