Clinical trials: Muscle Atrophy
Postpoliomyelitis Syndrome
A Randomized, Double Blind, Placebo Controlled Trial L-carnitine and Piracetam in the Treatment of Weakness, Muscle Fatigue and Muscle Pain in the Postpoliomyelitis Syndrome
This protocol aims to assess of L-carnitine and piracetam to relieve weakness, muscle fatigue and muscle pain in patients with Postpoliomyelitis Syndrome.
- Trial name
- NCT01549847
- Testing
- L-carnitine and piracetam
- Where
- Brazil
- Sponsor
- Biolab Sanus Farmaceutica
Duchenne Muscular Dystrophy
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT04906460
- Testing
- WVE-N531
- Where
- 6 sites in United States, Jordan and 1 more
- Volunteers
- 25 people
- Sponsor
- Wave Life Sciences USA, Inc.
Malnutrition, Sarcopenia
Evaluation of an Oral Nutritional Supplement Containing AN777
To evaluate the effect of an experimental oral nutritional supplement containing AN777 compared to another oral nutritional supplement in a malnourished, elderly population.
- Trial name
- NCT01191125
- Testing
- medical food with AN777, oral nutritional formula
- Where
- 23 sites in United States, Belgium and 6 more
- Volunteers
- 331 people
- Sponsor
- Abbott Nutrition
Duchenne Muscular Dystrophy
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD.
- Trial name
- NCT00758225
- Testing
- Idebenone
- Where
- Belgium
- Volunteers
- 21 people
- Sponsor
- Santhera Pharmaceuticals
Limb-Girdle Muscular Dystrophy, Type 2E
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
- Trial name
- NCT03652259
- Testing
- SRP-9003
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Knee Osteoarthritis, Disuse Atrophy
Effects of Kneehab 12-week Peri-operative Total Knee Arthroplasty
Determine the efficacy of neuromuscular electrical stimulation (NMES, Kneehab) in promoting accelerated recovery of quadriceps function in patients recovering from total knee arthroplasty (TKA) as measured by increases in…
- Trial name
- KneehabTKA (NCT01096524)
- Testing
- Standard Physiotherapy, Kneehab
- Where
- United Kingdom
- Volunteers
- 200 people
- Sponsor
- Bio-Medical Research, Ltd.
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA).
- Trial name
- CHERISH (NCT02292537)
- Testing
- Nusinersen
- Where
- 24 sites in United States, Canada and 8 more
- Volunteers
- 126 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
- Trial name
- NCT06288230
- Testing
- vesemnogene lantuparvovec
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Lantu Biopharma
Myotonic Dystrophy 1
Study of ATX-01 in Participants With DM1
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated.
- Trial name
- ArthemiR (NCT06300307)
- Testing
- ATX-01
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 56 people
- Sponsor
- ARTHEx Biotech S.L.
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III Non Ambulant
Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
Assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years
- Trial name
- NCT01302600
- Testing
- Olesoxime
- Where
- 22 sites in Belgium, France and 5 more
- Volunteers
- 165 people
- Sponsor
- Hoffmann-La Roche
Low Muscle Strength, Malnutrition
Efficacy of Oral Nutritional Supplement in Community-Dwelling Adults Following Acute Care
The study aims to determine the effects of Oral Nutritional Supplement (ONS) added to usual care on body weight compared with usual care alone in community-dwelling adults aged 55 years and older who are at risk of malnutrition…
- Trial name
- NCT07753239
- Testing
- ONS, Standard of Care (SOC)
- Where
- 7 sites in Thailand
- Volunteers
- 304 people
- Sponsor
- Abbott Nutrition
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
Safety and Efficacy of P-188 NF in DMD Patients
This is an open-label study to evaluate the safety, tolerability and efficacy of daily, subcutaneous dosing with P-188 NF (Carmeseal-MD™) in non-ambulatory boys with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03558958
- Testing
- P-188 NF
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Phrixus Pharmaceuticals, Inc.
Muscular Atrophy, Spinal
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
- Trial name
- PUPFISH (NCT05808764)
- Testing
- Risdiplam
- Where
- 13 sites in United States, Belgium and 6 more
- Volunteers
- 11 people
- Sponsor
- Hoffmann-La Roche
Limb Girdle Muscular Dystrophy
A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)
The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).
- Trial name
- NCT05906251
- Testing
- SRP-6004
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
Duchenne Muscular Dystrophy
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
- Trial name
- SIDEROS-E (NCT03603288)
- Testing
- idebenone 150 mg film-coated tablets
- Where
- 39 sites in United States, Austria and 7 more
- Volunteers
- 161 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT03532542
- Testing
- Casimersen, Golodirsen
- Where
- 50 sites in United States, Belgium and 11 more
- Volunteers
- 171 people
- Sponsor
- Sarepta Therapeutics, Inc.
Facioscapulohumeral Muscular Dystrophy, FSHD
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
- Trial name
- NCT07435129
- Testing
- Apitegromab
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis