Clinical trials: Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT07673809
- Testing
- GNR-097
- Where
- 6 sites in Belarus, Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Duchenne Muscular Dystrophy
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- Trial name
- NCT06280209
- Testing
- BMN 351
- Where
- 7 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
NS-050/NCNP-03 in Boys With DMD (Meteor50)
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD…
- Trial name
- NCT06053814
- Testing
- NS-050/NCNP-03
- Where
- 19 sites in United States, Canada and 3 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD
- Trial name
- FORZETTO (NCT07608432)
- Testing
- Zeleciment Rostudirsen (DYNE-251)
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Dyne Therapeutics
Duchenne Muscular Dystrophy
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)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study.
- Trial name
- ENVISION (NCT05881408)
- Testing
- delandistrogene moxeparvovec
- Where
- 46 sites in United States, Australia and 12 more
- Volunteers
- 148 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients.
- Trial name
- NCT07188012
- Testing
- SPOT-03
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
Duchenne Muscular Dystrophy (DMD)
Can External Vagus Nerve Stimulation Reduce Systemic Levels of Inflammatory Mediators in Duchenne Muscular Dystrophy Patients?
The intended investigation is a pilot study to evaluate the safety and efficacy of a novel transcutaneous auricular vagus nerve stimulator system, termed TRAVAGUS ONE, to reduce systemic levels of inflammatory mediators in…
- Trial name
- Travagus One (NCT07543302)
- Testing
- Transcutaneous auricular vagus nerve stimulation
- Where
- Sweden
- Volunteers
- 20 people
- Sponsor
- taVNS AB
Duchenne Muscular Dystrophy
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy
This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone.
- Trial name
- GUARDIAN (NCT06713135)
- Testing
- vamorolone 40 mg/mL oral suspension
- Where
- 18 sites in Belgium, Czechia and 7 more
- Volunteers
- 80 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
Long Term Follow-up for RGX-202
RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel…
- Trial name
- NCT06491927
- Testing
- No Intervention
- Where
- 5 sites in United States
- Volunteers
- 66 people
- Sponsor
- REGENXBIO Inc.
Duchenne Muscular Dystrophy Cardiomyopathy, Cardiomyopathy, Dilated
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young…
- Trial name
- DMD (NCT03340675)
- Testing
- Ifetroban
- Where
- 10 sites in United States
- Volunteers
- 46 people
- Sponsor
- Cumberland Pharmaceuticals
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Video Assessment Registry
ARISE is a prospective and longitudinal clinical study of individuals with Duchenne Muscular Dystrophy (DMD) aimed at creating a database of functional motor abilities in this population to support validation efforts of the…
- Trial name
- ARISE (NCT05712447)
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- The Emmes Company, LLC
Duchenne Muscular Dystrophy
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531…
- Trial name
- NCT07209332
- Testing
- WVE-N531
- Where
- 3 sites in Jordan, United Kingdom
- Volunteers
- 175 people
- Sponsor
- Wave Life Sciences USA, Inc.
Muscular Dystrophy, Duchenne
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will…
- Trial name
- MIS51ON (NCT03992430)
- Testing
- Eteplirsen
- Where
- 59 sites in United States, Colombia and 24 more
- Volunteers
- 160 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Duchenne
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety…
- Trial name
- NCT02369731
- Where
- 71 sites in Austria, Brazil and 14 more
- Volunteers
- 316 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants
The purpose of this trial is to evaluate the safety, tolerability, and Pharmacokinetics (PK) of a single oral dose of GRT6019 in healthy male participants, including food effect.
- Trial name
- NCT07317063
- Testing
- GRT6019
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Duchenne Muscular Dystrophy
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study.
- Trial name
- EXPEDITION (NCT05967351)
- Testing
- delandistrogene moxeparvovec
- Where
- 38 sites in United States, Belgium and 7 more
- Volunteers
- 400 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
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.
- Trial name
- NCT03373968
- Testing
- Givinostat
- Where
- 39 sites in United States, Belgium and 9 more
- Volunteers
- 206 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy
Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in…
- Trial name
- NCT03863119
- Testing
- Vamorolone
- Where
- 12 sites in United States, Canada and 1 more
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT1-EDO51 (NCT06079736)
- Testing
- PGN-EDO51
- Where
- 5 sites in Canada
- Volunteers
- 7 people
- Sponsor
- PepGen Inc