Clinical trials: Myopathies
Primary Mitochondrial Disease
An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
This is an observational study of patients with Primary Mitochondrial Disease with either signs or symptoms suggestive of myopathy.
- Trial name
- RePOWER (NCT03048617)
- Where
- 33 sites in United States, Australia and 7 more
- Volunteers
- 215 people
- Sponsor
- Stealth BioTherapeutics Inc.
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
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.
Limb-Girdle Muscular Dystrophy, Type 2E
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
- Trial name
- NCT03652259
- Testing
- SRP-9003
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Peripheral Neuropathy, Muscle Weakness
Biomarker for Metachromatic Leukodystrophy (BioMeta) Disease
Development of a new MS-based biomarker for the early and sensitive diagnosis of Metachromatic Leu-kodystrophy disease from blood (plasma)
- Trial name
- BioMeta (NCT01536327)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Systemic Lupus Erythematosus, Lupus Nephritis (LN)
An Exploratory Clinical Study of YTS109 Cell for R/R Autoimmune Diseases
This study evaluates the safety and efficacy of YTS109 cells in adults with relapsed/refractory autoimmune diseases, such as Systemic Lupus Erythematosus (SLE), including LN and SLE-ITP, Sjogren's Syndrome, etc.
- Conditions
- Systemic Lupus Erythematosus, Lupus Nephritis (LN), Sjogren's Syndrome, Inflammatory Myopathy
- Trial name
- NCT07236762
- Testing
- YTS109 cell
- Where
- China
- Volunteers
- 18 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP)
Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)
This is a long-term extension trial of RAY121 in patients with immunological diseases such as antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet's Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing…
- Conditions
- Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM)
- Trial name
- NCT06723106
- Testing
- RAY121
- Where
- 62 sites in Australia, Austria and 17 more
- Volunteers
- 144 people
- Sponsor
- Chugai Pharmaceutical
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.
Weakness, Muscle
Effects of INDUS810 on Body Composition, Muscular Performance, and Training Adaptations
The purpose of this 12-week, interventional study is to determine the effects of oral supplementation with Indus810 (dietary supplement with active ingredient Fenugreek) on body composition, muscular performance, and training…
- Trial name
- NCT03850106
- Testing
- Indus810
- Where
- United States
- Volunteers
- 67 people, healthy volunteers welcome
- Sponsor
- The Center for Applied Health Sciences, LLC
Cerebral Palsy, Muscle Spasticity
Dysport® Pediatric Lower Limb Spasticity Study
The purpose of this research study is to determine whether Dysport® is effective in the treatment of increased stiffness of the calf muscles and to evaluate the safety of this treatment in children with Cerebral Palsy.
- Conditions
- Cerebral Palsy, Muscle Spasticity, Children
- Trial name
- NCT01249417
- Testing
- Botulinum toxin type A
- Where
- 29 sites in United States, Chile and 4 more
- Volunteers
- 241 people
- Sponsor
- Ipsen
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
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD).
- Trial name
- NCT05154851
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Dupuytren's Contracture
A Pharmacokinetic Study of AA4500 (XIAFLEX™, Proposed Name) in Subjects With Dupuytren's Contracture
A Phase 1, open-label, single-dose pharmacokinetic study in subjects with Dupuytren's contracture conducted at one site in the United States.
- Trial name
- NCT00528931
- Testing
- collagenase clostridium histolyticum
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
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.
Sjogrens Disease, Idiopathic Inflammatory Myopathy (IIM)
OM336 in Seropositive Autoimmune Diseases
An early-phase clinical trial evaluating the safety, tolerability, and pharmacokinetics of OM336 in adult participants with seropositive autoimmune diseases. OM336 is administered subcutaneously in ascending dose cohorts.
- Trial name
- NCT07229144
- Testing
- OM336
- Where
- New Zealand
- Volunteers
- 39 people
- Sponsor
- Lakefront Biotherapeutics West LLC
Muscular Dystrophies
Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.
- Trial name
- NCT01153932
- Testing
- GSK2402968
- Where
- 13 sites in Australia, Belgium and 7 more
- Volunteers
- 53 people
- Sponsor
- GlaxoSmithKline
Becker Muscular Dystrophy
Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy
This is a Phase 1, open-label, dose escalation study aimed at evaluating the safety, early efficacy and potential biomarkers of (+)-epicatechin in patients with Becker or Becker-like Muscular Dystrophy (BMD).
- Trial name
- NCT04386304
- Testing
- (+)-Epicatechin
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Epirium Bio Inc.
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.
Fibromyalgia
Prospective Evaluation of RINCE to Reduce Fibromyalgia Effects - 202
The purpose of this study is to test the safety and efficacy of a noninvasive cortical electrostimulation therapy known as "Reduced Impedance Noninvasive Cortical Electrostimulation" (RINCE) in the treatment of pain associated…
- Trial name
- PERRFECT-202 (NCT02613949)
- Testing
- RINCE
- Where
- 4 sites in United States
- Volunteers
- 45 people
- Sponsor
- Cerephex Corporation