Clinical trials: Atrophy, Muscular
Duchenne Muscular Dystrophy
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT02329769
- Testing
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy (DMD)
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
This is an open-label long-term safety and efficacy study of orally administered SAT-3247 in patients with DMD including those who previously participated in SAT-3247-CL-101.
- Trial name
- NCT06867107
- Testing
- SAT-3247
- Where
- 6 sites in United States, Australia
- Volunteers
- 30 people
- Sponsor
- Satellos Bioscience, Inc.
SMA
EAP of Apitegromab for Patients With Spinal Muscular Atrophy
The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency .
- Trial name
- NCT06877689
- Testing
- Apitegromab
- Sponsor
- Scholar Rock, Inc.
Duchenne Muscular Dystrophy
Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage…
- Trial name
- NCT02667483
- Testing
- DS-5141b
- Where
- 2 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Duchenne Muscular Dystrophy
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01847573
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Processa Pharmaceuticals
Muscular Atrophy, Spinal
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
The study is a randomized, single oral dose, crossover study in up to three parts to investigate the relative bioavailability and bioequivalence of two different formulations of risdiplam 5 mg (dispersible tablets) versus the…
- Trial name
- NCT04718181
- Testing
- risdiplam, omeprazole
- Where
- 4 sites in United States
- Volunteers
- 131 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Muscular Atrophy, Spinal
A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).
- Trial name
- NCT07474311
- Where
- Czechia
- Volunteers
- 213 people
- Sponsor
- Novartis Pharmaceuticals
Sporadic Inclusion Body Myositis
Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients
This study evaluated the efficacy, safety and tolerability of multiple doses of bimagrumab/BYM338 vs placebo, when administered intravenously (i.v.), on physical function, muscle strength, and mobility in patients with sporadic…
- Trial name
- RESILIENT (NCT01925209)
- Testing
- BYM338/bimagrumab
- Where
- 38 sites in United States, Australia and 8 more
- Volunteers
- 251 people
- Sponsor
- Novartis Pharmaceuticals
Ulcerative Colitis (Disorder), Crohn Disease
Partial Enteral Nutrition to Prevent Weight Loss and Sarcopenia in IBD Patients at Nutritional Risk - SIMBA
This is a multicenter, open-label, randomized two-arm interventional nutritional study evaluating the effects of partial enteral nutrition (LH VIOLA) in patients with Inflammatory Bowel Disease (IBD) at risk of malnutrition.
- Conditions
- Ulcerative Colitis (Disorder), Crohn Disease, Malnutrition or Risk of Malnutrition, IBD
- Trial name
- SIMBA (NCT07433946)
- Testing
- LH VIOLA Supplementation
- Where
- 4 sites in Italy
- Volunteers
- 146 people
- Sponsor
- Lionhealth Srl Società Benefit
Muscular Dystrophy, Duchenne
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in…
- Trial name
- NCT03675126
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Sarcopenia in Elderly, Malnourished
The Effects of MediDrink Platinum+ and Fava Bean Protein Hydrolysate on Fitness in an Aged Population
Examining the effects of MediDrink Platinum+ containing novel protein blend SRP-01 alone or when combined with PeptiStrong on clinical responses in elderly adults with sarcopenia
- Conditions
- Sarcopenia in Elderly, Malnourished, Protein Supplementation, Muscle Mass
- Trial name
- NCT07322653
- Testing
- MediDrink Platinum+, MediDrink Platinum+ and PeptiStrong
- Where
- 2 sites in Hungary
- Volunteers
- 75 people
- Sponsor
- Nuritas Ltd
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
DMD/BMD is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00592553
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 174 people
- Sponsor
- PTC Therapeutics
Spinal Muscular Atrophy
A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy with EXG001-307 in SMA1 patients ,who joined the parent study (EXG001-307-102)
- Trial name
- NCT06588803
- Testing
- No intervention, only for observational studies
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Muscular Atrophy
A Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of ACE-031 in Healthy Postmenopausal Women
The purpose of this study is to establish safe dose levels of ACE-031 in healthy postmenopausal women following multiple dose administration. This study will also evaluate if ACE-031 has an effect on muscle.
- Trial name
- NCT00952887
- Testing
- ACE-031
- Where
- 2 sites in Canada
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
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
Muscular Atrophy, Spinal
Pediatric Spinal Muscular Atrophy (SMA) China Registry
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q…
- Trial name
- NCT05042921
- Where
- 25 sites in China
- Volunteers
- 600 people
- Sponsor
- Biogen
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
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with…
- Trial name
- NCT03508947
- Testing
- WVE-210201
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Healthy Volunteer, Atrophy, Muscular
A Clinical Study to Assess the Effects of SRT2104 Upon Immobilization-Induced Skeletal Muscle Atrophy in Healthy Human Volunteers
The main purpose of this study is to investigate the effect of SRT2104 upon energy production in muscle (specifically the maximum amount of energy produced with muscle contraction), how much sugar and fat are stored in the…
- Trial name
- NCT01039909
- Testing
- SRT2104
- Volunteers
- healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Muscular Dystrophy, Duchenne
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 (vesleteplirsen) administered as a single dose to patients with DMD amenable to exon 51 skipping…
- Trial name
- NCT03375255
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.