Clinical trials: Muscle Disorders
Pompe Disease
A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)
The primary purpose of this study is to evaluate the safety and tolerability profile of S-606001 in participants with LOPD.
- Trial name
- NCT07787650
- Testing
- S-606001
- Volunteers
- 15 people
- Sponsor
- Shionogi
Obesity & Overweight
Effect of a Food for Special Medical Purposes on Muscle Mass Preservation During GLP-1 RAs Weight Loss Treatment
This study evaluates whether a Food for Special Medical Purposes (FSMP) can help to preserve appendicular skeletal muscle mass (ASMM) in adults undergoing weight loss treatment with GLP-1 receptor agonists or dual GIP/GLP-1…
- Trial name
- MYO-PRESERVE (NCT07393360)
- Testing
- Myosave®
- Where
- 3 sites in Italy
- Volunteers
- 144 people
- Sponsor
- Pharmanutra S.p.a.
Rhabdomyolysis, Renal Failure
Myoglobin Removal by High Cut-off CVVHD
A high cut-off dialyzer (septeX) is tested for the removal of myoglobin which is not efficiently removed by standard high flux dialysis membranes.
- Trial name
- HicoRhabdo (NCT01467180)
- Testing
- septeX, HF CVVH
- Where
- Germany
- Volunteers
- 16 people
- Sponsor
- Vantive Health LLC
Tendinopathy
A Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Doses of SM04755 Following Topical Administration to Healthy Subjects
This study is a single-center, randomized, single-blind, placebo-controlled, multiple ascending dose study of SM04755 solution applied topically once daily for 14 days to intact skin overlying the inner thigh of healthy subjects…
- Trial name
- NCT03229291
- Testing
- SM04755, Vehicle
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Biosplice Therapeutics, Inc.
Bladder Cancer, Urothelial Carcinoma
A Phase 3 Study of UGN-102 for Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer
This global, randomized, controlled, open-label Phase 3 study was designed to assess the long-term efficacy and safety of UGN-102 (mitomycin) for intravesical solution with or without (±) transurethral resection of bladder tumors…
- Conditions
- Bladder Cancer, Urothelial Carcinoma, Urothelial Carcinoma Bladder
- Trial name
- ATLAS (NCT04688931)
- Testing
- UGN-102, TURBT
- Where
- 139 sites in United States, Bulgaria and 8 more
- Volunteers
- 282 people
- Sponsor
- UroGen Pharma Ltd.
Obstructive Sleep Apnea Syndrome
Respiratory Muscle Training in Obstructive Sleep Apnea Syndrome (OSAS) Patients
The purpose of this study is to determine whether respiratory muscle training by means of normocapnic hyperpnea leads to clinical and polysomnographical improvements in patients with mild to intermediate sleep apnea syndrome.
- Trial name
- NCT00936286
- Testing
- SpiroTiger
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- Idiag AG
Bladder (Urothelial, Transitional Cell) Cancer, Non-Muscle Invasive Bladder Carcinoma
Efficacy and Safety of Intravesical TARA-002 Compared With Investigator's Choice of Intravesical Chemotherapy in Participants With BCG-naïve High-grade Non-muscle Invasive Bladder Cancer
The goal of this clinical trial is to learn if TARA-002 can treat high-grade non-muscle invasive bladder cancer (NMIBC) in BCG-naïve adults 18 years of age or older.
- Conditions
- Bladder (Urothelial, Transitional Cell) Cancer, Non-Muscle Invasive Bladder Carcinoma, Non-muscle Invasive Bladder Cancer With Carcinoma in Situ
- Trial name
- ADVANCED-3 (NCT07480356)
- Testing
- TARA-002, Investigator's Choice of Intravesical Chemotherapy
- Where
- 11 sites in United States, Ukraine
- Volunteers
- 284 people
- Sponsor
- Protara Therapeutics
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Multiple Sclerosis (MS)
Safety and Effectiveness of Nabiximols Oromucosal Spray as Add-on Therapy in Participants With Spasticity Due to Multiple Sclerosis
This trial is being conducted to demonstrate the efficacy of nabiximols, compared with placebo, when added to standard of care, in the treatment of muscle spasms associated with multiple sclerosis (MS).
- Trial name
- RELEASE MSS3 (NCT04203498)
- Testing
- Nabiximols
- Where
- 37 sites in United States, Czechia and 3 more
- Volunteers
- 139 people
- Sponsor
- Jazz Pharmaceuticals
Advanced or Metastatic Solid Tumors, Non-Small Cell Lung Cancer
A Study in Advanced/Metastatic Solid Tumors With the Study Medicine (PF-07329640) When Given Alone or In Combination
The purpose of this study is to learn about the safety (the impact of the study drug on the participant's body), effects of the study drug alone or in combination with bevacizumab or sasanlimab, and to find the best dose.
