Clinical trials: Myopathies
Tenosynovial Giant Cell Tumor
A Study of Pexidartinib in Tenosynovial Giant Cell Tumor in Japan
This phase 2, multicenter, two-part, open-label, single-arm study will be conducted in Japan and will evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of pexidartinib in adult participants with symptomatic…
- Trial name
- NCT04703322
- Testing
- Pexidartinib
- Where
- 6 sites in Japan
- Volunteers
- 9 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Inclusion Body Myositis (IBM)
Long-term Extension Study of Ulviprubart (ABC008) in Subjects With Inclusion Body Myositis
ABC008-IBM-202 is an open-label, multicenter study to evaluate the safety and efficacy of long-term administration of ulviprubart (ABC008) in subjects with IBM who have completed either Study ABC008-IBM-101 or Study…
- Trial name
- NCT06450886
- Testing
- Ulviprubart (ABC008)
- Where
- 21 sites in United States, Australia
- Volunteers
- 270 people
- Sponsor
- Abcuro, Inc.
Spinal Muscular Atrophy
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to <18 years after the discontinuation of treatment with nusinersen or…
- Trial name
- STRENGTH (NCT05386680)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein.
- Trial name
- NCT00264888
- Testing
- PTC124
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy (DMD)
DMD Voice: Qualitative Interviews With Patients and Caregivers
The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers.
- Trial name
- NCT06925269
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Red Nucleus Enterprise Solutions, LLC
Pain Associated With Fibromyalgia
Treatment of Pain Associated With Fibromyalgia
The main objective of this trial is to compare change in weekly average daily pain score (ADPS) from baseline to Week 13 in participants receiving either dose of DS-5565 versus placebo.
- Trial name
- NCT02187471
- Testing
- DS-5565, Pregabalin
- Where
- 154 sites in United States, Argentina and 12 more
- Volunteers
- 1,301 people
- Sponsor
- Daiichi Sankyo
Blepharospasm, Hemifacial Spasm
Post Marketing Surveillance Study of Dysport
The purpose of this study is to provide further information regarding the risks and benefits of Dysport in marketed indications.
- Conditions
- Blepharospasm, Hemifacial Spasm, Cervical Dystonia, Spasmodic Torticollis
- Trial name
- NCT00210431
- Where
- 36 sites in France, Germany and 1 more
- Volunteers
- 783 people
- Sponsor
- Ipsen
Dermatomyositis
Study Evaluating Efficacy and Safety of Octanorm in Patients With Dermatomyositis
DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED PHASE III STUDY EVALUATING EFFICACY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH DERMATOMYOSITIS
- Trial name
- NCT03686969
- Testing
- Octanorm
- Where
- Russia
- Volunteers
- 1 people
- Sponsor
- Octapharma
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Myotonic Disorders
Open-label Extension Study in Paediatric Patients Who Have Completed the MEX-NM-301 Study.
Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed the MEX-NM-301 study.
- Trial name
- NCT04622553
- Testing
- Mexiletine
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Lupin Ltd.
Restrictive Burn Scars
Pilot Study of Azficel-T for the Treatment of Restrictive Burn Scars
This Phase II, double-blind, randomized, placebo-controlled pilot study is designed to determine whether injection of autologous fibroblasts can increase the mobility (decrease the restriction) of burn scars.
- Trial name
- NCT01858753
- Testing
- Autologous fibroblasts
- Where
- 9 sites in United States
- Volunteers
- 5 people
- Sponsor
- Castle Creek Biosciences, LLC.
Spasticity
Retrospective Real-Life Study From One Brazilian Reference Center Assessing Long-Term Experience In The Treatment Of Adult Spasticity With AbobotulinumtoxinA
The aim of the study is to describe the long-term use of abobotulinumtoxinA (Dysport®) in adult subjects affected with upper limb spasticity (ULS) +/- lower limb spasticity (LLS) who received treatment with Dysport® for a minimum…
- Trial name
- RETURN (NCT04446702)
- Where
- Brazil
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Ipsen
GNE Myopathy, Distal Myopathy With Rimmed Vacuoles (DMRV)
Efficacy Confirmation Study of NPC-09
GNE myopathy is a distal myopathy that is thought to be caused by a mutation in the GNE gene that encodes an enzyme in the biosynthetic process of aceneuramic acid (typical sialic acid).
- Conditions
- GNE Myopathy, Distal Myopathy With Rimmed Vacuoles (DMRV), Hereditary Inclusion Body Myopathy (hIBM), Nonaka Disease
- Trial name
- NCT04671472
- Testing
- NPC-09
- Where
- 5 sites in Japan
- Volunteers
- 14 people
- Sponsor
- Nobelpharma
Duchenne Muscular Dystrophy
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53…
- Trial name
- NCT02740972
- Testing
- NS-065/NCNP-01
- Where
- 7 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety and tolerability as well as pharmacokinetics (PK), pharmacodynamics (PD) and…
- Trial name
- DyNaMic (NCT04743557)
- Testing
- DYN101
- Where
- 2 sites in France, Germany
- Sponsor
- Dynacure
Spasticity
A Study to Investigate Arbaclofen ER Tablets for the Treatment of Spasticity in Patients With Multiple Sclerosis
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the safety and efficacy of oral arbaclofen ER tablets in MS patients with spasticity.
- Trial name
- NCT05179577
- Testing
- Arbaclofen
- Volunteers
- 442 people
- Sponsor
- RVL Pharmaceuticals, Inc.
Duchenne Muscular Dystrophy (DMD)
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids
- Trial name
- SIDEROS (NCT02814019)
- Testing
- Idebenone 150 mg film-coated tablets
- Where
- 63 sites in United States, Austria and 13 more
- Volunteers
- 255 people
- Sponsor
- Santhera Pharmaceuticals
Inclusion Body Myositis
Study of Arimoclomol in Inclusion Body Myositis (IBM)
Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients.
- Trial name
- NCT02753530
- Testing
- Arimoclomol
- Where
- 12 sites in United States, United Kingdom
- Volunteers
- 152 people
- Sponsor
- ZevraDenmark
Duchenne Muscular Dystrophy (DMD)
Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)
This is a study of investigational medicines ENTR-601-44 and ENTR-601-45 designed to evaluate the long-term safety and tolerability of study drugs in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- ELEVATE-LTE (NCT07682129)
- Testing
- ENTR-601-44, ENTR-601-45
- Where
- 17 sites in Belgium, Italy and 3 more
- Volunteers
- 80 people
- Sponsor
- Entrada Therapeutics, Inc.