Clinical trials: Neuromuscular Disorder
Amyotrophic Lateral Sclerosis (ALS)
A Study of Single Ascending Doses of NI112 in Adults With Amyotrophic Lateral Sclerosis (ALS)
This is a first-in-human study of NI112, an investigational medicine given as an injection under the skin, in adults with amyotrophic lateral sclerosis (ALS).
- Trial name
- NCT07906665
- Testing
- NI112
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Sachi Bioworks Inc.
Amyotrophic Lateral Sclerosis (ALS)
Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases
An open, multi- center phase Ⅰ clinical study evaluating the safety and efficacy of autologous human polyclonal regulatory T cell injection (NP001 cell injection) in patients with Neurodegenerative diseases (ALS).
- Trial name
- NCT06671236
- Testing
- Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Novabio Therapeutics
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
This is a follow-up study of subjects who received NTLA-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT05697861
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
Asthma, Heart Failure
The General Breathing Record Study
This study will test the use of a new handheld device (called the N-Tidal C), that measures a person's tidal breath carbon dioxide, in diagnosing the cause of someone's breathlessness.
- Conditions
- Asthma, Heart Failure, Pneumonia, Motor Neuron Disease
- Trial name
- GBRS (NCT03356288)
- Where
- United Kingdom
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- TidalSense
Muscular Dystrophy, Duchenne
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety…
- Trial name
- NCT02369731
- Where
- 71 sites in Austria, Brazil and 14 more
- Volunteers
- 316 people
- Sponsor
- PTC Therapeutics
Myotonic Dystrophy Type 1
Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance, ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and strength, pain, gastrointestinal functioning, and…
- Trial name
- NCT00577577
- Testing
- rhIGF-I/rhIGFBP-3
- Where
- 12 sites in United States
- Volunteers
- 69 people
- Sponsor
- Insmed Incorporated
Refractory Myasthenia Gravis
Allogeneic CD19-CAR-NK Cells in Patients With Refractory Myasthenia Gravis
This study is a single-center, prospective, nonrandom, single-arm trial.
- Trial name
- NCT07243366
- Testing
- CD19-CAR-NK Cells Injection
- Volunteers
- 15 people
- Sponsor
- Guangdong ProCapZoom Biosciences Co., Ltd.
Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis
Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)
The goal of this two-part clinical trial is: 1) to evaluate the safety and efficacy of Debamestrocel - MSC-NTF (NurOwn) compared to placebo in participants with early symptomatic ALS and moderate disease presentation in ALS…
- Trial name
- NCT06973629
- Testing
- Debamestrocel - MSC-NTF (NurOwn)
- Where
- 15 sites in United States
- Volunteers
- 200 people
- Sponsor
- Brainstorm-Cell Therapeutics
Cerebral Palsy, SMA II
Evaluation of the Exoskeleton ATLAS 2030 as Robot-assisted Physical Therapy to Children With Neuromuscular Diseases
This study aims to evaluate the safety, usability and acceptability of a motorized mobility assistance exoskeleton. The protocol explores the use of the motorized device during static and dynamic rehabilitation sessions focused…
- Conditions
- Cerebral Palsy, SMA II, Neuromuscular Diseases
- Trial name
- NCT04837157
- Testing
- Exoskeleton rehabilitation
- Where
- Spain
- Volunteers
- 6 people
- Sponsor
- MarsiBionics
Neuropathic Pain
Evaluation of an ATP-Containing Parenteral Vitamin B Complex in Patients With Symptomatic Diabetic Polyneuropathy
Epinosine B Forte ampoule is an intramuscular injectable supplement containing adenosine triphosphate (ATP), Cocarboxylase (vitamin B1 derivative), vitamin B12, and nicotinamide (vitamin B3).
- Trial name
- NCT07296354
- Testing
- adenosine triphosphate, Vitamin B Complex
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Egyptian International Pharmaceutical Industries Co
Proteus Syndrome, PIK3CA-Related Overgrowth Spectrum (PROS)
Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies
ARQ 092 is being investigated for patients with overgrowth diseases and/or vascular anomalies with genetic alterations of the PI3K/AKT pathway and may be available for patients who are ineligible for an ongoing ARQ 092 clinical…
- Conditions
- Proteus Syndrome, PIK3CA-Related Overgrowth Spectrum (PROS), Growth Disorders
- Trial name
- NCT03317366
- Testing
- ARQ 092
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Fibromyalgia, Pain
Pain Relief Investigation of Neuromodulation Therapy in an Adult Fibromyalgia Population
This study is being performed to determine the safety and efficacy of Fralex Neuromodulation Therapy (F-NMT), delivered by the Fralex PRIMA device, in reducing chronic musculoskeletal pain associated with Fibromyalgia.
