Clinical trials: Neuromuscular Disorder
Amyotrophic Lateral Sclerosis
An IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With ALS
The goal of this clinical trial is to evaluate the safety and efficacy of a single intrathecal injection of RJK002 in patients with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT06454682
- Testing
- RJK002 Intrathecal injection
- Where
- China
- Volunteers
- 9 people
- Sponsor
- RJK Biopharma Ltd
Systemic Lupus Erythematosus, Systemic Sclerosis
An Clinical Study of YTS109 Cell Injection in Subjects With Recurrent/Refractory Autoimmune Disease
An exploratory clinical study of the safety and efficacy of YTS109 cell injection in subjects with recurrent/refractory autoimmune disease
- Conditions
- Systemic Lupus Erythematosus, Systemic Sclerosis, Inflammatory Myopathy, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
- Trial name
- NCT06379646
- Testing
- YTS109
- Where
- China
- Volunteers
- 6 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
The purpose of this study is to: Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed Demonstrate…
- Trial name
- TRITON-PN (NCT07223203)
- Testing
- Nucresiran, Vutrisiran
- Where
- 61 sites in United States, Argentina and 20 more
- Volunteers
- 125 people
- Sponsor
- Alnylam Pharmaceuticals
Dermatomyositis
Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis
This is a Phase 2/3, double-blind, randomized, placebo-controlled, parallel group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in adult participants…
- Trial name
- NCT04999020
- Testing
- Ravulizumab
- Where
- 111 sites in United States, Australia and 11 more
- Volunteers
- 38 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
FSHD, FSHD1
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with…
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE (NCT05747924)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 90 people
- Sponsor
- Avidity Biosciences, Inc.
Iron Deficiency, Fibromyalgia
Blinded Randomized Placebo-Controlled Study Efficacy and Safety of Injectafer in ID Patients With Fibromyalgia
This study investigates the efficacy and safety of an IV iron Injectafer in the treatment of iron deficient patients with fibromyalgia.
- Trial name
- NCT02409459
- Testing
- Injectafer
- Where
- United States
- Volunteers
- 81 people
- Sponsor
- American Regent, Inc.
Herpes Zoster, Shingles
ZOSTAVAX™ (Zoster Vaccine Live) Long-term Effectiveness Study (V211-024)
This study will describe the impact of vaccination with ZOSTAVAX™ on the epidemiology of herpes zoster (HZ) in a cohort of vaccinated participants 50 years or age or older, compared to a cohort of unvaccinated participants.
- Trial name
- NCT01600079
- Testing
- ZOSTAVAX™
- Volunteers
- 1,505,647 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Amyotrophic Lateral Sclerosis
31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Amyotrophic Lateral Sclerosis (REPAIR-ALS)
REPAIR-ALS is a single-center open label pilot, sequential group, investigator and patient blinded study to assess the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been…
- Trial name
- REPAIR-ALS (NCT03843710)
- Testing
- Gold Nanocrystals
- Where
- United States
- Sponsor
- Clene Nanomedicine
Fibromyalgia
D-Ribose for Fatigue in Subjects With Fibromyalgia
To evaluate the safety and to determine the efficacy of D-ribose for the amelioration of fatigue in subjects with fibromyalgia. This is a phase IIa, randomized, double-blind, placebo-controlled, multi-center, multiple dose study…
- Trial name
- NCT01315210
- Testing
- D-Ribose Powder
- Where
- 9 sites in United States
- Volunteers
- 110 people
- Sponsor
- RiboCor, Inc.
AChR-Ab Seropositive Generalized Myasthenia Gravis, Myasthenia Gravis
ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod
This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis.
- Conditions
- AChR-Ab Seropositive Generalized Myasthenia Gravis, Myasthenia Gravis, MG, gMG
- Trial name
- NCT07284420
- Testing
- Efgartigimod IV, Empasiprubart IV
- Where
- 33 sites in United States, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- argenx
Diabetic Peripheral Neuropathy, Fibromyalgia
Pharmacokinetics of Two Formulation of Pregabalin
A Randomized, Single-Dose, Parallel-Group Study to Evaluate the Pharmacokinetic Profiles of Two Formulations of Pregabalin after Oral Administration in Healthy Volunteers under Fasting Conditions
- Conditions
- Diabetic Peripheral Neuropathy, Fibromyalgia, Partial Seizure Disorder
- Trial name
- NCT03712475
- Testing
- Pregabalin
- Where
- Taiwan
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Yung Shin Pharm. Ind. Co., Ltd.
