Clinical trials: Muscle Atrophy, open to volunteers
SMARD1, CMT2S
Gene Therapy for IGHMBP2-Related Diseases
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.
- Trial name
- NCT05152823
- Testing
- Gene Therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Megan Waldrop
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 (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
SMA - Spinal Muscular Atrophy
An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)
An Open Label Individual Patient (OLIP) Study of Nusinersen (BIIB058) in a Participant with Spinal Muscular Atrophy Who Previously Participated in an Investigational Study with Nusinersen
- Trial name
- NCT07846696
- Testing
- Nusinersen Injectable Product
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Liver Transplantation, Muscle Loss
Impact of Skeletal Muscle Quality and Loss on the Outcome of Liver Transplantation
CT imaging-based skeletal muscle assessment has been found to predict the outcomes of many diseases. Previous evidence revealed that pre-transplant muscle quality and post-transplant muscle loss were associated with transplant…
- Conditions
- Liver Transplantation, Muscle Loss, Muscle Quality
- Trial name
- NCT06188273
- Volunteers
- 800 people
- Sponsor
- Zhejiang University
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Registry for Duchenne and Becker Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- FAIR-DMD (NCT07402122)
- Where
- 30 sites in Austria, Germany
- Volunteers
- 1,500 people
- Sponsor
- Dr. Andreas Ziegler
Spinal Amyotrophy
SMAdult - Monitoring of Movement on Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare neuromuscular disease causing progressive muscle weakness related to degeneration of motor neurons.
- Trial name
- SMAdult (NCT07894185)
- Testing
- wearable sensors
- Where
- Belgium
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Myotonic Dystrophy 1
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared…
- Trial name
- FREEDOM2-DM1 (NCT06667453)
- Testing
- PGN-EDODM1
- Where
- 12 sites in Canada, New Zealand and 2 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Facioscapulohumeral Muscular Dystrophy, FSHD
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1
- Trial name
- FORTITUDE-3 (NCT07038200)
- Testing
- AOC-1020
- Where
- 46 sites in United States, Canada and 8 more
- Volunteers
- 200 people
- Sponsor
- Avidity Biosciences, Inc.
Spinal Muscular Atrophy
Neonatal Spinal Muscular Atrophy (SMA) Screening
Parents or legal guardian of neonates who signed agreement will receive SMA screening test if their neonates are affected with SMA.
- Trial name
- SMA (NCT03217578)
- Testing
- SMA Newborn Screening
- Where
- Taiwan
- Volunteers
- 250,000 people, healthy volunteers welcome
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Duchenne Muscular Dystrophy
The U.K. NorthStar Clinical Network
The goal of this natural history study is to capture the natural history of Duchenne Muscular Dystrophy (DMD) in children and adults in the United Kingdom. Children and adults with DMD will be invited to join.
- Trial name
- NS (NCT06711692)
- Where
- 26 sites in United Kingdom
- Volunteers
- 300 people
- Sponsor
- University College, London
Duchenne Muscular Dystrophy (DMD)
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
- Trial name
- NCT06900049
- Testing
- LE051
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases.
- Conditions
- Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Becker, Congenital Myopathy
- Trial name
- ARNseq-Musc (NCT06833489)
- Testing
- ARN extraction from muscle biopsies
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
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
Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim
Duchenne Muscular Dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death.
- Trial name
- NCT06093100
- Testing
- wearable technology
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Vanderbilt University Medical Center
Duchenne Muscular Dystrophy
Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy
The alternate-day regimen is hypothesized to reduce hypothalamic-pituitary-adrenal (HPA) axis suppression and decrease steroid-related toxicity.
- Trial name
- NCT07787689
- Testing
- Alternate-day prednisolone, Intermittent prednisolone
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- Muhammad Aamir Latif
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, Gamification
Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy
This research aims to improve the quality of life, occupational performance, occupational satisfaction and emotional health of young people with Duchenne muscular dystrophy compared to the classical occupational therapy program.
- Conditions
- Duchenne Muscular Dystrophy, Gamification, Occupational Therapy
- Trial name
- DMD (NCT06402942)
- Testing
- Gamified occupational therapy programme, Classic occupational therapy programme
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Başak Çağla Arslan
Duchenne Muscular Dystrophy, Duchenne Disease
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
This pilot study will investigate the potential efficacy of VM100, a nutritional supplement specifically formulated for patients with DMD, on quality of life and physical symptoms.
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD
- Trial name
- NCT07766980
- Testing
- VM100 Nutritional Supplement
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Florida