Clinical trials: Inherited Neuromuscular Conditions
Neurofibromatosis Type 1, Reading Disabilities
Neurobiology and Treatment of Reading Disability in NF-1
The goal of this trial is to determine if children with neurofibromatosis type 1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs as children with…
- Trial name
- NCT00624234
- Testing
- Tutoring Program I, Tutoring Program II
- Where
- United States
- Volunteers
- 184 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
Neurodegenerative Diseases, Parkinson Disease
Neurodegenerative Diseases Progression Markers (MARKERS-NDD)
MARKERS-NDD is a prospective, observational, longitudinal study, which aims to collect data from patients affected by neurodegenerative diseases (NDD) followed longitudinally for routine examinations performed as part of normal…
- Conditions
- Neurodegenerative Diseases, Parkinson Disease, Synucleinopathies, Lewy Body Disease
- Trial name
- MARKERS-NDD (NCT06596746)
- Testing
- Observation
- Where
- Italy
- Volunteers
- 600 people
- Sponsor
- Casa di Cura San Raffaele Cassino
Chronic Inflammatory Demyelinating Polyneuropathy, Peripheral Neuropathy
SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
The single-center, open-label Phase II study has the objective of assess the effect of MD1003 on motor and sensory conduction in patients suffering from demyelinating polyneuropathies in 15 subjects.
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy, Peripheral Neuropathy, Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A
- Trial name
- NCT02967679
- Testing
- MD1003
- Where
- France
- Volunteers
- 15 people
- Sponsor
- MedDay Pharmaceuticals SA
Sarcopenia, Hemophilia
Sarcopenia and Osteoporosis in the Patients With Hemophilia
Hemophilia is a bleeding disorder and repeated joint bleeding leads to hemophilic arthropathy. Among patients with hemophilia, vitamin D deficiency and hemophilic arthropathy have been associated with osteoporosis in several…
- Conditions
- Sarcopenia, Hemophilia, Osteoporosis, Osteopenia, Hemophilic Arthropathy
- Trial name
- NCT04535180
- Testing
- Dual-energy X-ray absorptiometry (DXA)
- Where
- Taiwan
- Volunteers
- 45 people
- Sponsor
- Tri-Service General Hospital (TSGH)
Irritable Bowel Syndrome, Digestive System Diseases
Efficacy of a Multi-strain Probiotic in the Treatment of Irritable Bowel Syndrome
The Canadian College of Naturopathic Medicine is conducting a research study on Irritable Bowel Syndrome (IBS), a common condition in North America.
- Conditions
- Irritable Bowel Syndrome, Digestive System Diseases, Colonic Diseases, Functional, Colitis, Mucous
- Trial name
- IBS (NCT01887834)
- Testing
- Kyodophilus multi-strain probiotic capsules
- Where
- Canada
- Volunteers
- 100 people
- Sponsor
- The Canadian College of Naturopathic Medicine
Breast Cancer
Phase II Trial of SAHA & Tamoxifen for Patients With Breast Cancer
Phase II trial to explore the efficacy of vorinostat and tamoxifen combined.
- Trial name
- NCT00365599
- Testing
- suberoylanilide hydroxamic acid (SAHA, Vorinostat), tamoxifen citrate (Tamoxifen)
- Where
- 8 sites in United States
- Volunteers
- 43 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
Other Diseases or Conditions
Mother-caregivers of Children With Duchenne Muscular Dystrophy
The incidence of Duchenne Muscular Dystrophy (DMD) is approximately 1 in 3.500 male newborns. During its progression there is loss of mobility, swallowing difficulties and a significant reduction in respiratory capacity.
- Trial name
- DMDK (NCT01921374)
- Testing
- sleep parameters, Hormonal profile, Inflammatory profile
- Where
- Brazil
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Monica Levy Andersen
Cerebral Palsy, Spinal Muscular Atrophy
Pediatric Powered Wheelchair Standing Devices: An Exploratory Study
For children who use a power wheelchair, a powered wheelchair standing device (PWSD) may be considered for daily use. A PWSD allows a child to electronically move between sitting and standing and can be driven in either position.
- Conditions
- Cerebral Palsy, Spinal Muscular Atrophy, Spinal Cord Injuries, Spina Bifida
- Trial name
- NCT05117827
- Testing
- Power Wheelchair Standing Device
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Grand Valley State University
Post-acute Infectious Syndrome (PAIS), Post-COVID 19 Condition (PCC or Long COVID)
CD19-B Cell Depletion in PAIS-ME/CFS Patients
Post-acute infection syndromes (PAIS) are long-lasting health problems that can develop after an infection. They include post-COVID-19 syndrome and similar illnesses following other infections.
