Clinical trials: Neuromuscular Disorder
Neuromuscular Disease, Neuromuscular Diseases (NMD)
A Phase 3 Study of NTLA-2001 in ATTRv-PN
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
- Conditions
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary
- Trial name
- NCT06672237
- Testing
- nexiguran ziclumeran
- Where
- 14 sites in Argentina, Brazil and 4 more
- Volunteers
- 60 people
- Sponsor
- Intellia Therapeutics
Back Pain
Spinal Cord Stimulation for Chronic and Intractable Back Pain
The purpose of this study is to evaluate the efficacy of spinal cord stimulation (SCS) using the Precision implantable neurostimulation device for chronic and intractable back pain in subjects who are not candidates for surgery…
- Trial name
- NCT00370695
- Testing
- Precision Spinal Cord Stimulation System
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Scientific Corporation
Amyotrophic Lateral Sclerosis
A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis
This study is conducted to evaluate the safety and tolerability of the drug product sNN0029, containing the growth factor VEGF165, when administered directly into one of the fluid filled cavities in the brain using an implanted…
- Trial name
- NCT00800501
- Testing
- sNN0029
- Where
- Belgium
- Volunteers
- 18 people
- Sponsor
- Newron Sweden AB
Diphtheria, Polio
Study of Immunogenicity and Safety of a Booster Dose of DTaP-IPV-HB-PRP~T Combined Vaccine in Healthy Turkish Infants
This is a follow-up of Study A3L10 (NCT00315055) Immunogenicity To describe the antibody persistence following a primary series vaccination of either DTaP-IPV-HB-PRP~T or PENTAXIM™ and ENGERIX B®.
- Conditions
- Diphtheria, Polio, Pertussis, Hepatitis B
- Trial name
- NCT00619502
- Testing
- DTaP-IPV-HB-PRP~T vaccine
- Where
- Turkey (Türkiye)
- Volunteers
- 254 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Fibromyalgia
Evaluation of Magnetic Fields to Treat Fibromyalgia
The purpose of this study is to see if a device called the Resonator can help to reduce pain and improve aspects of health and quality of life for people with fibromyalgia.
- Trial name
- NCT01262131
- Testing
- magnetic therapy protocol A, magnetic therapy Protocol B
- Volunteers
- 13 people
- Sponsor
- pico-tesla Magnetic Therapies, LLC
Generalized Myasthenia Gravis
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.
- Trial name
- ADAPT Jr (NCT04833894)
- Testing
- Efgartigimod IV
- Where
- 25 sites in United States, Austria and 10 more
- Volunteers
- 15 people
- Sponsor
- argenx
Refractory Generalized Myasthenia Gravis
Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.
- Trial name
- NCT01997229
- Testing
- Eculizumab
- Where
- 114 sites in United States, Argentina and 19 more
- Volunteers
- 125 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Spinal Muscular Atrophy
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
The aim of this retrospective medical chart review is to describe the clinical outcomes, clinical characteristics, and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with onasemnogene abeparvovec (OA)…
- Trial name
- SMA-REAL (NCT07737756)
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
Fructose Metabolism, Inborn Errors, Glycogen Storage Disease
Biomarker for Glycogen Storage Diseases (BioGlycogen)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Glycogen Storage Diseases from plasma. Testing for clinical robustness, specificity and long-term stability of the biomarker.
- Conditions
- Fructose Metabolism, Inborn Errors, Glycogen Storage Disease, Glycogen Storage Disease Type I, Glycogen Storage Disease Type II
- Trial name
- BioGlycogen (NCT02385162)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Myasthenia Gravis
Pharmacodynamic Study of CK-2017357 in Patients With Generalized Myasthenia Gravis
The primary objective of this early-stage clinical study is to demonstrate an effect of single doses of CK-2017357 on measures of skeletal muscle function and fatigability in patients with generalized myasthenia gravis (MG).
- Trial name
- NCT01268280
- Testing
- 250 mg CK-2017357, 500 mg CK-2017357
- Where
- 15 sites in United States
- Volunteers
- 32 people
- Sponsor
- Cytokinetics
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.
