Clinical trials: Neuromuscular Disease
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Snoring, Obstructive Sleep Apnea
Intraoral Tongue Stimulation for Treatment of Primary Snoring
Intraoral Neuromuscular Training for Treatment of Snoring Objective Studies show that improving pharyngeal muscle tone can ameliorate obstructive sleep apnoea (OSA) and snoring.
- Trial name
- NCT03829956
- Testing
- Intraoral tongue stimulator (Snoozeal)
- Where
- United Kingdom
- Volunteers
- 115 people
- Sponsor
- Signifier Medical Technologies
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
- Trial name
- LELANTOS-1 (NCT04371666)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 98 people
- Sponsor
- Kyntra Bio
Duchenne Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males…
- Trial name
- PTCEMF (NCT03642145)
- Testing
- Deflazacort
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
- Trial name
- NCT05693142
- Testing
- RGX-202
- Where
- 23 sites in United States, Canada
- Volunteers
- 65 people
- Sponsor
- REGENXBIO Inc.
Healthy Volunteer, Muscular Atrophy, Spinal
Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.
This non-drug, single center, 24-week, longitudinal study in ambulant spinal muscular atrophy (SMA) patients and in age- and gender-matched healthy volunteers will assess the detection of disease progression by magnetic resonance…
- Trial name
- NCT02044029
- Where
- Switzerland
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Myasthenia Gravis, Generalized
A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis
The purpose of this study is to evaluate the pharmacodynamic (PD) effect, safety, and tolerability of Povetacicept in participants with generalized myasthenia gravis (gMG).
- Trial name
- ETNA (NCT07501702)
- Testing
- Povetacicept
- Where
- 31 sites in United States, Australia and 3 more
- Volunteers
- 34 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Postherpetic Neuralgia
Study to Assess the Efficacy, Safety, and Tolerability of ADL5747 in Participants With Postherpetic Neuralgia
The primary purpose of this study is to evaluate the analgesic efficacy of ADL5747 in participants with postherpetic neuralgia (PHN).
- Trial name
- NCT01058642
- Testing
- ADL5747, Pregabalin
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Cubist Pharmaceuticals LLC, a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Coronary Artery Disease, Pain
Study of Ranexa in Patients With Coronary Artery Disease and Painful Polyneuropathy
This study was to determine whether ranolazine was effective in the treatment of neuropathic pain in patients with coronary artery disease.
- Conditions
- Coronary Artery Disease, Pain, Peripheral Nervous System Diseases, Polyneuropathy
- Trial name
- NCT00832572
- Testing
- Ranolazine
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Gilead Sciences
Myasthenia Gravis, MuSK, AChR Myasthenia Gravis
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.
- Trial name
- NCT03579966
- Testing
- Amifampridine Phosphate
- Where
- 3 sites in United States, Italy
- Volunteers
- 63 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Diabetic Neuropathic Pain
A Multicenter Study Comparing the Analgesic Effects and Safety of ABT-639 Compared to Placebo in Subjects With Diabetic Neuropathic Pain
People with diabetes can, over time develop nerve damage throughout the body with symptoms such as pain, tingling, or numbness (loss of feeling) in the hands, arms, feet and legs.
- Trial name
- NCT01345045
- Testing
- ABT-639, pregabalin
- Where
- 35 sites in United States, Czechia and 3 more
- Volunteers
- 193 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Poliomyelitis, Tetanus
Immunogenicity and Safety of GlaxoSmithKline Biologicals' Infanrix™-IPV+Hib Vaccine
This study is designed to evaluate the safety and immunogenicity of Infanrix™-IPV+Hib vaccine when administered as a primary vaccination course to healthy Korean infants at 2, 4 and 6 months of age.
- Conditions
- Poliomyelitis, Tetanus, Acellular Pertussis, Diphtheria
- Trial name
- NCT01309646
- Testing
- Infanrix™-IPV+Hib, Infanrix™ IPV, Hiberix™
- Where
- 11 sites in South Korea
- Volunteers
- 454 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Diphtheria, Tetanus
Regulatory Post-Marketing Surveillance Study for TETRAXIM™
The purpose of this study is to assess the safety of TETRAXIM™ administered in routine clinical practice according to Korea Food and Drug Administration Notification No.
