Clinical trials: Myopathies
Duchenne Muscular Dystrophy
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and…
- Trial name
- ESSENCE (NCT02500381)
- Testing
- SRP-4045, SRP-4053
- Where
- 76 sites in United States, Argentina and 22 more
- Volunteers
- 228 people
- Sponsor
- Sarepta Therapeutics, Inc.
Fibrodysplasia Ossificans Progressiva
Study Evaluating the Effect of Food on the Pharmacokinetics of Palovarotene and the Effect of Palovarotene on the Pharmacokinetics of the CYP3A4 Substrate Midazolam in Two Cohorts of Healthy Adult Subjects
Study to evaluate the effect of food and the effect of swallowing capsule whole versus sprinkling on apple sauce on the pharmacokinetics (PK)/bioavailability of palovarotene, and evaluate the effect of palovarotene on the PK of…
- Trial name
- NCT04829773
- Testing
- Palovarotene, midazolam
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Clementia Pharmaceuticals Inc.
Muscle Spasticity
BOTOX® Economic Spasticity Trial (BEST)
This is a study to investigate if patients who have had a stroke and suffer from spasticity might benefit from being given BOTOX® in addition to the normal Standard Care.
- Trial name
- NCT00549783
- Testing
- Botulinum Toxin Type A 900kD
- Where
- 4 sites in Canada, Germany and 2 more
- Volunteers
- 274 people
- Sponsor
- Allergan
Fibromyalgia
Study of Milnacipran for the Treatment of Fibromyalgia
Antidepressants of all varieties represent a common form of therapy for many chronic states including fibromyalgia. The majority of antidepressants increase the levels of serotonin or norepinephrine in the central nervous system.
- Trial name
- NCT00098124
- Testing
- milnacipran hydrochloride
- Where
- 80 sites in United States
- Volunteers
- 1,200 people
- Sponsor
- Forest Laboratories
PreDiabetes, Older Adults
Vivo Prediabetes Study: Online, Live, and Interactive Strength Training for Older Adults With Prediabetes
This research trial studies the effect of an online, live and interactive strength training program on physical function and strength in older adults with prediabetes.
- Conditions
- PreDiabetes, Older Adults, Muscle Loss, Muscle Weakness
- Trial name
- NCT05497960
- Testing
- Vivo workout
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Eric Levian
Fibrodysplasia Ossificans Progressiva
Urine Sample Collection From FOP Patients
The purpose of this study is to collect urine samples from patients with fibrodysplasia ossificans progressiva (FOP) for the assessment of biomarkers related to disease, disease progression and for prediction of flare-ups of the…
- Trial name
- FOP (NCT02066324)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
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
Duchenne Muscular Dystrophy
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory…
- Trial name
- HORIZON (NCT06597656)
- Testing
- delandistrogene moxeparvovec, Plasmapheresis
- Where
- 3 sites in United States
- Volunteers
- 3 people
- Sponsor
- Sarepta Therapeutics, Inc.
Fibromyalgia, Myofascial Pain Syndromes
A Study to Evaluate eFFIcacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With fibRoMyalgia
The use of low-dose CBP dosed nightly at bedtime for FM was supported by the results of Tonix' TNX-CY-F202 Phase 2b study (also referred to as the BESTFIT Study).
- Conditions
- Fibromyalgia, Myofascial Pain Syndromes, Muscular Diseases, Musculoskeletal Diseases
- Trial name
- AFFIRM (NCT02436096)
- Testing
- TNX-102 SL Tablet, 2.8mg
- Where
- 35 sites in United States
- Volunteers
- 519 people
- Sponsor
- Tonix Pharmaceuticals, Inc.
Myotonic Dystrophy Type 1, DM1
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy…
- Conditions
- Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia
- Trial name
- HARBOR-OLE (NCT07008469)
- Testing
- Del-desiran (AOC 1001)
- Where
- 35 sites in United States, Canada and 8 more
- Volunteers
- 199 people
- Sponsor
- Avidity Biosciences, Inc.
