Clinical trials: Hereditary Muscular Dystrophy
Facioscapulohumeral Muscular Dystrophy
Bone Health in Facioscapulohumeral Muscular Dystrophy
This is a cross-sectional single visit study to determine bone health in individuals with FSHD.
- Trial name
- FSHD (NCT02413190)
- Where
- 2 sites in United States, Australia
- Volunteers
- 94 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Duchenne Muscular Dystrophy
Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02858362
- Testing
- Ezutromid
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 43 people
- Sponsor
- Summit Therapeutics
Duchenne Muscular Dystrophy
Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
While it has been known for many years that corticosteroid use benefits boys with Duchenne Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4 years of age.
- Trial name
- NCT02167217
- Testing
- Prednisolone
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- Washington University School of Medicine
Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with…
- Trial name
- NCT03362502
- Testing
- PF-06939926
- Where
- 22 sites in United States
- Volunteers
- 23 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
Phase I/II Study of SRP-4053 in DMD Patients
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.
- Trial name
- NCT02310906
- Testing
- SRP-4053
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Sarepta Therapeutics, Inc.
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
Duchenne Muscular Dystrophy
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients. There will be a modified intravascular limb infusion (ILI) procedure that will be used to sequentially deliver vector to…
- Trial name
- NCT03333590
- Testing
- rAAVrh74.MCK.GALGT2
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
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 (DMD)
Twice Weekly Steroids and Exercise as Therapy for DMD
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular…
- Trial name
- NCT04322357
- Testing
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
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…
- Trial name
- NCT02740972
- Testing
- NS-065/NCNP-01
- Where
- 7 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy
Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
The proposed clinical trial is an outgrowth of the safety record and functional improvement seen in the BMD follistatin gene therapy trial.
- Trial name
- NCT02354781
- Testing
- rAAV1.CMV.huFollistin344
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Jerry R. Mendell
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)
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…
- Trial name
- NCT05185622
- Testing
- Vamorolone
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients.
- Trial name
- NCT03760029
- Testing
- Visit frequency
- Where
- 8 sites in China
- Volunteers
- 312 people
- Sponsor
- Pfizer
Facioscapulohumeral Muscular Dystrophy
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Study A083-02 is a multi-center, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of locally-acting ACE-083 in patients with Facioscapulohumeral muscular dystrophy…
- Trial name
- NCT02927080
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 95 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Duchenne Muscular Dystrophy
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
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…
- Trial name
- NCT02530905
- Testing
- SRP-4045
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion. The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants…
- Trial name
- NCT03959189
- Testing
- ERX-963
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Expansion Therapeutics, Inc.
Myopathy, Myositis
Ultrasound in Muscle Biopsy
The objective of the proposed study is to evaluate the clinical utility of muscle ultrasonography for improving the diagnostic yield and safety of core muscle biopsy.
- Conditions
- Myopathy, Myositis, Muscular Dystrophy
- Trial name
- NCT01790178
- Testing
- Ultrasound
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Duke University
Congenital Myotonic Dystrophy
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
- Trial name
- NCT03692312
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 56 people
- Sponsor
- AMO Pharma Limited
Muscular Dystrophy (DMD)
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
- Trial name
- NCT02515669
- Testing
- RO7239361
- Where
- 12 sites in United States, Canada
- Volunteers
- 43 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy
- Trial name
- NCT01557400
- Testing
- Ataluren
- Where
- 21 sites in Australia, Belgium and 8 more
- Volunteers
- 94 people
- Sponsor
- PTC Therapeutics