Clinical trials: Muscle Atrophy
Sarcopenia
Identification of Relevant Biological, Imaging, Mobility and Clinical Markers for Clinical Research in Sarcopenia
The objective of this trial is to constitute a cohort of sarcopenic versus non-sarcopenic patients to validate the most relevant biological, imaging, mobility and clinical markers considered individually or in association for the…
- Trial name
- NCT04569487
- Testing
- Biopsy
- Where
- 2 sites in Belgium
- Volunteers
- 16 people
- Sponsor
- Artialis
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
LGMD2I
Study of BBP-418 in Patients With LGMD2I
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). This is an open label study to determine the safety and tolerability of ascending dose levels of BBP-418 in the…
- Trial name
- NCT04800874
- Testing
- BBP-418
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- ML Bio Solutions, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Sarcopenia
Clinical Trial of CG-BM1 Allogeneic Human Bone Marrow Mesenchymal Stem Cell Injection in the Treatment of Sarcopenia
1. To evaluate the safety and tolerability of CG-BM1 for the treatment of patients with Sarcopenia. 2. To observe the preliminary effectiveness of CG-BM1 in treating Sarcopenia patients.
- Trial name
- CGBM1-SAR-01 (NCT07865676)
- Testing
- CG-BM1- 5.0 × 10^7 cells, CG-BM1-1.0 × 10^8 cells
- Where
- China
- Volunteers
- 30 people
- Sponsor
- GuangZhou Cellgenes Biotechnology Co., Ltd.
Duchenne Muscular Dystrophy
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the…
- Trial name
- NCT01753804
- Testing
- Observational study
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 269 people
- Sponsor
- BioMarin Pharmaceutical
Sarcopenia
Clinical Trial to Evaluate the Efficacy of Neuromuscular Electrical Stimulation Therapy in Patients With Sarcopenia
The goal of this clinical trial is to exploratorily evaluate the safety and efficacy of a home-based lower extremity muscle strengthening and physical performance enhancement program using Neuromuscular Electrical Stimulation…
- Trial name
- NMES (NCT07668258)
- Testing
- neuromuscular electrical stimulation
- Volunteers
- 40 people
- Sponsor
- Exosystems
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
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
FSHD - Facioscapulohumeral Muscular Dystrophy
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Muscular Dystrophy (FSHD) 1 or 2.
- Trial name
- NCT07086521
- Testing
- ULSC
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Restem, LLC.
Diabetic Neuropathies
Efficacy and Safety of TAK-583 in Subjects With Diabetic Peripheral Neuropathy
The purpose of this study is to determine the safety and effectiveness of TAK-583, once daily (QD), in the treatment of neuropathy caused by diabetes mellitus.
- Trial name
- NCT00760955
- Testing
- TAK-583
- Where
- 38 sites in United States, Canada
- Volunteers
- 338 people
- Sponsor
- Takeda