Dermatomyositis
A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis
This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with dermatomyositis (DM).
- Trial name
- VALOR (NCT05437263)
- Testing
- Brepocitinib
- Where
- 109 sites in United States, Argentina and 20 more
- Volunteers
- 241 people
- Sponsor
- Priovant Therapeutics, Inc.
Diabetic Peripheral Neuropathy
A Study to Evaluate the Efficacy, Safety, and Tolerability of Tapentadol ER Compared With Placebo in Patients With Chronic, Painful Diabetic Peripheral Neuropathy
The purpose of this study is to evaluate the efficacy, safety, and tolerability of orally administered tapentadol extended release (ER) at dosages of 100 to 250 mg twice daily compared with placebo in patients with moderate to…
- Trial name
- NCT01041859
- Testing
- Tapentadol extended release (ER)
- Where
- 72 sites in United States, Canada and 1 more
- Volunteers
- 460 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Oculopharyngeal Muscular Dystrophy
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Subjects who have enrolled in the oculopharyngeal muscular dystrophy (OPMD) natural history study (Study BNTC-OPMD-NH-001) and have completed at least 6 months of follow up in Study BNTC-OPMD-NH-001 may be eligible to participate…
- Trial name
- NCT06185673
- Testing
- BB-301: Dose escalation phase 1b cohort 1, BB-301: Dose escalation phase 1b cohort 2, BB-301: Dose escalation phase 1b cohort 3
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Benitec Biopharma, Inc.
Neoplasms, Melanoma
Study of ASN003 in Subjects With Advanced Solid Tumors
The study is divided into two parts. The first part of the study will test various doses of ASN003 to find out the highest safe dose to test in three specific groups.
- Conditions
- Neoplasms, Melanoma, Colorectal Neoplasm, Carcinoma, Non-small Cell Lung
- Trial name
- NCT02961283
- Testing
- ASN003 ascending doses, ASN003 MTD
- Where
- 5 sites in United States
- Volunteers
- 24 people
- Sponsor
- Asana BioSciences
Lumbosacral Radicular Pain
Corticosteroid Lumbar Epidural Analgesia for Radiculopathy
This is a research study of SP-102, an experimental medication designed to relieve pain in patients with a specific type of leg pain.
- Trial name
- NCT03372161
- Testing
- SP-102
- Where
- 37 sites in United States
- Volunteers
- 401 people
- Sponsor
- Scilex Pharmaceuticals, Inc.
Facioscapulohumeral Muscular Dystrophy (FSHD)
Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies
- Trial name
- NCT02239224
- Testing
- ATYR1940
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 20 people
- Sponsor
- aTyr Pharma, Inc.
Muscle Spasticity
An Adult Spasticity Registry of OnabotulinumtoxinA Treatment
This is a registry study in adults with spasticity to determine onabotulinumtoxinA use in clinical practice. Treatment will be administered in accordance with physician standard practice.
- Trial name
- ASPIRE (NCT01930786)
- Testing
- onabotulinumtoxinA
- Where
- 55 sites in United States, France and 5 more
- Volunteers
- 744 people
- Sponsor
- Allergan
Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
Primary: The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus…
- Conditions
- Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired
- Trial name
- EAP-001 (NCT02189720)
- Testing
- Amifampridine Phosphate
- Where
- United States
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Pain Associated With Fibromyalgia
An Open-Label Extension Study of DS-5565 for 52 Weeks in Pain Associated With Fibromyalgia
This is an open-label study of DS-5565 in subjects who either completed participation in a preceding Phase 3 study of DS-5565 in fibromyalgia (FM); i.e.
- Trial name
- NCT02234583
- Testing
- DS-5565
- Where
- 276 sites in United States, Australia and 27 more
- Volunteers
- 2,485 people
- Sponsor
- Daiichi Sankyo
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