Clinical trials: Duchenne
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
- Trial name
- ENDEAVOR (NCT04626674)
- Testing
- delandistrogene moxeparvovec
- Where
- 12 sites in United States
- Volunteers
- 83 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
- Trial name
- ENVOL (NCT06128564)
- Testing
- delandistrogene moxeparvovec
- Where
- 7 sites in Belgium, France and 4 more
- Volunteers
- 13 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory…
- Trial name
- SHIELD DMD (NCT06450639)
- Testing
- Satralizumab
- Where
- 18 sites in United States, Denmark and 4 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
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 deramiocel (CAP-1002) in study participants with Duchenne…
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-3 (NCT05126758)
- Testing
- Deramiocel (CAP-1002)
- Where
- 20 sites in United States
- Volunteers
- 106 people
- Sponsor
- Capricor Inc.
Duchenne Muscular Dystrophy
A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
The purpose of this study is to evaluate the efficacy and safety of TAS-205 in patients with Duchenne muscular dystrophy
- Trial name
- NCT04587908
- Testing
- TAS-205 [Ambulatory Cohort] [Non-ambulatory Cohort]
- Where
- 5 sites in Japan
- Volunteers
- 104 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Duchenne Muscular Dystrophy
Study of UC-MSC in DMD Patients
The primary objective of this study is to provide UC-MSC treatment to patients with DMD. Secondary objectives will be to further evaluate treatment-related adverse events as well as changes in DMD-related functional…
- Trial name
- NCT06579352
- Testing
- Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- MED Institute Inc.
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-44 (NCT07037862)
- Testing
- ENTR-601-44
- Where
- 14 sites in Belgium, Italy and 2 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-45 (NCT07038824)
- Testing
- ENTR-601-45
- Where
- 15 sites in Belgium, Italy and 3 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Duchenne Muscular Dystrophy (DMD)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
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…
- Trial name
- DELIVER (NCT05524883)
- Testing
- DYNE-251
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 86 people
- Sponsor
- Dyne Therapeutics
Duchenne Muscular Dystrophin (DMD)
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys.
- Trial name
- MUSCLE (NCT06594094)
- Testing
- HG302
- Where
- China
- Volunteers
- 4 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
DMD, Duchenne Muscular Dystrophy
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
- Conditions
- DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
- Trial name
- EXPLORE44OLE (NCT06244082)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 39 people
- Sponsor
- Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to <18 years) patients with DMD.
- Trial name
- ULYSSES (NCT05933057)
- Testing
- Givinostat
- Where
- 29 sites in Belgium, Canada and 10 more
- Volunteers
- 138 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy.
- Trial name
- NCT04281485
- Testing
- PF-06939926
- Where
- 53 sites in United States, Australia and 13 more
- Volunteers
- 114 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
Duchenne Muscular Dystrophy
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05683379
- Testing
- AAV8 DetectCDx
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- REGENXBIO Inc.
Duchene Muscular Dystrophy
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine…
- Trial name
- PROVIDUS (NCT07127978)
- Where
- 9 sites in United States
- Volunteers
- 300 people
- Sponsor
- ITF Therapeutics LLC
Duchenne Muscular Dystrophy (DMD)
Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)
This is a study of investigational medicines ENTR-601-44 and ENTR-601-45 designed to evaluate the long-term safety and tolerability of study drugs in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- ELEVATE-LTE (NCT07682129)
- Testing
- ENTR-601-44, ENTR-601-45
- Where
- 17 sites in Belgium, Italy and 3 more
- Volunteers
- 80 people
- Sponsor
- Entrada Therapeutics, Inc.