Clinical trials: Neuromuscular Disorders
Myotonic Dystrophy 1, DM1
The Spanish National Registry for Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1) is a rare genetic neuromuscular condition that can affect multiple organs and varies widely in how it presents.
- Conditions
- Myotonic Dystrophy 1, DM1, Myotonic Dystrophy Type 1, Myotonic Dystrophy, Congenital
- Trial name
- DM1-Hub (NCT07385443)
- Testing
- Patient Registry
- Where
- 8 sites in Spain
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Fundació Institut Germans Trias i Pujol
Neurofibromatosis 1 and 2 (NF1 and NF2)
A Single Center Pilot Study to Assess the Intra-observer Reliability of Measuring Muscle Strength Using a Hand Held Dynamometer in Children and Adults With Neurofibromatosis Type 1 (NF1) and Type 2 (NF2)
Patients with clinically confirmed neurofibromatosis type 1 (NF1) or neurofibromatosis type 2 (NF2) or a known neurofibromatosis (NF) mutation aged 5 years and above will be eligible to participate and will be recruited from the…
- Trial name
- NCT03672838
- Testing
- hand-held dynamometer
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- NYU Langone Health
Thyroid Diseases, Cognition Disorders
Mohawk Culture, Behavior, Toxicant Exposure and Health
This study will determine the pathways between Mohawk cultural identification and specific behaviors related to pollutant exposure, and determine the effects of these factors and the pollutant exposure on physiologically and…
- Trial name
- NCT00043238
- Where
- United States
- Volunteers
- 220 people
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
Duchenne Muscular Dystrophy
Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
Researchers at Children's Hospital Boston Neurology Department invite children to participate in a new research study. Researchers are looking for boys ages 2 - 30 with Duchenne Muscular Dystrophy (DMD) and healthy boys ages 2 …
- Trial name
- QED (NCT01491555)
- Where
- United States
- Volunteers
- 73 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Neuromuscular Diseases
Reliability Study of a Motor Function Measure Digitalized Playful Completion Modules
Neuromuscular diseases include more than 200 rare disorders affecting muscles or the nervous system. Functional scales assessing motor function are tools allowing to measure the evolution of motor impairment of patients with a…
- Trial name
- MFM-Play (NCT05227274)
- Testing
- Items completion with or without a MFM-Play
- Where
- 10 sites in France
- Volunteers
- 70 people
- Sponsor
- Hospices Civils de Lyon
Neuropathic Pain
Neuropathic Pain Database
To establish the infrastructure for a national neuropathic pain database. To determine the longterm outcome of the management of chronic neuropathic pain including pain relief, disability, and quality of life.
- Trial name
- NePDatabase (NCT00669006)
- Where
- Canada
- Volunteers
- 789 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Myasthenia Gravis, CIDP - Chronic Inflammatory Demyelinating Polyneuropathy
A Worldwide Pregnancy Safety Study to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to Efgartigimod During Pregnancy and/or Breastfeeding.
This is a multi-country, prospective safety study of pregnant women exposed to efgartigimod or efgartigimod PH20 SC any time within 25 days prior to conception or any time during pregnancy.
- Trial name
- NCT06299748
- Testing
- Efgartigimod
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 279 people
- Sponsor
- argenx
Diphtheria, Tetanus
Assessment of the Immunogenicity and Safety of PENTAXIM™ in Philippines
The present clinical study will assess the immunogenicity and reactogenicity of the subsequent administration of Aventis Pasteur's DTacP-IPV//PRP~T combined vaccine (PENTAVAC™/PENTAXIM), as a three-dose primary vaccination in 6…
- Conditions
- Diphtheria, Tetanus, Polio, Pertussis
- Trial name
- NCT00254917
- Testing
- Diphteria, Tetanus, Polio, Acellular Pertussis, Hib vaccine
- Where
- Philippines
- Volunteers
- 387 people, healthy volunteers welcome
- Sponsor
- Sanofi
Spasticity/Paresis, Cerebral Palsy
Efficacy of Extracorporeal Shock Wave Therapy in Children With Cerebral Palsy
Spastic plantar and palmar flexion deformities are very common in children with cerebral palsy (CP). These deformities usually involve spasticity of the plantar or palmar muscle complexes, weakness of the antagonist dorsiflexor…
- Trial name
- NCT06128616
- Testing
- ESWT, Botulinum Toxin Type A, Serial Casting
- Volunteers
- 40 people
- Sponsor
- Kocaeli University
Peroneal Muscular Atrophy
Genetics of Charcot-Marie-Tooth Dystrophy and Related Diseases
This is a cross-sectional study to clarify the gene lineage distribution of CMT genes in CMT patients in my country, draw a frequency map of CMT gene distribution, and assist in determining the genetic diagnosis strategy of CMT…
- Trial name
- NCT04967716
- Testing
- basic information, venous blood test
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Peking University Third Hospital
Coccyx Disorder
Analgesic Efficacy of Capsaicin 8% Patch (QUTENZA®) in Coccygodygnia
A retrospective study over a period of 6 years to explore analgesic efficacy of 8% Capsaicin patch (QUTENZA®) in coccygodynic patients, as well as neuropathic predictive factors of therapeutic response.
- Trial name
- CAPSACOXp (NCT05983263)
- Testing
- Capsaicin
- Where
- France
- Volunteers
- 106 people
- Sponsor
- Nantes University Hospital
Familial Dysautonomia
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
The purpose of this placebo controlled interventional study is to collect preliminary data on administering dexmedetomidine in patients with Familial Dysautonomia (FD) during a rapid cessation of autonomic crisis.
- Trial name
- NCT06148311
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Carpal Tunnel Syndrome
Median Nerve Strain and Applied Pressure Measurement in the Carpal Tunnel Syndrome Patients
The aim of this study is to investigate diagnostic accuracy of median nerve strain and applied pressure measurement . Idiopathic carpal tunnel syndrome patients and healthy controls will be evaluated by ultrasound.
- Trial name
- NCT04027998
- Testing
- strain and applied pressure measurement
- Where
- Japan
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Tokyo Medical University
Cerebral Palsy, Spina Bifida
The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities
Youth with physical disabilities face greater restrictions to participation in community-based activities than their typically developing peers, which can lead to poor health outcomes.
- Conditions
- Cerebral Palsy, Spina Bifida, Muscular Dystrophies, Musculoskeletal Disorder
- Trial name
- NCT03851107
- Testing
- Engagement in 6-week community-based activity program
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- McGill University
Duchenne Muscular Dystrophy
Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)
The aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid…
- Trial name
- NCT01125709
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 30 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Facioscapulohumeral
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
The goal of this observational study is to learn the patient journey, disease burden, living situation, quality of life and etc. in patients with Facioscapulohumeral Muscular Dystrophy in all sex/gender and age groups.
- Trial name
- NCT06517498
- Testing
- No Intervention
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Xi'an Jiaotong University
Spinal Muscular Atrophy
Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
This global, retrospective, non-interventional, medical chart review (MCR), descriptive study collected patient-level data in regions outside the US.
- Trial name
- NCT06178393
- Where
- United States
- Volunteers
- 162 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Dystrophy
Effect of Aquatic Therapy on Pulmonary Functions in Patients With Muscular Dystrophy
This study aimed to assess the efficacy of aquatic therapy on pulmonary functions in patients with muscular dystrophy.
- Trial name
- NCT04574934
- Testing
- aquatic therapy
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- South Valley University
Attention Deficit Hyperactivity Disorder, ADHD
Imaging Stimulant and Non Stimulant Treatments for ADHD: A Network Based Approach
The growing number of medications used to treat attention-deficit/hyperactivity disorder (ADHD) raises important questions about whether different medications have similar or different therapeutic mechanisms of action.
- Trial name
- NCT01678209
- Testing
- fMRI scans, Atomoxetine arm, Methylphenidate arm
- Where
- United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Icahn School of Medicine at Mount Sinai
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 2 sites in New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.