Facioscapulohumeral Muscular Dystrophy (FSHD)
A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
The purpose of this study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal antibody that binds to human latent myostatin, in ambulant adult…
- Trial name
- MANOEUVRE (NCT05548556)
- Testing
- RO7204239
- Where
- 9 sites in United States, Denmark and 2 more
- Volunteers
- 51 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03039686
- Testing
- RO7239361
- Where
- 44 sites in United States, Argentina and 11 more
- Volunteers
- 166 people
- Sponsor
- Hoffmann-La Roche
Skin Laxity, Muscle Contracture
Evaluation of the Immediate Effect and Safety of DMA™ Novel Muscle Stimulation Technology
A total of at least 15 and up to 17 healthy subjects at a single site, aged 25-45 years old, who wish to receive a lifting effect and improved muscle definition, will be included in the study.
- Trial name
- NCT04296201
- Testing
- DMA treatment
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Lumenis Be Ltd.
Fibromyalgia
Safety and Efficacy of AGN 203818 for Pain Associated With Fibromyalgia Syndrome
This study will explore the safety and effectiveness of different doses of AGN 203818 in treating the pain associated with fibromyalgia syndrome. The study is being conducted in 2 parts.
- Trial name
- NCT00445705
- Testing
- AGN 203818
- Where
- 3 sites in United States, Switzerland and 1 more
- Volunteers
- 211 people
- Sponsor
- Allergan
Delayed Onset Muscle Soreness, DOMS
COMBOPROFEN for Treatment of Muscular Pain Associated With Delayed Onset Muscle Soreness (DOMS)
The study is designed as a proof of concept, single-center, randomized, double-blind, placebo controlled study to assess the safety and efficacy of Comboprofen (Ibuprofen + Magnesium + Vitamin C) compared to placebo, Ibuprofen…
- Trial name
- NCT03223519
- Testing
- Comboprofen, Ibuprofen, Magnesium
- Where
- Spain
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Spherium Biomed
Primary Mitochondrial Disease
OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)
The goal of this clinical trial is to learn about the treatment effects of the investigational new drug OMT-28 in patients with Primary Mitochondrial Disease.
- Trial name
- PMD-OPTION (NCT05972954)
- Testing
- OMT-28
- Where
- 9 sites in Germany, Italy and 1 more
- Volunteers
- 28 people
- Sponsor
- Omeicos Therapeutics GmbH
Duchenne Muscular Dystrophy
Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne…
- Trial name
- NCT01396239
- Testing
- AVI-4658 (Eteplirsen)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Limb-girdle Muscular Dystrophy
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- EMERGENE (NCT06246513)
- Testing
- SRP-9003, Glucocorticoid
- Where
- 10 sites in United States, Belgium and 4 more
- Volunteers
- 17 people
- Sponsor
- Sarepta Therapeutics, Inc.
Relapsed/Refractory Autoimmune Diseases
A Study of LCAR-AIO in Subjects With Relapsed/Refractory Autoimmune Diseases
This is a prospective, single-arm, open-label, dose-exploration and expansion clinical study of LCAR-AIO in adult subjects with relapsed/refractory autoimmune diseases.
- Trial name
- NCT06866080
- Testing
- LCAR-AIO T cells
- Where
- 4 sites in China
- Sponsor
- Nanjing Legend Biotech Co.
Spasticity
Safety Study of SPARC1104
Open-label, safety study of SPARC1104 in subjects with spasticity due to multiple sclerosis
- Trial name
- NCT01797185
- Testing
- SPARC1104 modified dose regimen I, SPARC1104 modified dose regimen II, SPARC1104 modified dose regimen III
- Where
- 51 sites in United States
- Volunteers
- 375 people
- Sponsor
- Sun Pharma Advanced Research Company Limited