Clinical trials: Neuromuscular Diseases
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne Muscular Dystrophy (DMD).
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-2 (NCT03406780)
- Testing
- CAP-1002
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Capricor Inc.
Myasthenia Gravis, Neuromuscular Manifestations
Expediting Myasthenia Gravis (MG) Diagnostic Evaluation: A Novel, Proof-of-Concept for Undiagnosed, Symptomatic Patients That Uses Social Media Targeting and Self-Assessment
This is a fully remote, site-less, prospective, observational study enrolling adults in the United States (excluding U.S. territories) with undiagnosed neuromuscular symptoms.
- Conditions
- Myasthenia Gravis, Neuromuscular Manifestations, Neuromuscular Diseases
- Trial name
- NCT06381284
- Testing
- Social media recruitment, Self-Assessment, Follow-up
- Where
- United States
- Volunteers
- 215 people, healthy volunteers welcome
- Sponsor
- ZS Associates
Myasthenia Gravis, Neuromuscular Disease
Validation and Scaling of Screening Program for Undiagnosed Myasthenia Gravis-Social Media Campaign Paired With a Self-moderated Assessment
This study expands and validates the pilot study NCT06381284. It is a fully remote, site-less, prospective, observational study enrolling adults in the United States (excluding U.S.
- Conditions
- Myasthenia Gravis, Neuromuscular Disease, Neuromuscular Manifestations
- Trial name
- NCT06866483
- Testing
- Social media recruitment, Self-Assessment, Follow-up
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- ZS Associates
Fibromyalgia, Myofascial Pain Syndromes
Repeat of: A Study to Evaluate Efficacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With Fibromyalgia
The present trial is designed to assess the safety and efficacy of TNX-102 SL 2.8 mg tablets, taken daily at bedtime after 12 weeks of treatment in patients with fibromyalgia.
- Conditions
- Fibromyalgia, Myofascial Pain Syndromes, Muscular Diseases, Nervous System Diseases
- Trial name
- RE-AFFIRM (NCT02829814)
- Testing
- TNX-102 SL Tablet, 2.8 mg
- Where
- 32 sites in United States
- Volunteers
- 51 people
- Sponsor
- Tonix Pharmaceuticals, 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.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <12 years).
- Trial name
- LELANTOS-2 (NCT04632940)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 52 sites in United States, Australia and 9 more
- Volunteers
- 73 people
- Sponsor
- Kyntra Bio
Facioscapulohumeral Muscular Dystrophy (FSHD)
Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
This is a study to evaluate the safety and efficacy of Losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks.
- Trial name
- FSHD (NCT04003974)
- Testing
- Losmapimod oral tablet
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 80 people
- Sponsor
- Fulcrum Therapeutics
Myasthenia Gravis
The Impact of Myasthenia Gravis in the Real World
An international, non-interventional, prospective, community recruited, longitudinal, lifestyle study which aims to collect Real-World Evidence (RWE) from people living with myasthenia gravis (MG) in order to map the disease's…
- Trial name
- NCT04176211
- Where
- United Kingdom
- Volunteers
- 2,847 people
- Sponsor
- Vitaccess Ltd
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)
The CENTAUR trial was a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.
- Conditions
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Diseases, Neurodegenerative Diseases
- Trial name
- CENTAUR (NCT03127514)
- Testing
- AMX0035
- Where
- 25 sites in United States
- Volunteers
- 137 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Spinal Muscular Atrophy
A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
The primary objective of the study is to explore the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
- Trial name
- DigiNOA (NCT05109637)
- Testing
- Konectom NMD Application
- Where
- 5 sites in Germany
- Volunteers
- 93 people
- Sponsor
- Biogen
Cerebral Palsy, Acquired Brain Injury
Proof of Concept of Pediatric and Adolescent EXPLORER V2 Exoskeleton in Children With Neurological and Neuromuscular Disease
The goal of this clinical trial is to analyze the usability and safety of the prototype gait exoskeleton EXPLORE V2 in children with neurological and neuromuscular disease.
- Conditions
- Cerebral Palsy, Acquired Brain Injury, Neuromuscular Diseases in Children
- Trial name
- NCT05914818
- Testing
- EXPLORER V2
- Where
- 5 sites in Spain
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- MarsiBionics
Neuromuscular Diseases, Cerebral Palsy
Rehabilitative Effect of the Use of a Gait Exoskeleton in Patients With Neuromuscular Disease or Cerebral Palsy
The purpose of this multicentric study is to assess the rehabilitative effect derived from the use of the ATLAS exoskeleton in children with neuromuscular diseases or cerebral palsy and with a level of less than 3 or less in the…
- Conditions
- Neuromuscular Diseases, Cerebral Palsy, SMA II
- Trial name
- NCT04813601
- Testing
- ATLAS 2030
- Where
- Spain
- Volunteers
- 10 people
- Sponsor
- MarsiBionics
ALS
Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS
The purpose of this study is to evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS.
- Trial name
- NCT04987671
- Testing
- AMX0035
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Neuromuscular Diseases, Scoliosis
Evaluation of the Feasibility of Remote Monitoring of Mechanical In-exsufflation Devices in Paralytic and Neuromuscular Patients Treated at Home.
The implementation of an mechanical in-exsufflator device (MI-E) requires specific expertise because it is a complex device that requires fine-tuning of the settings according to different clinical situations to optimize its…
- Trial name
- TELE-INEX (NCT05723289)
- Testing
- MI-E Telemonitoring
- Where
- France
- Volunteers
- 10 people
- Sponsor
- KerNel Biomedical
Chronic Inflammatory Demyelinating Polyradiculoneuropathy
A Study of HyQvia and Gammagard Liquid (Kiovig) in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
The aim of this study is to learn more about the following treatment options in adults with CIDP: Subcutaneous self-infusion with HyQvia. Intravenous infusion with Gammagard/Kiovig.
- Trial name
- NCT02549170
- Testing
- HYQVIA, IGIV GAMMAGARD LIQUID/KIOVIG, IGIV GAMUNEX®-C
- Where
- 83 sites in United States, Argentina and 20 more
- Volunteers
- 138 people
- Sponsor
- Baxalta now part of Shire
Healthy Volunteer, Muscular Dystrophies
Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers
This is a Phase 1 2-part, single-center, open-label study in healthy male volunteers. Part A will assess the absorption, metabolism, excretion, and pharmacokinetics of one oral dose of radiolabeled EDG-5506.
- Conditions
- Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT05730842
- Testing
- EDG-5506 Tablet, Radiolabeled EDG-5506 Suspension, Radiolabeled EDG-5506 Intravenous
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Myotonic Dystrophy Type 1
A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1
This study will test the safety, tolerability, and pharmacokinetics of multiple escalating doses of ISIS-DMPKRx administered subcutaneously to adult patients with DM1.
- Trial name
- NCT02312011
- Testing
- IONIS-DMPKRx
- Where
- 8 sites in United States
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Muscular Dystrophy, Duchenne
Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically…
- Trial name
- MoveDMD® (NCT02439216)
- Testing
- Edasalonexent
- Where
- 5 sites in United States
- Volunteers
- 31 people
- Sponsor
- Catabasis Pharmaceuticals
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