- Conditions
- Post-acute Infectious Syndrome (PAIS), Post-COVID 19 Condition (PCC or Long COVID), Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS)
- Trial name
- PIONEER_PAIS (NCT07724834)
- Testing
- Inebilizumab
- Where
- Germany
- Volunteers
- 38 people
- Sponsor
- Charite University, Berlin, Germany
Colorectal Cancer Metastatic
Influence of BRAF and PIK3K Status on the Efficacy of 5-Fluorouracil/Leucovorin/Oxaliplatin (FOLFIRI) Plus Bevacizumab or Cetuximab in Patients With RAS Wild-type Metastatic Colorectal Carcinoma and < 3 Circulating Tumor Cells (CTC)
The purpose of the study is to explore the influence of BRAF and PIK3K status on the efficacy of FOLFIRI plus Bevacizumab or Cetuximab, as first line therapy of patients with RAS wild-type metastatic colorectal carcinoma and < 3…
- Trial name
- VISNU-2 (NCT01640444)
- Testing
- FOLFIRI + bevacizumab, FOLFIRI + cetuximab
- Where
- Spain
- Volunteers
- 240 people
- Sponsor
- Spanish Cooperative Group for the Treatment of Digestive Tumours (TTD)
Osteoporosis, Osteoporotic Fractures
Multifaceted Intervention Using Telehealth to Reduce the Risk of Falls and Fractures in Older Men
Men sustain over one-third of osteoporosis-related fractures worldwide. The burden of osteoporotic fractures in older men is substantial, and men suffer significantly worse fracture-related outcomes than women.
- Conditions
- Osteoporosis, Osteoporotic Fractures, Osteoporosis, Age-Related, Muscle Loss
- Trial name
- MisterFIt (NCT05927623)
- Testing
- Multifaceted Virtual Fracture Prevention Program, Attention Control
- Where
- 5 sites in Canada
- Volunteers
- 45 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Aneurysms, Arteriovenous Malformations
Neurosurgical Outcome Network
The evaluation of neurosurgical outcomes varies from center to center, and the predictive factors that determine these outcomes are not fully known or shared.
- Conditions
- Aneurysms, Arteriovenous Malformations, Cavernomas, Skull Base Tumors
- Trial name
- NEON (NCT06724029)
- Where
- 27 sites in Italy
- Volunteers
- 4,500 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Nausea, Vomiting
The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
This is a pilot clinical trial of dronabinol to treat disabling attacks of nausea and vomiting in patients with familial dysautonomia (FD, also known as Riley Day syndrome or hereditary sensory and autonomic neuropathy type III).
- Conditions
- Nausea, Vomiting, Familial Dysautonomia
- Trial name
- NCT02608931
- Testing
- Dronabinol
- Where
- United States
- Sponsor
- NYU Langone Health
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
CIDP (Chronic Inflammatory Demyelinating Polyradiculoneuropathy), CMT (Charcot Marie Tooth Disease)
Diffusion Tensor Imaging in Chronic Inflammatory Demyelinating Polyneuropathy (PIDC)
The main purpose of this study is to assess the clinical feasibility of diffusion tensor imaging (DTI) for the diagnosis of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).
- Trial name
- PIDC (NCT03460951)
- Testing
- cervical MRI
- Where
- France
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
Hereditary Spastic Paraplegia, SPG47
Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia
The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biological samples (skin and/or blood and/or saliva) from male and female…
- Conditions
- Hereditary Spastic Paraplegia, SPG47, SPG50, SPG51
- Trial name
- HSP (NCT04712812)
- Where
- United States
- Volunteers
- 700 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Neonatal Brachial Plexus Palsy
Forced Lefthandedness in Neonatal Brachial Plexus Palsy (NBPP) Children
The aim of the project is to assess the neurophysiological and functional consequences of forced lefthandedness in a population with right neonatal brachial plexus palsy (NBPP).
- Trial name
- NBPP (NCT04376372)
- Testing
- Functional testing, Magnetic resonance imaging, Electromyography
- Where
- Belgium
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- University Hospital, Ghent
Myotonic Dystrophy, Sudden Cardiac Death
RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy
This is a prospective multicentric Italian study to evaluate the arrhythmic risk in myotonic dystrophy type 1.
- Trial name
- NCT00127582
- Testing
- Electrophysiological study, pacemaker , implantable cardiac defibrillator or loop-recorder implant
- Where
- Italy
- Volunteers
- 537 people
- Sponsor
- Catholic University of the Sacred Heart
Pompe Disease, Muscle Weakness
Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.
The purpose of this research study is to determine if exercise will help improve muscle strength, endurance, and quality of life in individuals with Pompe disease.
- Trial name
- ExercisPompe (NCT02654886)
- Testing
- Resistance Exercise Training
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, Irvine
Circulating Tumor Cell, Metastatic Prostate Cancer
Cabazitaxel in mCRPC Patients With AR-V7 Positive Circulating Tumor Cells (CTCs)
After failure on docetaxel, which has been the standard first line therapy for patients with metastatic castration-resistant prostate cancer (mCRPC), several treatment options are currently available.
- Trial name
- CABA-V7 (NCT03050866)
- Testing
- Cabazitaxel, Antihistamine, Corticosteroid
- Where
- 9 sites in Netherlands
- Volunteers
- 140 people
- Sponsor
- Erasmus Medical Center