Clinical trials: Skeletal Myopathy
Age-related Muscle Decline
CALM-AF-AI: Counteracting Age-related Loss of Muscle With AAV-Follistatin Combined With Angiogenesis-Inducing VEGF Plasmid Gene Therapy
This Phase 1/2a, open-label, non-randomized study is designed to evaluate the safety and tolerability of intramuscular AAV9-Follistatin gene therapy administered either as monotherapy or in combination with a VEGF-encoding…
- Trial name
- CALM-AF-AI (NCT07443826)
- Testing
- AAV9-Follistatin gene therapy, VEGF Plasmid
- Where
- Honduras
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Unlimited Biotechnology LLC
Fibrodysplasia Ossificans Progressiva
A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called andecaliximab. The study will include pediatric and adult patients with fibrodysplasia ossificans progressiva (FOP).
- Trial name
- ANDECAL (NCT06508021)
- Testing
- Andecaliximab
- Where
- 3 sites in United States
- Volunteers
- 92 people
- Sponsor
- Ashibio Inc
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals 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
Post-Acute Sequelae of SARS-CoV-2 (PASC) Infection
Efficacy, Safety, Tolerability of AXA1125 in Fatigue After COVID-19 Infection
This study will compare the effects of AXA1125, an orally active mixture of amino acids, compared to placebo, on improving muscle function (metabolism) following moderate exercise in subjects with fatigue-Predominant Post-Acute…
- Trial name
- NCT05152849
- Testing
- AXA1125
- Where
- United Kingdom
- Volunteers
- 41 people
- Sponsor
- Axcella Health, Inc
Fibrodysplasia Ossificans Progressiva (FOP)
A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)
Primary Objective(s): To describe the demographic characteristics of people living with FOP and a matched cohort of non-FOP people living in the United States (U.S.) To describe the prevalence of clinical characteristics of…
- Trial name
- NCT06064656
- Testing
- Non-Interventional
- Where
- United States
- Volunteers
- 131 people
- Sponsor
- Regeneron Pharmaceuticals
Musculoskeletal Disorders, Skeletal Disorder
Observational Prospective PMCF Study to Confirm Performance and Safety of Intelect® Devices in Real World
DJO UK Ltd (ENOVIS) is conducting this study to confirm the performance and safety of the Chattanooga Intelect® devices in real world use.
- Conditions
- Musculoskeletal Disorders, Skeletal Disorder, Muscle Disorder
- Trial name
- INTELECT (NCT06809283)
- Where
- 4 sites in France, Italy and 1 more
- Volunteers
- 144 people
- Sponsor
- DJO UK Ltd
Danon Disease
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2…
- Trial name
- NCT03882437
- Testing
- RP-A501
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Fibrodysplasia Ossificans Progressiva, FOP
A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP.
- Trial name
- OPTIMA-2 (NCT07559513)
- Testing
- garetosmab
- Volunteers
- 18 people
- Sponsor
- Regeneron Pharmaceuticals