Clinical trials: Neuromuscular Disorder
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.
Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc)
A Clinical Study of YTS109 Cell in R/R Autoimmune Diseases
This study evaluates the safety and efficacy of YTS109 cells in adults with relapsed/refractory autoimmune diseases, such as Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc), etc.
- Conditions
- Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc), Inflammatory Myopathy, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
- Trial name
- NCT06978647
- Testing
- YTS109 cell
- Where
- China
- Volunteers
- 12 people
- Sponsor
- China Immunotech (Beijing) Biotechnology Co., Ltd.
Neuralgia, Postherpetic
Dose Ranging Trial to Determine the Safety and Efficacy of EMA401 in Patients With PHN
The study is a randomized, double blind, placebo-controlled, parallel-group comparison (two dose levels of EMA401 versus a placebo group), of safety and efficacy in patients with postherpetic neuralgia.
- Trial name
- NCT02426411
- Testing
- EMA401 200 mg, EMA401 600 mg
- Sponsor
- Spinifex Pharmaceuticals Pty Ltd
Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
A Study of HyQvia in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) in Routine Clinical Care
The main aims of this study are to understand why adults with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) chose a certain treatment, why they changed to HyQvia from another therapy, how satisfied they are…
- Trial name
- HYbISCUE (NCT06538064)
- Testing
- No Intervention
- Where
- 15 sites in United States, Denmark and 1 more
- Volunteers
- 30 people
- Sponsor
- Takeda
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.
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
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
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), CIDP
Pre-Approval Access for Efgartigimod PH20 SC in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
The aim of the Pre-Approval Access program is to provide efgartigimod PH20 SC treatment to patients with CIDP who are ineligible to participate in a clinical study and have unmet medical need despite available treatment options.
- Trial name
- NCT04777734
- Testing
- Efgartigimod PH20 SC
- Sponsor
- argenx
Radiculopathy, Myelopathy
Pivotal IDE Study of the BRYAN(R) Cervical Disc Prosthesis in the Treatment of DDD Versus ACDF
The purpose of this study is to establish the safety and effectiveness of the BRYAN(R) Cervical Disc Prosthesis in treating single-level degenerative disc disease of the cervical spine.
- Conditions
- Radiculopathy, Myelopathy, Cervical Degenerative Disc Disease
- Trial name
- NCT00437190
- Testing
- ATLANTIS™ Cervical Plate System and allograft, BRYAN Cervical Disc Prosthesis
- Where
- 38 sites in United States
- Volunteers
- 494 people
- Sponsor
- Medtronic Spinal and Biologics
Diabetic Neuropathies, Diabetes Mellitus, Insulin-Dependent
Treatment for Symptomatic Peripheral Neuropathy in Patients With Diabetes
The purpose of this protocol is to determine if an investigational drug known as LY333531 is effective in treating nerve malfunction in diabetes.
- Conditions
- Diabetic Neuropathies, Diabetes Mellitus, Insulin-Dependent, Diabetes Mellitus, Non-Insulin-Dependent
- Trial name
- NCT00044408
- Testing
- Ruboxistaurin mesylate
- Where
- 35 sites in United States, Canada and 6 more
- Volunteers
- 200 people
- Sponsor
- Chromaderm, Inc.
Diphtheria, Tetanus
To Evaluate the Immunogenicity and Safety of DTaP-IPV Vaccine Administered as a Boosting Dose to Healthy Children of 4-6 Years
The study objective is to assess the immunogenicity and safety of DTaP-IPV combination vaccine administered as a boosting dose to healthy 4 to 6-year-old children who received three doses of primary immunization against…
- Conditions
- Diphtheria, Tetanus, Pertussis, Poliomyelitis
- Trial name
- Aladdin (NCT04618640)
- Testing
- DTaP-IPV combination vaccine
- Where
- 26 sites in South Korea
- Volunteers
- 249 people, healthy volunteers welcome
- Sponsor
- Boryung Biopharma Co., Ltd.
Spasticity, Muscle
Validation of the Spasticity Related Quality of Life Questionnaire
The purpose of the study is to describe the psychometric properties (validity, reliability and responsiveness) of the SQOL-6D instrument in the context of routine clinical treatment of upper limb spasticity.
- Trial name
- SQOL-6D (NCT03442660)
- Testing
- Data collection
- Where
- 8 sites in United Kingdom
- Volunteers
- 104 people
- Sponsor
- Ipsen
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
Cervical Disc Disease
A Clinical Trial Comparing the BAGUERA C to the Marketed Mobi-C® for the Treatment of Cervical Disc Disease at 2 Contiguous Levels
The proposed investigation is a multi-center, prospective, randomized, controlled comparison of the BAGUERA®C to the control, a similar, legally marketed total disc replacement device in subjects with symptomatic cervical disc…
- Trial name
- NCT04564885
- Testing
- BAGUERA®C Cervical Disc Prosthesis, Mobi-C Cervical Disc
- Where
- 26 sites in United States
- Volunteers
- 300 people
- Sponsor
- Spineart USA
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
Upper Extremity Impairments, Cerebral Palsy (CP)
Obi Medical Robot: Evaluating Effectiveness Related to Usability
This mixed-methods usability study evaluates the effectiveness of the Obi Gen 3 robotic feeding device in meeting user needs among providers, caregivers, and patients.
- Conditions
- Upper Extremity Impairments, Cerebral Palsy (CP), ALS - Amyotrophic Lateral Sclerosis, Rett Syndrome
- Trial name
- NCT07151950
- Testing
- One-week trial of Obi Medical device, which provides robotic assistance with feeding to persons with upper extremity impairments who are unable to self-feed in the traditional manner using utensils
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Desin LLC
Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathy
A Study of CC-97540, CD-19-Targeted Nex-T CAR T Cells, in Participants With Severe, Refractory Autoimmune Diseases (Breakfree-1)
The purpose of this study is to establish the tolerability, preliminary efficacy, and pharmacokinetics of CC-97540 in participants with severe, refractory autoimmune diseases (Breakfree-1).
- Conditions
- Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathy, Systemic Sclerosis, Rheumatoid Arthritis
- Trial name
- NCT05869955
- Testing
- CC-97540, Fludarabine, Cyclophosphamide
- Where
- 46 sites in United States, Belgium and 4 more
- Volunteers
- 270 people
- Sponsor
- Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
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
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