Clinical trials: Myopathies
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.
Fibromyalgia
Cefaly® Device in the Treatment of Patients With Fibromyalgia.
The objective of this pilot open trial was to assess the efficacy and safety of external trigeminal nerve stimulation (e-TNS) with the Cefaly® device in fibromyalgia.
- Trial name
- NCT02546362
- Testing
- Cefaly
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Cefaly Technology
Tetraplegia, Spinal Cord Injury
ECoG Direct Brain Interface for Individuals With Upper Limb Paralysis
The purpose of this research study is to demonstrate that individuals with upper limb paralysis due to spinal cord injury, brachial plexus injury, amyotrophic lateral sclerosis and brain stem stroke can successfully achieve…
- Conditions
- Tetraplegia, Spinal Cord Injury, Brachial Plexus Injury, Muscular Dystrophy
- Trial name
- NCT01393444
- Testing
- Implantation of ECoG sensors on the brain surface
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Pittsburgh
Fibromyalgia
Combined Behavioral and Analgesic Trial for Fibromyalgia
This is a randomized control study that involves a combination of a drug treatment, behavioral health, and placebo controls. Some participants will receive a medication approved by the Food and Drug Administration for treatment…
- Trial name
- COMBAT-FM (NCT01598753)
- Testing
- Tramadol, Cognitive Behavior Therapy for FM, Health Education
- Where
- 2 sites in United States
- Volunteers
- 134 people
- Sponsor
- University of Washington
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
Fibromyalgia
"SMART-FM": Smart-Phone Based Digital Therapeutic for Management of Fibromyalgia
This is a non-significant risk, multi-center, randomized, active-controlled study to compare the effectiveness of two digital therapies in the management of fibromyalgia over 12 weeks.
- Trial name
- SMART-FM (NCT05005351)
- Testing
- Digital ACT, Digital Symptom Tracker
- Where
- 7 sites in United States
- Volunteers
- 67 people
- Sponsor
- Swing Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02420379
- Testing
- eteplirsen
- Where
- 13 sites in United States
- Volunteers
- 33 people
- Sponsor
- Sarepta Therapeutics, Inc.
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.
Weakness, Muscle, Delirium in Old Age
YOOMI: Effect of Gamified Physical Therapy Exercise Software on Inpatient Mobility
Patients admitted to the hospital often develop functional impairments due to being in bed most of the day. Each day of bedrest leads to significant muscle loss.
- Conditions
- Weakness, Muscle, Delirium in Old Age, Muscle Atrophy or Weakness, Muscle Loss
- Trial name
- YOOMI (NCT05815017)
- Testing
- Yoomi Physical Therapy Software
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Rutgers, The State University of New Jersey
Polymyositis, Dermatomyositis
Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
This study determined the efficacy, safety, tolerability and the PK profile of BAF312, a novel immunomodulator, in polymyositis and dermatomyositis patients who were not responsive to traditional immunosuppressive and/or…
- Trial name
- NCT01148810
- Testing
- BAF312
- Where
- 6 sites in United States, Czechia and 3 more
- Volunteers
- 18 people
- Sponsor
- Novartis Pharmaceuticals
Becker Muscular Dystrophy
A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
The ARCH study was an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).
- Trial name
- ARCH (NCT05160415)
- Testing
- Sevasemten
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Edgewise Therapeutics, Inc.
Chronic Fatigue Syndrome
Use of Xyrem to Improve Sleep in Chronic Fatigue Syndrome
Chronic fatigue syndrome is a disabling illness for which there is no specific treatment. As a group, CFS patients have disturbed sleep with frequent arousals and the sense of not having slept upon awakening.
- Trial name
- NCT00498485
- Testing
- Sodium Oxybate
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- University of Medicine and Dentistry of New Jersey
Low Back Pain, Pain
Evaluation of Stepped Care for Chronic Pain in Iraq/Afghanistan Veterans (ESCAPE)
The purpose of this study is to determine if a stepped-care intervention makes pain symptoms better and reduces activity limitations because of pain. Our two primary hypotheses are that in OIF/OEF veterans with chronic pain: 1.
- Conditions
- Low Back Pain, Pain, Pain, Intractable
- Trial name
- ESCAPE (NCT00386243)
- Testing
- Cognitive behavioral therapy, Pain self-management program, Co-Analgesic Therapy
- Where
- United States
- Volunteers
- 242 people
- Sponsor
- VA Office of Research and Development
Spinal Cord Injury, Muscle Spasticity
Safety and Efficacy of Oral Fampridine-SR for the Treatment of Spasticity Resulting From Spinal Cord Injury
Normally, nerve fibers carry electrical impulses through the spinal cord, providing communication between the brain and the arms and legs.
- Trial name
- NCT00041717
- Testing
- Fampridine-SR
- Where
- 45 sites in United States, Canada
- Volunteers
- 213 people
- Sponsor
- Acorda Therapeutics
Statin-related Muscle Pain, Muscle Aches
7 Tesla MRI Study in Patients With Statin Related Muscle Complaints
Roughly 5-10% of statin-treated patients report muscle pain, aches, weakness, cramps, stiffness, or "heaviness" - typically occurring symmetrically in the legs.
- Conditions
- Statin-related Muscle Pain, Muscle Aches, Muscle Weakness, Muscle Cramp
- Trial name
- NCT04575090
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Myotonic Dystrophy
Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
The purpose of this study is to investigate the effects of mexiletine treatment for 6 months on ambulation, myotonia, muscle function and strength, pain, gastrointestinal functioning, cardiac conduction, and quality of life in…
- Trial name
- NCT01406873
- Testing
- Mexiletine
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- University of Rochester
Dermatomyositis
Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis (Idiopathic Inflammatory Myopathy)
Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study")
- Trial name
- IIM (NCT02728752)
- Testing
- Octagam 10%
- Where
- 37 sites in United States, Canada and 8 more
- Volunteers
- 95 people
- Sponsor
- Octapharma
Covid19, Muscle Atrophy
Electrical Stimulation for Critically Ill Covid-19 Patients
Unfortunately, hospital-acquired weakness is highly prevalent among COVID-19 hospitalized patients, who often require prolonged bed-rest or paralytics for an extended period of time in order to maintain oxygenation.
- Conditions
- Covid19, Muscle Atrophy, Muscle Weakness
- Trial name
- Phase I (NCT04685213)
- Testing
- Electrical Stimulation
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Baylor College of Medicine
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <12 years).
- Trial name
- LELANTOS-2 (NCT04632940)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 52 sites in United States, Australia and 9 more
- Volunteers
- 73 people
- Sponsor
- Kyntra Bio
Temporomandibular Joint Dysfunction Syndrome
Tailored Self-Management of Temporomandibular Disorders (TMD) Using Health Information Technology
The purpose of this pilot project is to determine the feasibility and acceptability of proposed methods for a subsequent clinical trial of a tailored self-management program (PACT) designed to decrease pain in participants with…
- Trial name
- TMDPACT (NCT03036215)
- Testing
- PACT Experimental Arm
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- HealthPartners Institute