Clinical trials: Neuromuscular Disorders
Becker Muscular Dystrophy
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG).
- Trial name
- NCT01539772
- Where
- 16 sites in United States, Canada and 2 more
- Volunteers
- 85 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Neurologic Disorder, Duchenne
Physical Therapy Treatment on Children and Adolescents With Neurological Pathologies
To evaluate the positive effects of a home-based physical therapy intervention added to the usual physical therapy programs performed in children with neurological pathologies that induce sensorimotor impairments that affect…
- Conditions
- Neurologic Disorder, Duchenne, Pediatric Disorder, Sensorimotor Disorder Nos
- Trial name
- NCT05313295
- Testing
- Home-based physiotherapy, Usual Physiotherapy
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- Universidad de Almeria
Nerve Injury
Autologous Fat in Peripheral Nerve Injury
The aim of this study is to assess the efficacy of autologous fat graft in enhancing peripheral nerve regeneration. The investigators hypothesize that fat grafting will allow for faster and greater recovery of motor and sensory…
- Trial name
- NCT04653129
- Testing
- Autologous Fat Grafting, Primary Nerve Repair
- Volunteers
- 44 people
- Sponsor
- Assiut University
Muscular Atrophy, Spinal
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
- Trial name
- NCT04256265
- Testing
- Risdiplam
- Where
- 29 sites in United States
- Sponsor
- Genentech, Inc.
Duchenne Muscular Dystrophy
Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study
Dushenne muscular dystrophy (DMD), which is characterized by the deficiency of dystrophin protein, prevents the muscle from maintaining its normal activity, causing progressive damage to the heart, respiratory muscles and…
- Trial name
- NCT07172737
- Testing
- Respiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Izmir Katip Celebi University
Systemic Lupus Erythematosus (SLE), Diffuse Systemic Sclerosis
YTS109 in Pediatric Relapsed/Refractory Autoimmune Diseases
This exploratory, single-arm, open-label study will evaluate the safety and preliminary efficacy of YTS109 cell therapy in pediatric patients with relapsed/refractory autoimmune diseases, including systemic lupus erythematosus…
- Conditions
- Systemic Lupus Erythematosus (SLE), Diffuse Systemic Sclerosis, Idiopathic Inflammatory Myopathies (IIMs), ANCA Associated Vasculitis (AAV)
- Trial name
- NCT07439029
- Testing
- YTS109 cell
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Fudan University
Myopathy, Neuropathy
Identification of Tongue Involvement in Late-Onset Pompe Disease
This purpose of this study is to determine if tongue strength and tongue ultrasound measurements differentiates patients with untreated late-onset Pompe Disease (LOPD) from patients with acquires/hereditary myopathies or…
- Conditions
- Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
- Trial name
- NCT02765828
- Testing
- Observational study
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- Duke University
Diphtheria, Tetanus
Study of DTaP-IPV-Hep B-PRP~T Combined Vaccine Compared With PENTAXIM™ and ENGERIX B® PEDIATRICO in Argentinean Infants
Primary Objective: To demonstrate that the immune response of the DTaP-IPV-Hep B-PRP~T is non-inferior for all valences to those of the association of PENTAXIM™ and ENGERIX B® PEDIATRICO one month after a three-dose primary…
- Conditions
- Diphtheria, Tetanus, Pertussis, Haemophilus Influenzae Type b
- Trial name
- NCT00831311
- Testing
- DTaP-IPV-HB-PRP~T, DTaP-IPV//PRP~T combined vaccine & Recombinant hep B vaccine
- Where
- Argentina
- Volunteers
- 624 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Alström Syndrome (ALMS)
Clinical Study of a Single Ciliopathy: Alström Syndrome
The aim of the study is to characterize the clinical manifestations of ALMS within the ciliopathies to prevent complications and determine preventive and therapeutic targets.
- Trial name
- NCT02890550
- Where
- 14 sites in France
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Pompe Disease
Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices
The incidence of type II glycogen-storage disease (Pompe disease) varies depending on ethnicity and geographic region. As of 2010, nine studies have been published documenting the incidence of Pompe disease.
