Clinical trials: Myopathies
Duchenne Muscular Dystrophy (DMD)
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
The primary purpose of this study is to evaluate the safety and tolerability of ASP0367. This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.
- Trial name
- NCT04184882
- Testing
- Bocidelpar
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Astellas Pharma 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.
Chronic Widespread Pain, Fibromyalgia
Efficacy and Safety of IGN-ES001 in Chronic Widespread Pain With or Without Fibromyalgia
This is a randomized, double-blind, placebo-controlled exploratory trial to investigate efficacy and safety of food supplement IGN-ES001 in patients with chronic widespread pain (CWP) with or without fibromyalgia (FM).
- Trial name
- NCT03058224
- Testing
- IGN-ES001, Parol 500 mg Tablets (acetaminophen)
- Where
- 23 sites in Turkey (Türkiye)
- Volunteers
- 230 people
- Sponsor
- IgNova GmbH
Duchenne Muscular Dystrophy
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT04906460
- Testing
- WVE-N531
- Where
- 6 sites in United States, Jordan and 1 more
- Volunteers
- 25 people
- Sponsor
- Wave Life Sciences USA, Inc.
Hyperhidrosis, Muscle Spasticity
Safety and Efficacy of BOTOX® (Botulinum Toxin Type A) in Korea
This post marketing surveillance study in Korea will evaluate the safety and efficacy of BOTOX (botulinum toxin Type A) in patients who receive treatment according to standard of care for primary axillary hyperhidrosis, focal…
- Conditions
- Hyperhidrosis, Muscle Spasticity, Glabellar Lines
- Trial name
- NCT02043145
- Testing
- botulinum toxin Type A
- Where
- South Korea
- Volunteers
- 727 people
- Sponsor
- Allergan
Duchenne Muscular Dystrophy (DMD)
Study of Eteplirsen in DMD Patients
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51.
- Trial name
- PROMOVI (NCT02255552)
- Testing
- eteplirsen
- Where
- 37 sites in United States
- Volunteers
- 109 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
Childhood-onset Systemic Lupus Erythematous, ANCA-Associated Vasculitis (AAV)
Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders
Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis
- Conditions
- Childhood-onset Systemic Lupus Erythematous, ANCA-Associated Vasculitis (AAV), Juvenile Myasthenia Gravis, Juvenile Dermatomyositis
- Trial name
- NCT07089121
- Testing
- Descartes-08
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- Cartesian Therapeutics
Joint Pain, Musculoskeletal Pain
IDEA-033 Open Label Study
The main aim of the study is to evaluate safety, efficacy, compliance, and usage of epicutaneously applied IDEA-033 in joint / musculoskeletal pain or soft tissue inflammation.
- Conditions
- Joint Pain, Musculoskeletal Pain, Stiffness, Soft Tissue Inflammation in Designated Target Area(s)
- Trial name
- NCT00372333
- Testing
- IDEA-033
- Where
- Germany
- Volunteers
- 491 people
- Sponsor
- IDEA AG
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Thymidine Kinase 2 Deficiency
An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study…
- Trial name
- Continuation (NCT03845712)
- Testing
- doxecitine and doxribtimine
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 47 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Pain
An Effectiveness and Safety Study Comparing Acetaminophen (3900 mg/Day) to Ibuprofen (1200 mg/Day) in the Treatment of Post-Race Muscle Soreness.
The purpose of this study is to compare the effectiveness and safety of acetaminophen extended release caplets and ibuprofen in relieving the muscle soreness that occurs after a marathon.
- Trial name
- NCT00240838
- Testing
- acetaminophen extended release caplets
- Volunteers
- 483 people, healthy volunteers welcome
- Sponsor
- Johnson & Johnson Consumer and Personal Products Worldwide
Acute Compartment Syndrome
The Use of Near Infrared Spectroscopy in the Diagnosis of Acute Compartment Syndrome
This is a prospective observational study to determine the reliability and accuracy of Near Infrared Spectroscopy (NIRS) to detect oxygen flow in the extremities of injured and non-injured soldiers over time.
- Trial name
- NCT01171638
- Testing
- Near infrared spectroscopy (NIRS)
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- J&M Shuler
Multiple Sclerosis, Spasticity, Muscle
Performance and Long-term Safety of FlowOx2.0™ in Patients With Multiple Sclerosis - Impact on Spasticity and Pain
The study is a 4-week double-blind, randomized, controlled, parallel design investigation to investigate the impact of intermittent negative pressure on spasticity and pain in people with multiple sclerosis (pwMS).
- Conditions
- Multiple Sclerosis, Spasticity, Muscle, Pain, Chronic
- Trial name
- FO-NP002 (NCT05562453)
- Testing
- FlowOx 2.0
- Where
- Sweden
- Volunteers
- 56 people
- Sponsor
- Otivio AS
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Systemic Sclerosis Cutaneous Lupus E Myopathies Systemic Lupus Erythematosus
Rollover Study for Participants Who Have Completed a Previous Clinical Study With Anifrolumab (Saphnelo) and Clinically Benefited From Continued Treatment
The purpose of this study is to provide continued access to anifrolumab to eligible participants who are continuing to derive clinical benefit from treatment at the end of a parent clinical study, as assessed by the Investigator…
- Trial name
- ROSY-S (NCT07691970)
- Testing
- Anifrolumab
- Where
- 153 sites in United States, Argentina and 33 more
- Volunteers
- 1,072 people
- Sponsor
- AstraZeneca
Duchenne Muscular Dystrophy
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study.
- Trial name
- EXPEDITION (NCT05967351)
- Testing
- delandistrogene moxeparvovec
- Where
- 38 sites in United States, Belgium and 7 more
- Volunteers
- 400 people
- Sponsor
- Sarepta Therapeutics, Inc.
Fibromyalgia
Study of Milnacipran in Patients With Inadequate Response to Duloxetine for the Treatment of Fibromyalgia
The objective of this study is to evaluate the safety, tolerability and efficacy of milnacipran in patients with an inadequate response to duloxetine for the treatment of fibromyalgia.
- Trial name
- NCT01077375
- Testing
- Milnacipran
- Where
- 26 sites in United States
- Volunteers
- 107 people
- Sponsor
- Forest Laboratories
Compartment Syndrome
Phase I, Arteriocyte Magellan MAR01 Therapy - Compartment Syndrome and Battlefield Trauma
Compartment syndrome (CS) is a condition resulting from increased pressure within a compartment, which compromises circulation and can lead to critical limb ischemia.
- Trial name
- Magellan MAR01 (NCT01837264)
- Testing
- Magellan®
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Arteriocyte, Inc.
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd