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Duchenne Muscular Dystrophy clinical trials

View clinical trials related to Duchenne Muscular Dystrophy.

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NCT ID: NCT02740972 Completed - Clinical trials for Duchenne Muscular Dystrophy

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Start date: December 2016
Phase: Phase 2
Study type: Interventional

The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.

NCT ID: NCT02704325 Withdrawn - Clinical trials for Duchenne Muscular Dystrophy

Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2

Start date: April 2016
Phase: Phase 1/Phase 2
Study type: Interventional

The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients that will involve direct intramuscular injection to the extensor digitorum brevis muscle (EDB).

NCT ID: NCT02667483 Completed - Clinical trials for Duchenne Muscular Dystrophy

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Start date: October 2015
Phase: Phase 1/Phase 2
Study type: Interventional

This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage for subsequent studies.

NCT ID: NCT02636686 No longer available - Clinical trials for Duchenne Muscular Dystrophy

Extension Study of Drisapersen in DMD Subjects

Start date: n/a
Phase: N/A
Study type: Expanded Access

This is a phase IIIb, multi-centre, open-label extension study in male subjects with DMD who previously have been treated with drisapersen, aiming at assessing the safety and efficacy of drisapersen.

NCT ID: NCT02606136 Terminated - Clinical trials for Duchenne Muscular Dystrophy

Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

MISSION
Start date: January 4, 2016
Phase: Phase 2
Study type: Interventional

This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.

NCT ID: NCT02592941 Approved for marketing - Clinical trials for Duchenne Muscular Dystrophy

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Start date: n/a
Phase: N/A
Study type: Expanded Access

The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.

NCT ID: NCT02571205 Completed - Clinical trials for Duchenne Muscular Dystrophy

Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy

Start date: November 2015
Phase:
Study type: Observational

"Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy" is a single centre observational study that aims to follow the progress of 20 adolescents with Duchenne Muscular Dystrophy (DMD) and delayed puberty who are treated by the Newcastle muscle team, as they are treated with testosterone to induce puberty. The participants will all be treated with the standard stepwise regimen of testosterone injections every 4 weeks and data will be collected to help determine the effectiveness and tolerability of the current treatment regimen. The investigators will use the data to explore the effect of testosterone on pubertal development, growth, muscle strength and function, bone mineral density and body composition and characterise any side effects. Semi-structured interviews will also be carried out to learn the boys' views on the tolerability of the regimen. The study will last up to a maximum of 27 months in total for each participant, but may be less if they are happy with pubertal development before this time. It is important to do this study because from the investigator's limited experience in this group, testosterone treatment seems to be well liked and tolerated but the best treatment regimen to use remains unknown and there is no current consensus. It is not currently part of the standard of care in DMD but it would be important to include it if this study can show that it is an effective treatment for pubertal delay.

NCT ID: NCT02530905 Completed - Clinical trials for Duchenne Muscular Dystrophy

Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

Start date: October 8, 2015
Phase: Phase 1
Study type: Interventional

This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 45 skipping.

NCT ID: NCT02525302 Terminated - Clinical trials for Duchenne Muscular Dystrophy

HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

Start date: May 2015
Phase: Phase 2
Study type: Interventional

This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies. HALO-DMD-03 will provide safety and strength and function data on continuous long-term dosing. Data from this study will be used to inform the safety, tolerability, and dose selection for a future trial of HT-100 in boys with Duchenne Muscular Dystrophy (DMD).

NCT ID: NCT02516085 Completed - Clinical trials for Duchenne Muscular Dystrophy

Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin

Start date: January 2012
Phase: Phase 1
Study type: Interventional

The purpose of the study is to show that the intake of L-arginine and metformin improves muscle function and delays disease progression in patients with Duchenne's muscular dystrophy.