- Trial name
- IPANEMA (NCT02838368)
- Where
- United States
- Volunteers
- 921 people
- Sponsor
- University of California, Irvine
Cerebral Palsy
Individual Patient Expanded Access IND of Hope Biosciences Autologous Adipose-derived Mesenchymal Stem Cells for the Treatment of Cerebral Palsy
This is an Individual Patient Expanded Access Protocol of Autologous HB-adMSCs for the Treatment of Cerebral Palsy (CP) with the primary goal of treating 1 individual with CP who has exhausted all treatment options, his condition…
- Trial name
- NCT04029896
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Charcot-Marie-Tooth Disease
A Registered Cohort Study on Charcot-Marie-Tooth Disease
The aim of the study is to analyze the natural history data data from Charcot-Marie-Tooth disease and related disorders in China, to assess the clinical, genetic, epigenetic features of patients with Charcot-Marie-Tooth disease…
- Trial name
- NCT04010188
- Where
- China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Ning Wang, MD., PhD.
Spasticity, Post-Stroke
JCP Study of Investigation of Patient Background Characteristics
To collect the background characteristics of juvenile cerebral palsy patients aged 2-year or older prescribed BOTOX for the treatment of equines foot due to lower limb spasticity, compare them with those of patients whose deaths…
- Trial name
- NCT01332474
- Testing
- BOTOX
- Volunteers
- 326 people
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05683379
- Testing
- AAV8 DetectCDx
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- REGENXBIO Inc.
Dystonic Cerebral Palsy
Intrathecal Baclofen and Pediatric Dystonia
The goal of this study is to better understand outcomes in children with dystonic cerebral palsy (CP) receiving intrathecal baclofen (ITB), ventral dorsal rhizotomy (VDR), or deep brain stimulation (DBS) as part of standard…
- Trial name
- PREDICT-ITB (NCT06606574)
- Testing
- ITB Titration Protocol, Ventral Dorsal Rhizotomy, Deep Brain Stimulation
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Baylor College of Medicine
Limb-Girdle Muscular Dystrophy, Type 2E
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
- Trial name
- NCT03652259
- Testing
- SRP-9003
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Diabetes Mellitus, Polyneuropathies
Cost-effectiveness of In-shoe Pressure Measurement for Therapeutic Shoes
The purpose of this study is determine whether the use of plantar pressure measurements in the production of customized therapeutic footwear for patients with diabetes and a history of foot ulceration results in lower production…
- Trial name
- NCT02061059
- Testing
- block 1, block 2, standard
- Where
- 2 sites in Netherlands
- Volunteers
- 40 people
- Sponsor
- Maastricht University
Neuroblastoma, Rhabdomyosarcoma
Alpha/Beta T and B Cell Depletion With Zoledronic Acid for Solid Tumors
Hematopoietic stem cell transplantation can cure patients with blood cancer and other underlying diseases. αβ-T cell and B cell depletion has been introduced to decrease GVHD and PTLD and has demonstrated effectiveness for…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Synovial Sarcoma, Peripheral Nerve Sheath Tumors
- Trial name
- NCT06625190
- Testing
- Miltenyi CliniMACS Prodigy ® system, Zoledronic acid
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Florida
Neurofibromatosis 1, Social Skills
Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1
This is a pilot randomized control trial of the UCLA PEERS protocol delivered via Telehealth with teens with neurofibromatosis type 1 whose parents report that they have difficulty making and keeping friends.
- Trial name
- NCT04860362
- Testing
- PEERS
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Milwaukee
Neurofibroma, Plexiform, Neurofibromatosis Type I
R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas
This study will examine whether the experimental drug R115777 (Tipifarnib) can shrink or slow the growth of plexiform neurofibromas in children and young adults with neurofibromatosis type 1 (NF1) and determine what side effects…
- Trial name
- NCT00021541
- Testing
- tipifarnib
- Where
- 12 sites in United States, Germany
- Volunteers
- 62 people
- Sponsor
- National Cancer Institute (NCI)
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