- Conditions
- Diphtheria, Tetanus, Pertussis, Poliomyelitis
- Trial name
- NCT01437423
- Testing
- TETRAXIM™: Combined adsorbed diphtheria, tetanus, acellular pertussis and enhanced inactivated polio
- Where
- South Korea
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Dermatomyositis, Polymyositis
A Study to Understand How the Study Medicine Dazukibart Works in People With Idiopathic Inflammatory Myopathies
The purpose of this study is to understand how the study medicine, dazukibart, works in people with active idiopathic inflammatory myopathies (dermatomyositis [DM] or polymyositis [PM]).
- Trial name
- NCT06698796
- Testing
- Dazukibart
- Where
- 26 sites in United States, Argentina and 10 more
- Volunteers
- 211 people
- Sponsor
- Pfizer
Neuralgia
P4 (Pregabalin for Peripheral Posttraumatic Pain)
To evaluate the efficacy of pregabalin compared to placebo in the treatment of posttraumatic peripheral neuropathic pain
- Trial name
- P4 (NCT00292188)
- Testing
- pregabalin
- Where
- 44 sites in Belgium, Canada and 9 more
- Volunteers
- 255 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Cubital Tunnel Syndrome, Nerve Compression
Promoting Healing of Nerves Through Electrical Stimulation
This study is evaluating a new therapeutic use of electrical stimulation to promote nerve healing and improve functional recovery following surgical intervention for nerve compression.
- Conditions
- Cubital Tunnel Syndrome, Nerve Compression, Nerve Injury, Ulnar Neuropathies
- Trial name
- PHONES (NCT04662320)
- Testing
- Checkpoint BEST System
- Where
- 5 sites in United States
- Volunteers
- 100 people
- Sponsor
- Checkpoint Surgical Inc.
Central Nervous System (CNS) Lesions, Brain Metastases
Phase 2 Ascending Dose Safety and Efficacy Study of RVP-001, a Manganese-based MRI Contrast Agent
This Phase 2 clinical trial will study RVP-001, a new manganese-based MRI contrast agent, in people who are known to have gadolinium-enhancing central nervous system (CNS) lesions, for example brain tumors or multiple sclerosis.
- Conditions
- Central Nervous System (CNS) Lesions, Brain Metastases, Brain Neoplasms, Brain Neoplasms, Benign
- Trial name
- NCT06322342
- Testing
- RVP-001
- Where
- 5 sites in United States
- Volunteers
- 18 people
- Sponsor
- Reveal Pharmaceuticals Inc.
Generalized Myasthenia Gravis
Clinical Study of Cizutamig in Generalized Myasthenia Gravis (gMG)
The purpose of this study is to assess the safety, tolerability, PK, PD, immunogenicity, and preliminary clinical activity of Cizutamig in patients with Generalized Myasthenia Gravis.
- Trial name
- NCT07215650
- Testing
- Cizutamig
- Where
- 10 sites in China
- Volunteers
- 44 people
- Sponsor
- Candid Therapeutics
Painful Diabetic Neuropathy, Diabetic Neuropathy, Painful
Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy
The purpose of this study is to explore the overall safety profile and durability of efficacy of Engensis (VM202) in painful diabetic peripheral neuropathy.
- Trial name
- NCT04055090
- Testing
- Long-Term Follow-Up of Patients who Received Engensis (VM202)
- Where
- 14 sites in United States
- Volunteers
- 101 people
- Sponsor
- Helixmith Co., Ltd.
Plexiform Neurofibroma
Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas
This study aims to demonstrate that in subjects with symptomatic, inoperable plexiform neurofibromas associated with neurofibromatosis type 1, TQ-B3234 capsules significantly improve the objective response rate at Week 24…
- Trial name
- NCT07407803
- Testing
- TQ-B3234 capsules
- Where
- 29 sites in China
- Volunteers
- 177 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
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