- Trial name
- NCT00450905
- Testing
- PRIMA Device
- Where
- 21 sites in United States, Canada
- Volunteers
- 200 people
- Sponsor
- Fralex Therapeutics
Systemic Lupus Erythematosus (SLE), Idiopathic Inflammatory Myopathies(IIM)
A Study of LUCAR-G79D in Subjects With Relapsed/Refractory Systemic Lupus Erythematosus (r/r SLE) and Idiopathic Inflammatory Myopathies (r/r IIM)
This is a prospective, single-arm, open-label and dose-escalation investigator initialed study to evaluate LUCAR-G79D in adult subjects with r/r SLE and r/r IIM.
- Trial name
- NCT07331272
- Testing
- LUCAR-G79D T cells
- Where
- 5 sites in China
- Volunteers
- 38 people
- Sponsor
- Nanjing Legend Biotech Co.
Spinal Muscular Atrophy, Neuromuscular Diseases
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec…
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
- Trial name
- RESILIENT (NCT05337553)
- Testing
- taldefgrobep alfa
- Where
- 53 sites in United States, Belgium and 7 more
- Volunteers
- 269 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Polio, Poliomyelitis
Sequential Inactivated Poliomyelitis Vaccine Followed by Oral Poliomyelitis Vaccine Versus Oral Poliomyelitis Vaccine
The purpose of this study is to generate data to support the registration extension of IMOVAX Polio to be used in a sequential vaccination.
- Trial name
- NCT01475539
- Testing
- Injectable inactivated types 1, 2 and 3 poliovirus + Live oral Polio, Live oral poliovirus type 1, 2 and 3
- Where
- China
- Volunteers
- 456 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Polio, Measles
the Five-year Antibody Persistence After Immunization With IPV, MMR and HepA-L Vaccines
This study evaluated the antibody persistence of Chinese children five years after they received four doses of sIPV, two doses of MMR vaccine and one dose of HepA-L vaccine.
- Conditions
- Polio, Measles, Rubella, Mumps
- Trial name
- NCT07338851
- Where
- 6 sites in China
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- China National Biotec Group Company Limited
Autoimmune Diseases, Cardiovascular Disorders
Mesenchymal Stem Cells for the Treatment of Various Chronic and Acute Conditions
This multi-arm, multi-site study investigates the safety, tolerability, and efficacy of stem cell therapy for the treatment of various acute and chronic conditions.
- Conditions
- Autoimmune Diseases, Cardiovascular Disorders, Diabetes Complications, Integumentary Disease
- Trial name
- NCT04684602
- Testing
- PrimePro™/ PrimeMSK™
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Thomas Advanced Medical LLC
Low Muscle Strength, Malnutrition
Efficacy of Oral Nutritional Supplement in Community-Dwelling Adults Following Acute Care
The study aims to determine the effects of Oral Nutritional Supplement (ONS) added to usual care on body weight compared with usual care alone in community-dwelling adults aged 55 years and older who are at risk of malnutrition…
- Trial name
- NCT07753239
- Testing
- ONS, Standard of Care (SOC)
- Where
- 7 sites in Thailand
- Volunteers
- 304 people
- Sponsor
- Abbott Nutrition
Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
A Study of TAK-411 in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
CIDP is an autoimmune disease. This means that the body's germ fighting (immune) system attacks itself. In CIDP, the immune system attacks the protective covering around the nerves called myelin.
- Trial name
- CASCA (NCT06798012)
- Testing
- TAK-411
- Where
- 22 sites in United States, Canada and 1 more
- Volunteers
- 36 people
- Sponsor
- Takeda
Bacterial Infections, Virus Diseases
Safety, Tolerability, and Immunogenicity of V419 in Healthy Infants When Given at 2, 3, 4 and 12 Months (V419-007)
This study will determine whether participants who receive the vaccine V419 at 2, 3, 4, and 12 months of age have an acceptable immune response to the vaccine.
- Trial name
- NCT01341639
- Testing
- V419, INFANRIX™ hexa, RotaTeq
- Volunteers
- 1,250 people, healthy volunteers welcome
- Sponsor
- MCM Vaccines B.V.
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