Clinical trials: Muscle Disorders
- Condition Muscle Disorders
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Duchenne Muscular Dystrophy
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
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.
- Trial name
- NCT02525302
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Akashi Therapeutics
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Heart Diseases, ASD - Atrial Septal Defect
Abbott Structural Heart Device Registry
The Abbott Structural Heart (SH) Registry is being conducted to confirm the safety and performance of Abbott's SH devices in a post-market, real-world setting.
- Conditions
- Heart Diseases, ASD - Atrial Septal Defect, VSD - Muscular Ventricular Septal Defect, PFO - Patent Foramen Ovale
- Trial name
- SH-Registry (NCT06590467)
- Testing
- Amplatzer™ Occlusion Devices, Epic™ Surgical Tissue Heart Valve devices
- Where
- 36 sites in United States, Denmark and 8 more
- Volunteers
- 2,500 people
- Sponsor
- Abbott Medical Devices
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of…
- Trial name
- NCT06644534
- Testing
- TTI-0102
- Where
- 2 sites in France, Netherlands
- Volunteers
- 9 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Fibrodysplasia Ossificans Progressiva
In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation (heterotopic ossification…
- Trial name
- NCT02521792
- Testing
- Palovarotene
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Congenital Myotonic Dystrophy
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who…
- Trial name
- REACH CDM X (NCT05004129)
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 76 people
- Sponsor
- AMO Pharma Limited
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
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
Cerebral Palsy
Individual Patient Expanded Access IND of Hope Biosciences Autologous Adipose-derived Mesenchymal Stem Cells for the Treatment of Cerebral Palsy
This is an Individual Patient Expanded Access Protocol of Autologous HB-adMSCs for the Treatment of Cerebral Palsy (CP) with the primary goal of treating 1 individual with CP who has exhausted all treatment options, his condition…
- Trial name
- NCT04029896
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Healthy Volunteer, Muscular Atrophy, Spinal
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
- Trial name
- NCT05575011
- Testing
- BIIB115
- Where
- 16 sites in Belgium, Canada and 7 more
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Biogen
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.
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients.
- Trial name
- NCT06769633
- Testing
- Givinostat Hydrochloride, Givinostat Hydrochloride - Cohort 2
- Where
- 9 sites in Belgium, Italy and 2 more
- Volunteers
- 18 people
- Sponsor
- Italfarmaco
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
Spinal Muscular Atrophy
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S.
- Trial name
- NCT07448610
- Testing
- Onasemnogene Abeparvovec-brve
- Where
- 6 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
DM1, Myotonic Dystrophy
Global Study of Del-desiran for the Treatment of DM1
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy…
- Conditions
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia
- Trial name
- HARBOR (NCT06411288)
- Testing
- AOC 1001 (del-desiran)
- Where
- 34 sites in United States, Canada and 8 more
- Volunteers
- 159 people
- Sponsor
- Avidity Biosciences, Inc.
Muscular Dystrophy, Duchenne, Ambulatory Care
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
- Trial name
- DELOS (NCT01027884)
- Testing
- Idebenone
- Where
- 23 sites in United States, Austria and 8 more
- Volunteers
- 65 people
- Sponsor
- Santhera Pharmaceuticals
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
Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Duchenne Muscular Dystrophy(DMD)
- Trial name
- NCT05338099
- Testing
- EN001
- Where
- South Korea
- Volunteers
- 7 people
- Sponsor
- ENCell