Clinical trials: MYOPATHY
Solid Tumor, Tumors of Any Histology With Activating Trk (NTRK) Point or NTRK Fusion Mutations
Phase 1 Study of PLX7486 as Single Agent in Patients With Advanced Solid Tumors
The objective of this study is to determine the safety, pharmacokinetics, maximum tolerated dose/recommended Phase 2 dose, and efficacy of PLX7486.
- Conditions
- Solid Tumor, Tumors of Any Histology With Activating Trk (NTRK) Point or NTRK Fusion Mutations, Tenosynovial Giant Cell Tumor
- Trial name
- NCT01804530
- Testing
- PLX7486 TsOH
- Where
- 4 sites in United States
- Volunteers
- 59 people
- Sponsor
- Plexxikon
Fibromyalgia
This Study is An Open-Label Trial Of Pregabalin In Patients With Fibromyalgia
The main purpose of this protocol is to evaluate the long-term safety of pregabalin in patients who participated in the previous fibromyalgia Study A0081100 and who wish to receive open-label pregabalin therapy.
- Trial name
- NCT00346034
- Testing
- Pregabalin
- Where
- 51 sites in Canada, Denmark and 10 more
- Volunteers
- 357 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Cardiac Diseases, Muscular Weakness
Biomarker for Pompe Disease (BioPompe)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Pompe disease from blood (plasma)
- Conditions
- Cardiac Diseases, Muscular Weakness, Hepato-splenomegaly, Lung Disease
- Trial name
- BioPompe (NCT01457443)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Polymyalgia Rheumatica
Sarilumab Efficacy and Safety in Adults With Early Polymyalgia Rheumatica
This is a randomized, double-blind, placebo-controlled, parallel-group, Phase 3, 3-arm study to assess whether treatment with sarilumab at either 150 mg q2w (once every two weeks) or at 200 mg q2w, each given with a 52-week…
- Trial name
- ZIRCON (NCT07286214)
- Testing
- Sarilumab
- Where
- 85 sites in United States, Austria and 8 more
- Volunteers
- 300 people
- Sponsor
- Sanofi
Multiple Sclerosis (MS)
Safety and Effectiveness of Nabiximols Oromucosal Spray as Add-on Therapy in Participants With Spasticity Due to Multiple Sclerosis
This trial is being conducted to demonstrate the efficacy of nabiximols, compared with placebo, when added to standard of care, in the treatment of muscle spasms associated with multiple sclerosis (MS).
- Trial name
- RELEASE MSS3 (NCT04203498)
- Testing
- Nabiximols
- Where
- 37 sites in United States, Czechia and 3 more
- Volunteers
- 139 people
- Sponsor
- Jazz Pharmaceuticals
Danon Disease
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2…
- Trial name
- NCT03882437
- Testing
- RP-A501
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Mitochondrial Myopathies, Mitochondrial Pathology
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary…
- Conditions
- Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases
- Trial name
- NuPower (NCT05162768)
- Testing
- Elamipretide
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 102 people
- Sponsor
- Stealth BioTherapeutics Inc.
Becker Muscular Dystrophy
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 175 adult participants in a cohort…
- Trial name
- NCT05291091
- Testing
- Sevasemten 10 mg, Sevasemten 5 mg, Sevasemten 12.5 mg
- Where
- 51 sites in United States, Australia and 10 more
- Volunteers
- 244 people
- Sponsor
- Edgewise Therapeutics, Inc.
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
Spasticity
Study of SPARC1103 in Subjects With Spasticity
Study of SPARC1103 in subjects with spasticity
- Trial name
- NCT02027025
- Testing
- SPARC1103 low dose, SPARC1103 high dose
- Where
- 23 sites in United States, Russia and 1 more
- Volunteers
- 142 people
- Sponsor
- Sun Pharma Advanced Research Company Limited
Muscle Spasticity
Safety and Preliminary Effectiveness of AV650 in Patients With Spasticity Due to Spinal Cord Injury
A drug called AV650 (tolperisone HCl) will be given to patients who have spasticity following a spinal cord injury. This study has three purposes: 1. To determine whether AV650 is safe for patients with spinal cord injury; 2.