AChR-Ab Seropositive Generalized Myasthenia Gravis, Myasthenia Gravis (MG)
ADAPT Forward 2 - ISA2 - a Study to Evaluate the Safety, Tolerability and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis
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), MG - Myasthenia Gravis, gMG
- Trial name
- ADAPT Forward2 (NCT07673627)
- Testing
- Empasiprubart IV, Efgartigimod PH20 SC PFS
- Where
- 8 sites in United States, Greece and 1 more
- Volunteers
- 40 people
- Sponsor
- argenx
Myositis
A Study to Understand How the Study Medicine (PF-06823859) Works in People With Active Idiopathic Inflammatory Myopathies [Dermatomyositis (DM) and Polymyositis (PM)]
The purpose of the study is to understand how the study medicine PF-06823859 (dazukibart) works in people with idiopathic inflammatory myopathies (DM and PM).
- Trial name
- NCT05895786
- Testing
- PF-06823859
- Where
- 138 sites in United States, Argentina and 18 more
- Volunteers
- 318 people
- Sponsor
- Pfizer
Chronic Pain, Spasticity, Muscle
Medtronic PSR TDD PMCF
The purpose of this study is to provide continuing evaluation and reporting of safety and performance of the SynchroMed II Infusion System within its intended use. Data will support post-market surveillance obligations.
- Trial name
- NCT04714385
- Where
- 15 sites in Belgium, Czechia and 6 more
- Volunteers
- 209 people
- Sponsor
- Medtronic
Systemic Lupus Erythematosus, Lupus Nephritis
A Study of KITE-363 in Participants With Refractory Autoimmune Diseases
This study will have two Phases: Phase 1a and Phase 1b. The goal of this clinical study is to learn more about the study drug KITE-363, to establish dosing, tolerability, safety, and preliminary efficacy of KITE-363 in…
- Conditions
- Systemic Lupus Erythematosus, Lupus Nephritis, Systemic Sclerosis, Idiopathic Inflammatory Myopathy
- Trial name
- NCT07038447
- Testing
- KITE-363, Fludarabine, Cyclophosphamide
- Where
- 10 sites in United States, Australia and 2 more
- Volunteers
- 52 people
- Sponsor
- Kite, A Gilead Company
Venous Leg Ulcer
Intermittent Pneumatic Compression of the Foot vs Geko Plus R-2 Neuromuscular Electrostimulation Device in Venous Leg Ulcer Patients
Chronic venous leg ulcers (VLU) are painful, debilitating wounds that place a significant burden on the patient, their family, and healthcare resources.
- Trial name
- NCT02912858
- Testing
- geko plus R-2
- Sponsor
- Firstkind Ltd
Amyotrophic Lateral Sclerosis
A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.
- Trial name
- COURAGE-ALS (NCT04944784)
- Testing
- Reldesemtiv
- Where
- 84 sites in United States, Australia and 14 more
- Volunteers
- 489 people
- Sponsor
- Cytokinetics
Guillain-Barré Syndrome
A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barré Syndrome (GBS)
This study is a multi center, open-label, study of ANX005 in combination with IVIg in subjects diagnosed with GBS.
- Trial name
- NCT04035135
- Testing
- ANX005, Intravenous immunoglobulin
- Where
- 2 sites in Bangladesh, Denmark
- Volunteers
- 14 people
- Sponsor
- Annexon, Inc.
Pediatrics, Muscle Spasticity
BOTOX® Open-Label Treatment in Pediatric Upper Limb Spasticity
This study will evaluate the long-term safety of BOTOX® (botulinum toxin Type A) for the treatment of pediatric upper limb spasticity.
- Conditions
- Pediatrics, Muscle Spasticity, Cerebral Palsy, Stroke
- Trial name
- NCT01603615
- Testing
- Botulinum Toxin Type A
- Where
- 48 sites in United States, Canada and 7 more
- Volunteers
- 220 people
- Sponsor
- Allergan
Fibromyalgia
Pregabalin In Adolescent Patients With Fibromyalgia
The main purpose of this study is to evaluate the long-term safety of pregabalin in adolescent patients who participated in the previous fibromyalgia study (A0081180) and who wish to recieve open-label pregabalin
- Trial name
- NCT01020526
- Testing
- pregabalin
- Where
- 26 sites in United States, Czechia and 1 more
- Volunteers
- 63 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
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