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Muscular Dystrophy, Duchenne clinical trials

View clinical trials related to Muscular Dystrophy, Duchenne.

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NCT ID: NCT06274983 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

DMD- Interactive Virtual Reality Study

DMD-IVR
Start date: April 20, 2023
Phase: N/A
Study type: Interventional

This study will help determine if an Interactive Virtual Reality system can improve the physiotherapy of young patients with Duchenne muscular dystrophy (DMD).

NCT ID: NCT06241950 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

Start date: January 29, 2024
Phase: Phase 1
Study type: Interventional

This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.

NCT ID: NCT05938023 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy

Start date: May 18, 2023
Phase: Phase 2
Study type: Interventional

This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambulant boys with Duchenne Muscular Dystrophy aged 10 to <18 years old. The study includes a randomised, double-blind, placebo-controlled treatment period (Part A), followed by an open labelled treatment period (Part B).

NCT ID: NCT05661071 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

Neuropsychological Profiles of Children With Duchenne Muscular Dystrophy and Its Effects on Motor Functions

Start date: May 11, 2022
Phase:
Study type: Observational

This study was planned to determine neuropsychological profiles of children with Duchenne Muscular Dystrophy and investigation of its effects on motor functions & compare to typically developed peers.

NCT ID: NCT05514249 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

Treatment of a Single Patient With CRD-TMH-001

Start date: August 31, 2022
Phase: Phase 1
Study type: Interventional

The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.

NCT ID: NCT05429372 Active, not recruiting - Clinical trials for Muscular Dystrophy, Duchenne

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Start date: August 8, 2022
Phase: Phase 2
Study type: Interventional

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

NCT ID: NCT05185622 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Start date: March 21, 2022
Phase: Phase 2
Study type: Interventional

This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in steroid-naïve boys ages 2 to <4 years, and glucocorticoid-treated and currently untreated boys ages 7 to <18 years with DMD.

NCT ID: NCT05135663 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy (DMD)

Extension Study of NS-089/NCNP-02 in DMD

Start date: June 23, 2021
Phase: Phase 2
Study type: Interventional

This is the extension study of NS-089/NCNP-02 (Study NCNP/DMT02), which is designed to assess the safety, tolerability and efficacy of NS-089/NCNP-02 in patients with Duchenne muscular dystrophy (DMD).

NCT ID: NCT05096221 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

EMBARK
Start date: October 27, 2021
Phase: Phase 3
Study type: Interventional

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

NCT ID: NCT04906460 Active, not recruiting - Clinical trials for Duchenne Muscular Dystrophy

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Start date: September 28, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

This is a Phase 1b/2a open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 2 parts, Part A and Part B. Part A is complete.