Clinical trials: Neuromuscular Disorder
- Condition Neuromuscular Disorder
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
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.
Amyotrophic Lateral Sclerosis
Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS)
This was a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820 compared with placebo in male and female participants, 18 to 80…
- Trial name
- HIMALAYA (NCT05237284)
- Testing
- SAR443820
- Where
- 63 sites in United States, Belgium and 11 more
- Volunteers
- 305 people
- Sponsor
- Sanofi
TTR-mediated Amyloidosis, Amyloidosis, Hereditary
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis.
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies
- Trial name
- NCT01960348
- Testing
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Where
- 52 sites in United States, Argentina and 19 more
- Volunteers
- 225 people
- Sponsor
- Alnylam Pharmaceuticals
Hepatitis B, Polio
Immunogenicity of DTaP-IPV-Hep B-PRP~T Combined Vaccine Compared With PENTAXIM™ and ENGERIX B® at 2-3-4 Months Schedule
To demonstrate that the immune response to hepatitis B antigen of the DTaP-IPV-Hep B-PRP~T is non-inferior to that of the association of PENTAXIM™ and ENGERIX B® one month after a three dose (2-3-4 month) primary series.
- Conditions
- Hepatitis B, Polio, Diphtheria, Pertussis
- Trial name
- NCT00315055
- Testing
- DTaP-IPV-HB-PRP~T vaccine, DTaP-IPV//PRP~T combined, Hepatitis B vaccine
- Where
- Turkey (Türkiye)
- Volunteers
- 310 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Tetanus, Poliomyelitis
Study to Evaluate Antibody Persistence in Children Previously Vaccinated With Infanrix Hexa™ or Infanrix-IPV/Hib™
The aim of this study is to assess antibody persistence in infants who received three doses of Infanrix hexa™ (DTPa-HBV-IPV/Hib) or Infanrix-IPV/Hib™ (DTPa-IPV/Hib) at 3, 5 and 11 months of age in study NCT00307034.
- Conditions
- Tetanus, Poliomyelitis, Haemophilus Influenzae Type b, Acellular Pertussis
- Trial name
- NCT01358825
- Testing
- Blood Sampling
- Where
- 4 sites in Norway, Sweden
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Acellular Pertussis, Diphtheria
Immunogenicity and Reactogenicity of a Booster Dose of GSK Bio's DTPa-HBV-IPV/Hib Vaccine
The purpose of this booster study is to evaluate, in subjects primed in the primary study 106786, the persistence, at the time of the booster vaccination, of antibodies elicited by the different formulation of DTPa-HBV-IPV/ Hib…
- Conditions
- Acellular Pertussis, Diphtheria, Poliomyelitis, Haemophilus Influenzae Type b
- Trial name
- NCT00627458
- Testing
- Infanrix Hexa
- Where
- 6 sites in Finland
- Volunteers
- 403 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis
Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS)
The purpose of this research study is to evaluate the safety and effectiveness of CK-2017357 when taken with or without riluzole (also called Rilutek®) in patients with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT01709149
- Testing
- CK-2017357, Riluzole
- Where
- 75 sites in United States, Canada and 6 more
- Volunteers
- 711 people
- Sponsor
- Cytokinetics
Healthy, Primary Immune Thrombocytopenia
A First in HumanTrial to Evaluate The Safety, Tolerability, And Pharmacokinetics Of PF-06755347
This phase 1 single ascending dose study will provide a first in human assessment of safety and tolerability of PF-06755347 in healthy adult males as well as adult males and females with Immune Thrombocytopenia (ITP).
- Conditions
- Healthy, Primary Immune Thrombocytopenia, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
- Trial name
- NCT03275740
- Testing
- PF-06755347 intravenous healthy participant, PF-06755347 subcutaneous healthy participant, PF-06755347 subcutaneous ITP
- Where
- 6 sites in United States, Belgium and 3 more
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Pfizer
Fibromyalgia
A Study Comparing Duloxetine and Placebo in the Treatment of Fibromyalgia
The purpose of this study is to confirm the efficacy and safety of duloxetine 60-120 mg once daily in comparison to placebo on symptom improvement in patients meeting criteria for fibromyalgia aged 18 and older.
- Trial name
- NCT00673452
- Testing
- duloxetine hydrochloride
- Where
- 46 sites in United States, Puerto Rico
- Volunteers
- 530 people
- Sponsor
- Eli Lilly and Company
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
- Trial name
- NCT04886518
- Testing
- Pitolisant Oral Tablet
- Where
- 13 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Harmony Biosciences Management, Inc.
Herniated Disc, Radiculopathy
Slotted Hole Versus Fixed Hole C-Tek
The purpose of this study is to compare the fusion rates between the EBI, LLC C-Tek™ Anterior Cervical Plate, Slotted Hole Design versus the Fixed Hole Design.
- Conditions
- Herniated Disc, Radiculopathy, Spondylolysis
- Trial name
- NCT00585923
- Testing
- Fixed hole C-Tek™ Plate, Slotted hole C-Tek™ Plate
- Where
- United States
- Volunteers
- 115 people
- Sponsor
- Zimmer Biomet
ICU Acquired Weakness
Evaluation of FDY-5301 in Major Trauma Patients in ICU
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of FDY-5301 compared to placebo in major trauma ICU patients at risk of intensive care unit acquired weakness (ICUAW)
- Trial name
- NCT04430283
- Testing
- FDY-5301
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Faraday Pharmaceuticals, Inc.
RYR-1 Myopathy
S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
This study proposes to test S 48168 (ARM210) in a Phase 1 trial in RYR1-RM patients, specifically. The objectives of this study are to explore the safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD)/target…
- Trial name
- NCT04141670
- Testing
- S48168
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- RyCarma Therapeutics, Inc.
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
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
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.