- Conditions
- Advanced or Metastatic Solid Tumors, Non-Small Cell Lung Cancer, Colorectal Cancer, Urothelial Cancer
- Trial name
- LTbR (NCT06448364)
- Testing
- PF-07329640, sasanlimab, bevacizumab
- Where
- 7 sites in United States, Puerto Rico
- Volunteers
- 4 people
- Sponsor
- Pfizer
Myositis
A Study of Nipocalimab in Participants With Active Idiopathic Inflammatory Myopathies
The purpose of this study is to evaluate the efficacy and safety of Nipocalimab versus placebo in participants with active idiopathic inflammatory myopathies (IIM).
- Trial name
- SPIREA (NCT05379634)
- Testing
- Nipocalimab, Glucocorticoids
- Where
- 57 sites in United States, Canada and 11 more
- Volunteers
- 36 people
- Sponsor
- Janssen Research & Development, LLC
Duchenne Muscular Dystrophy
A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular…
- Trial name
- NCT02310763
- Testing
- PF-06252616
- Where
- 66 sites in United States, Australia and 6 more
- Volunteers
- 121 people
- Sponsor
- Pfizer
NMIBC
A Study of Intravesical SHR-1501 Combination With BCG Versus Investigator-selected Chemotherapy in Subjects With BCG-unresponsive NMIBC
This is a multicenter, randomized, open-label phase III study to determine the efficacy and safety of intravesical SHR-1501 combined with Bacillus Calmette Guerin (BCG) versus investigator-selected chemotherapy in patients with…
- Trial name
- NCT07424287
- Testing
- SHR-1501 for Injection, BCG for Injection, Epirubicin Hydrochloride for Injection
- Where
- 2 sites in China
- Volunteers
- 236 people
- Sponsor
- Suzhou Suncadia Biopharmaceuticals Co., Ltd.
Immune Mediated Necrotizing Myopathy
Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
The purpose of the study is to evaluate the safety and efficacy of zilucoplan in patients with Immune-Mediated Necrotizing Myopathy (IMNM).
- Trial name
- NCT04025632
- Testing
- zilucoplan
- Where
- 18 sites in United States, France and 2 more
- Volunteers
- 27 people
- Sponsor
- Ra Pharmaceuticals
Fibrodysplasia Ossificans Progressiva
A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva
This is a three period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2) and a follow-up treatment period (period 3).
- Trial name
- LUMINA-1 (NCT03188666)
- Testing
- REGN2477
- Where
- 11 sites in United States, Canada and 6 more
- Volunteers
- 44 people
- Sponsor
- Regeneron Pharmaceuticals
Cocaine Addiction
Efficacy and Safety of TV-1380 as Treatment for Facilitation of Abstinence in Cocaine-Dependent Subjects
The primary objective of this study is to assess the efficacy and safety of TV-1380 [Recombinant human serum albumin (HSA) mutated butyrylcholinesterase (AlbuBChE)] in facilitating abstinence in cocaine-dependent subjects.
- Trial name
- NCT01887366
- Testing
- TV-1380 150 mg, TV-1380 300 mg
- Where
- 19 sites in United States, Spain
- Volunteers
- 208 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
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
Myotonic Dystrophy 1
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared…
- Trial name
- FREEDOM2-DM1 (NCT06667453)
- Testing
- PGN-EDODM1
- Where
- 12 sites in Canada, New Zealand and 2 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Fibromyalgia
Study of Duloxetine Versus Placebo in the Treatment of Fibromyalgia Syndrome
To assess the efficacy of Duloxetine compared with placebo in the treatment of pain in patients with Fibromyalgia syndrome, with or without major depressive disorder.
- Trial name
- NCT00190866
- Testing
- Duloxetine
- Where
- 35 sites in United States, Puerto Rico
- Volunteers
- 210 people
- Sponsor
- Eli Lilly and Company
Ischemic Heart Disease
Safety Study of Gene Therapy for Ischemic Heart Disease in Korea
The purpose of this study is to evaluate the safety of VM202 (Engensis) direct injection into the cardiac muscles of the coronary artery territory where complete revascularization could not be done even through Coronary Artery…
- Trial name
- Engensis (NCT01422772)
- Testing
- VM202-0.5 mg, VM202-1.0 mg, VM202-2.0 mg
- Where
- South Korea
- Volunteers
- 9 people
- Sponsor
- Helixmith Co., Ltd.