- Trial name
- NCT00531466
- Testing
- tolperisone HCl (AV650)
- Where
- 10 sites in United States, Canada
- Volunteers
- 120 people
- Sponsor
- Avigen
Duchenne Muscular Dystrophy
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
- Trial name
- MISSION (NCT02606136)
- Testing
- Pamrevlumab
- Where
- 10 sites in United States
- Volunteers
- 21 people
- Sponsor
- Kyntra Bio
Chronic Fatigue Syndrome, Orthostatic Hypotension
Study To Assess The Clinical Benefit Of Droxidopa In Subjects With Chronic Fatigue Syndrome
A subset of patients suffering from chronic fatigue syndrome exhibit symptoms of neurally mediated hypotension. While the underlying pathophysiology of chronic fatigue syndrome is not precisely understood, a dysfunction of the…
- Conditions
- Chronic Fatigue Syndrome, Orthostatic Hypotension, Neurally Mediated Hypotension, Neurogenic Orthostatic Hypotension
- Trial name
- CFS201 (NCT00977171)
- Testing
- Droxidopa
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Chelsea Therapeutics
Duchenne Muscular Dystrophy
Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this Expanded Access Program is to provide idebenone as a treatment for eligible participants with Duchenne Muscular Dystrophy before it is commercially available in the United States (U.S.) for the…
- Trial name
- NCT03433807
- Testing
- Idebenone
- Where
- 12 sites in United States
- Sponsor
- Santhera Pharmaceuticals
Polymyalgia Rheumatica
Efficacy and Safety of Olokizumab in Patients With Polymyalgia Rheumatica (PROMISE)
The primary objective of the study is to evaluate the efficacy of olokizumab (OKZ) 64 mg administered subcutaneously every 2 weeks compared with placebo in participants with polymyalgia rheumatica (PMR).
- Trial name
- NCT07670546
- Testing
- Olokizumab
- Where
- 20 sites in Russia
- Volunteers
- 125 people
- Sponsor
- R-Pharm International, LLC
Duchenne Muscular Dystrophy
Extension Study of Drisapersen in DMD Subjects
This is a phase IIIb, multi-centre, open-label extension study in male subjects with DMD who previously have been treated with drisapersen, aiming at assessing the safety and efficacy of drisapersen.
- Trial name
- NCT02636686
- Testing
- Drisapersen
- Where
- 39 sites in United States, Argentina and 18 more
- Sponsor
- BioMarin Pharmaceutical
Pediatrics, Muscle Spasticity
BOTOX® Treatment in Pediatric Upper Limb Spasticity
This study will evaluate the safety and efficacy of BOTOX® (botulinum toxin Type A) in pediatric patients with upper limb spasticity.
- Conditions
- Pediatrics, Muscle Spasticity, Cerebral Palsy, Stroke
- Trial name
- NCT01603602
- Testing
- botulinum toxin Type A
- Where
- 45 sites in United States, Canada and 7 more
- Volunteers
- 235 people
- Sponsor
- Allergan
Inclusion Body Myositis
Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of Inclusion Body Myositis (IBM)
- Trial name
- NCT04049097
- Testing
- Arimoclomol
- Where
- 12 sites in United States, United Kingdom
- Volunteers
- 121 people
- Sponsor
- ZevraDenmark
Interstitial Pneumonia Associated With Polymyositis/Dermatomyositis
A Survey for Long-term Use of Prograf Capsules in Patient With Interstitial Pneumonia
This study is to evaluate the safety and the efficacy of Prograf in patients with interstitial pneumonia associated with polymyositis / dermatomyositis in acute clinical setting.
- Trial name
- NCT02159651
- Testing
- tacrolimus
- Where
- 8 sites in Japan
- Volunteers
- 179 people
- Sponsor
- Astellas Pharma Inc