Clinical trials: Atrophy, Muscle
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Duchenne
Spironolactone Versus Prednisolone in DMD
This is a randomized, open-label, pilot clinical trial of spironolactone suspension versus oral prednisolone for use in Duchenne muscular dystrophy.
- Trial name
- NCT03777319
- Testing
- Spironolactone, Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
- Trial name
- NCT04886518
- Testing
- Pitolisant Oral Tablet
- Where
- 13 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Harmony Biosciences Management, Inc.
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE01 (NCT00439569)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Westat
ICU Acquired Weakness
Evaluation of FDY-5301 in Major Trauma Patients in ICU
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of FDY-5301 compared to placebo in major trauma ICU patients at risk of intensive care unit acquired weakness (ICUAW)
- Trial name
- NCT04430283
- Testing
- FDY-5301
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Faraday Pharmaceuticals, Inc.
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
- Trial name
- LELANTOS-1 (NCT04371666)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 98 people
- Sponsor
- Kyntra Bio
Sarcopenia
Effect of an EAA/Whey Composition on Protein Metabolism
Sarcopenia, a progressive loss of muscle mass, strength and function, is an inevitable natural process of aging. While it may be impossible to completely reverse the progress of sarcopenia, it is well established that intake of…
- Trial name
- NCT03502941
- Testing
- 6.3 g of EAAs mixture and whey protein isolate, 12.6 g of EAAs mixture and whey protein isolate, 12.6 g of whey protein isolate
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Duchenne Muscular Dystrophy
CoQ10 and Prednisone in Non-Ambulatory DMD
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD.
- Trial name
- NCT00308113
- Testing
- Prednisone, Coenzyme Q10
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Duchenne
A Study of Tadalafil for Duchenne Muscular Dystrophy
The main purpose of this study is to determine if tadalafil can slow the decline in walking ability of boys who have Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01865084
- Testing
- Tadalafil
- Where
- 62 sites in United States, Argentina and 14 more
- Volunteers
- 331 people
- Sponsor
- Eli Lilly and Company
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the…
- Trial name
- NCT00568698
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Stanford University
Sarcopenia
VR-based Rehabilitation in the Treatment and Prevention of Sarcopenia of Older Residents
Background: Sarcopenia is the progressive loss of skeletal muscle mass and decline of muscle function associated with aging. The prevalence of sarcopenia among people older than 65 years old in Taiwan is over 20%.
- Trial name
- NCT03809104
- Testing
- Virtual reality-based rehabilitation programs
- Where
- Taiwan
- Volunteers
- 43 people
- Sponsor
- Cishan Hospital, Ministry of Health and Welfare
Traumatic Injury, Muscle Injury
Musculotendinous Tissue Repair Unit and Reinforcement (MTURR)
The objective of the study is to assess mechanical strength and function in subjects undergoing Musculotendinous Tissue Unit Repair and Reinforcement (MTURR) with the use of biologic scaffolds for the restoration of both…
- Conditions
- Traumatic Injury, Muscle Injury, Tendon Injury, Soft Tissue Injury
- Trial name
- MTURR (NCT01292876)
- Testing
- Extracellular Matrix
- Where
- 2 sites in United States
- Volunteers
- 17 people
- Sponsor
- University of Pittsburgh
Spinal Muscular Atrophy
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in…
- Trial name
- SHINE (NCT02594124)
- Testing
- nusinersen
- Where
- 47 sites in United States, Australia and 12 more
- Volunteers
- 292 people
- Sponsor
- Biogen
Muscular Atrophy
A Study of LY2495655 in Older Participants Undergoing Elective Total Hip Replacement
The primary objective of this study is to test the hypothesis that appendicular lean body mass (aLBM) will increase after 12 weeks of LY2495655 treatment versus placebo in older participants undergoing elective total hip…
- Trial name
- NCT01369511
- Testing
- LY2495655
- Where
- 45 sites in United States, Austria and 9 more
- Volunteers
- 400 people
- Sponsor
- Eli Lilly and Company
Prostate Cancer, Prostatic Neoplasms
Safety and Efficacy Studies of Panobinostat and Bicalutamide in Patients With Recurrent Prostate Cancer After Castration
This trial is designed to investigate the safety, dosing schedule, and efficacy of the combination treatment of Panobinostat (a histone deacetylase inhibitor) and hormone therapy for recurrent prostate cancer.
- Trial name
- NCT00878436
- Testing
- Panobinostat, Bicalutamide
- Where
- 4 sites in United States
- Volunteers
- 52 people
- Sponsor
- NYU Langone Health
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
Male Hypogonadism, Muscle Atrophy
5-Alpha Reductase and Anabolic Effects of Testosterone
The purpose of this study is to determine whether a higher-than-replacement dose of testosterone and finasteride can be combined to safely increase muscle strength in older men who have a low blood concentration of testosterone.
- Conditions
- Male Hypogonadism, Muscle Atrophy, Sarcopenia, Benign Prostate Hypertrophy
- Trial name
- NCT00475501
- Testing
- Testosterone Enanthate, Finasteride
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- VA Office of Research and Development