Clinical trials: Myopathy
Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Fibrodysplasia Ossificans Progressiva
In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation (heterotopic ossification…
- Trial name
- NCT02521792
- Testing
- Palovarotene
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Post-Stroke Spasticity, Cerebrovascular Accident
Study Of GSK1358820 In Patients With Post-Stroke Upper Limb Spasticity
This is a study to confirm the superior efficacy of a single treatment of GSK1358820 over placebo in patients with post-stroke upper limb spasticity of both the wrist and finger flexors using the Modified Ashworth Scale (MAS)…
- Trial name
- NCT00460564
- Testing
- GSK1358820
- Where
- 18 sites in Japan
- Volunteers
- 109 people
- Sponsor
- GlaxoSmithKline
FSHD - Facioscapulohumeral Muscular Dystrophy
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Muscular Dystrophy (FSHD) 1 or 2.
- Trial name
- NCT07086521
- Testing
- ULSC
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Restem, LLC.
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
- Trial name
- ENHANCE (NCT07542314)
- Testing
- ELEVIDYS, Sirolimus, Glucocorticoids
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sarepta Therapeutics, Inc.
Fibromyalgia
Treatment of Fibromyalgia Using Deep Shaped-Field Transcranial Magnetic Stimulation a Clinical Feasibility Study
Deep shaped-field transcranial magnetic stimulation is a new type of transcranial magnetic stimulation technology which may provide a drug-free method for treating fibromyalgia.
- Trial name
- NCT01229852
- Testing
- DSF-rTMS
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Cervel Neurotech, Inc.
Tennis Elbow
ALGRX 4975 in the Treatment of Tennis Elbow
Tennis elbow is a painful condition that results from repetitive arm movement with overuse of muscles, e.g. when playing tennis. A single injection of ALGRX 4975 has the potential to reduce pain for a period of weeks to months.
- Trial name
- NCT00130949
- Testing
- ALGRX 4975
- Where
- Slovakia
- Volunteers
- 45 people
- Sponsor
- AlgoRx Pharmaceuticals
FSHD, FSHD1
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with…
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE (NCT05747924)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 90 people
- Sponsor
- Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy
Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02858362
- Testing
- Ezutromid
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 43 people
- Sponsor
- Summit Therapeutics
Fibromyalgia
Tool for Fibromyalgia Diagnosis and Effect of Extra Virgin Olive Oil
PRONACERA THERAPEUTICS S.L. is a young biotechnological company focused on the development of genetic diagnostic tools and treatments for pathologies in different medical areas such as reproduction, fibromyalgia and rare diseases…
- Trial name
- FIBROKIT (NCT05921409)
- Testing
- Extra Virgin Olive oil-supplemented Mediterranean Diet Intervention, Olive oil-supplemented Mediterranean Diet Intervention
- Where
- Spain
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Pronacera Therapeutics SL
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
The objectives of this study are to characterize the disease impact, the disease management and resource utilization, and provide benefits to the LC-FAOD community, by use of a convenient online platform for participants (or…
- Trial name
- NCT04812106
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Dupuytren's Disease
Retreatment of Recurrent Dupuytren's Contractures
The objectives of this study are to assess the safety and efficacy of AA4500 in the retreatment of recurrent contractures in joints that were effectively treated with AA4500 in a previous Auxilium-sponsored Phase 3 study.
- Trial name
- NCT01498640
- Testing
- Collagenase clostridium histolyticum
- Where
- 13 sites in United States, Australia and 2 more
- Volunteers
- 52 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Spasticity, Post-Stroke
JCP Study of Investigation of Patient Background Characteristics
To collect the background characteristics of juvenile cerebral palsy patients aged 2-year or older prescribed BOTOX for the treatment of equines foot due to lower limb spasticity, compare them with those of patients whose deaths…
- Trial name
- NCT01332474
- Testing
- BOTOX
- Volunteers
- 326 people
- Sponsor
- GlaxoSmithKline
Fibromyalgia
A Study to Assess the Analgesic Efficacy and Safety of ASP0819 in Patients With Fibromyalgia
This study assessed analgesic efficacy of ASP0819 relative to placebo as well as the safety and tolerability. This study assessed treatment differences in physical function as well as the improvements in overall subject status…
- Trial name
- NCT03056690
- Testing
- ASP0819
- Where
- 24 sites in United States
- Volunteers
- 186 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis
Study of Vimseltinib for Tenosynovial Giant Cell Tumor
This is a multicenter Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called vimseltinib for the treatment of tenosynovial giant cell tumor (TGCT) in cases where surgical removal of the…
- Conditions
- Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath, Tenosynovial Giant Cell Tumor, Diffuse
- Trial name
- MOTION (NCT05059262)
- Testing
- Vimseltinib
- Where
- 35 sites in United States, Australia and 11 more
- Volunteers
- 123 people
- Sponsor
- Deciphera Pharmaceuticals, LLC
Arthritis, Joint Pain
A Controlled Study to Evaluate the Effects of a Topical Cream on Symptoms of Arthritis, Joint, and Muscle Pain
This is a randomized, triple-blind, controlled clinical trial evaluating the effects of Saje Pain Release Joint \& Arthritis Cream on symptoms of arthritis, joint pain, and muscle pain in adults aged 20-65.
- Conditions
- Arthritis, Joint Pain, Muscle Pain, Musculoskeletal Pain
- Trial name
- NCT07767669
- Testing
- Intervention (Saje Pain Release Joint & Arthritis Cream)
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Saie Beauty
Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with…
- Trial name
- NCT03362502
- Testing
- PF-06939926
- Where
- 22 sites in United States
- Volunteers
- 23 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
Phase I/II Study of SRP-4053 in DMD Patients
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.
- Trial name
- NCT02310906
- Testing
- SRP-4053
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Sarepta Therapeutics, Inc.
Dermatomyositis, Myositis
A Study to Evaluate Safety and Efficacy of Empasiprubart in Adults With Dermatomyositis
This study will evaluate the safety and efficacy of empasiprubart compared with placebo in adult participants with dermatomyositis (DM). The study duration will be approximately 92 weeks for all participants.
- Trial name
- empacific (NCT06284954)
- Testing
- Empasiprubart IV
- Where
- 28 sites in United States, Georgia and 5 more
- Volunteers
- 3 people
- Sponsor
- argenx
Diabetic Peripheral Neuropathy, Fibromyalgia
Pharmacokinetics of Two Formulation of Pregabalin
A Randomized, Single-Dose, Parallel-Group Study to Evaluate the Pharmacokinetic Profiles of Two Formulations of Pregabalin after Oral Administration in Healthy Volunteers under Fasting Conditions
- Conditions
- Diabetic Peripheral Neuropathy, Fibromyalgia, Partial Seizure Disorder
- Trial name
- NCT03712475
- Testing
- Pregabalin
- Where
- Taiwan
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Yung Shin Pharm. Ind. Co., Ltd.