Clinical trials: Muscle Disorder
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
Stroke, Brain Injuries
Comparative Efficacy of Three Preparations of Botox-A in Treating Spasticity
The study seeks to compare the effectiveness of three preparations of BOTOX-A® in treating muscle tightness and spasms in the feet and ankles of people with stroke.
- Conditions
- Stroke, Brain Injuries, Spasticity
- Trial name
- NCT00178646
- Testing
- Botox
- Where
- 2 sites in United States
- Volunteers
- 33 people
- Sponsor
- The University of Texas Health Science Center, Houston
Asthma
Airway Smooth Muscle and Asthma Severity
Our hypothesis is that the severity of asthma is determined by the way in which airway smooth muscle cells grow and release inflammatory mediators.
- Trial name
- NCT00779870
- Testing
- bronchoscopy
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Imperial College London
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.
RYR-1 Myopathy
S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
This study proposes to test S 48168 (ARM210) in a Phase 1 trial in RYR1-RM patients, specifically. The objectives of this study are to explore the safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD)/target…
- Trial name
- NCT04141670
- Testing
- S48168
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- RyCarma Therapeutics, Inc.
Dermatomyositis
A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis
This study is designed to evaluate the safety and efficacy of an oral medicine (called apremilast) for treating skin involvement in patients with the disease dermatomyositis.
- Trial name
- NCT01140503
- Testing
- Apremilast
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Stanford University
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
Adult Fibrosarcoma, Adult Leiomyosarcoma
AZD0530 in Treating Patients With Recurrent Locally Advanced or Metastatic Soft Tissue Sarcoma
This phase II trial is studying how well AZD0530 works in treating patients with recurrent locally advanced, or metastatic soft tissue sarcoma.
- Conditions
- Adult Fibrosarcoma, Adult Leiomyosarcoma, Adult Liposarcoma, Adult Malignant Fibrous Histiocytoma
- Trial name
- NCT00659360
- Testing
- saracatinib
- Where
- 4 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- National Cancer Institute (NCI)
Uterine Fibroids, Heavy Menstrual Bleeding
An Extension Study to Evaluate the Efficacy and Safety of Elagolix in Premenopausal Women With Heavy Menstrual Bleeding Associated With Uterine Fibroids
This is an extension study for women who have already received six months of treatment in the phase III clinical trial M12-815 (NCT02654054) or M12-817 (NCT02691494), and will evaluate the long-term efficacy and safety of…
- Trial name
- NCT02925494
- Testing
- Estradiol/Norethindrone Acetate, Elagolix
- Where
- 124 sites in United States, Canada and 1 more
- Volunteers
- 433 people
- Sponsor
- AbbVie
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
Giant Cell Tumors
Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project
TGCT is a rare disease that is difficult to manage, surgical resection is the primary treatment currently available. To date no disease registry exists and there is little data available detailing the management of patients with…
- Trial name
- TOP (NCT02948088)
- Where
- 15 sites in United States, Austria and 6 more
- Volunteers
- 183 people
- Sponsor
- Daiichi Sankyo
Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis
Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)
The goal of this clinical trial is to assess the efficacy and safety of Pimicotinib (ABSK021) in patients with Tenosynovial Giant Cell Tumor (TGCT).
- Conditions
- Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath
- Trial name
- NCT05804045
- Testing
- Pimicotinib(ABSK021)
- Where
- 40 sites in United States, Canada and 5 more
- Volunteers
- 94 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Myasthenia Gravis, MuSK, AChR Myasthenia Gravis
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.
- Trial name
- NCT03579966
- Testing
- Amifampridine Phosphate
- Where
- 3 sites in United States, Italy
- Volunteers
- 63 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Fibromyalgia
Fibromyalgia Activity Study With Transcutaneous Electrical Nerve Stimulation (FAST)
Pain associated with fibromyalgia interferes with daily function, work, and social activities resulting in a decreased quality of life. People with fibromyalgia also have a significant amount of fatigue and a fear of movement.
- Trial name
- FAST (NCT01888640)
- Testing
- TENS
- Where
- 2 sites in United States
- Volunteers
- 301 people
- Sponsor
- Kathleen Sluka
Musculoskeletal Pain, Chronic Pain
Living in Full Even With Pain Study
This project will increase our understanding of the mechanisms underlying changes in opioid medication use associated with three psychosocial chronic pain (CP) treatments.
- Trial name
- LIFE (NCT03916276)
- Testing
- Cognitive Therapy (CT), Mindfulness Meditation (MM), Behavioral Activation (BA)
- Where
- United States
- Volunteers
- 91 people
- Sponsor
- University of Washington
Bladder Cancer
Avelumab Plus Bacille Calmette-Guerin (BCG) in Patients With Non-muscle Invasive Bladder Cancer
The purpose of this study is to test the safety of avelumab and Bacille Calmette-Guerin (BCG) and see what effects (good and bad) that this combination treatment has on subjects with recurrent bladder cancer.
- Trial name
- OU-SCC-ABC (NCT03892642)
- Testing
- Avelumab, BCG
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- University of Oklahoma
Intensive Care Unit Syndrome, Intensive Care Neurological Disorder
Vanderbilt ICU Recovery Program Pilot Trial
Every year, millions of Americans are admitted to the intensive care unit. Due to advances in critical care, mortality rates are decreasing, increasing the number of ICU survivors.
- Conditions
- Intensive Care Unit Syndrome, Intensive Care Neurological Disorder, Intensive Care (ICU) Myopathy, Intensive Care Psychiatric Disorder (Diagnosis)
- Trial name
- VIP (NCT03124342)
- Testing
- VANDERBILT ICU RECOVERY PROGRAM
- Where
- United States
- Volunteers
- 232 people
- Sponsor
- Vanderbilt University Medical Center
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