Clinical trials: Spinal Deformities
Spina Bifida, Cerebral Palsy
Grand Valley State University (GVSU) Skills on Wheels
Manual wheelchairs (MWCs) are widely used by children with physical disabilities, yet many of these children are unable to use their wheelchair independently. Instead, they depend on others to push them.
- Conditions
- Spina Bifida, Cerebral Palsy, Genetic Disease, Spinal Cord Injuries
- Trial name
- NCT05339932
- Testing
- Manual Wheelchair Skills Training
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Grand Valley State University
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Friedreich Ataxia, Friedreich's Ataxia
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
- Trial name
- FORWARD-FA (NCT07778836)
- Testing
- Nomlabofusp
- Where
- 4 sites in United States
- Volunteers
- 150 people
- Sponsor
- Larimar Therapeutics, Inc.
Friedreich Ataxia
Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients
Friedreich's Ataxia (FA) is an autosomal recessive disease the mutation of which leads to a deficiency of a protein called frataxin, which is responsible for the symptoms of the disease.
- Trial name
- CalcitriolFA (NCT04801303)
- Testing
- Calcitriol, Blood analysis for frataxin's level measurement, Blood analysis for hypercalcemia's control
- Where
- Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Berta Alemany
Spina Bifida
A Collaboration Between the EAU-ESPU Pediatric Urology Guidelines and the IFSBH
Children with spina bifida require care from various medical and paramedical specialists throughout their life. The availability and organization of the care needed differs between the European countries.
- Trial name
- NCT06725745
- Testing
- Survey
- Where
- Netherlands
- Volunteers
- 281 people
- Sponsor
- Erasmus Medical Center
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
Cardiac Output and Fatigue in Friedreich's Ataxia
This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will…
- Trial name
- NCT07444333
- Testing
- Aerobic Exercise
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Scott Barbuto
Calculi, Urinary, Neurogenic Bladder Disorder
Urinary Calculi After Bladder Augmentation in Children
The goal of this observational study is to analyse the occurence of urinary calculi after bladder augmentation in children: location of the calculi (kidney or bladder) type of bladder augmentation with higher rate of urinary…
- Conditions
- Calculi, Urinary, Neurogenic Bladder Disorder, Spina Bifida, Child, Only
- Trial name
- LITAPED (NCT06734403)
- Testing
- Bladder Augmentation
- Where
- France
- Volunteers
- 34 people
- Sponsor
- University Hospital, Clermont-Ferrand
Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis)
Invasive Home Ventilation in Denmark
The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark.
- Conditions
- Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis), Spinal Cord Injuries (SCI), Duchenne Muscular Dystrophy (DMD)
- Trial name
- HOMEVENT DK (NCT07467187)
- Where
- Denmark
- Volunteers
- 450 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Chiari Malformations
Dynamic MRI and Quantitative MR CSF Flow Studies in Craniovertebral Junction Anomalies
The purpose of this study is to decide which position of the neck affects cerebral spinal fluid (CSF) flow between the spinal cord and the brain.
- Trial name
- NCT00795080
- Testing
- Dynamic cervical MRI scans
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Scoliosis
Prospective Multicenter Evaluation of a New Predictive Model for the Progression of Adolescent Idiopathic Scoliosis
Scoliosis is a three-dimensional deformity affecting the orientation and position of the spine. Locally, the shape of the vertebra is also affected.
- Trial name
- NCT02434003
- Where
- 9 sites in United States, Canada and 4 more
- Volunteers
- 1,200 people
- Sponsor
- St. Justine's Hospital
Cerebral Palsy, Spina Bifida
The Impact of Participation on Body Functions Among Youth With Physical Disabilities
Youth with physical disabilities experience greater limitations to participation in community- based activities than do their average-developing peers, which can result in poor health outcomes.
- Conditions
- Cerebral Palsy, Spina Bifida, Musculoskeletal Disorders
- Trial name
- NCT04288453
- Testing
- Engagement in a 8-week community-based activity program
- Where
- Canada
- Volunteers
- 21 people
- Sponsor
- McGill University
Duchenne's Muscular Dystrophy, Scoliosis
Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy
Nearly all patients with Duchenne's Muscular Dystrophy (DMD) have scoliosis. Posterior instrumented spinal fusion, which is a surgery to correct scoliosis, has been shown to improve quality of life and satisfaction of both…
- Trial name
- NCT00207857
- Where
- United States
- Sponsor
- Children's Healthcare of Atlanta
Crohn's Disease, Rheumatoid Arthritis
OTIS Cimzia Pregnancy Registry
The purpose of the OTIS Autoimmune Diseases in Pregnancy Study is to monitor planned and unplanned pregnancies exposed to certain medications, to evaluate the possible teratogenic effect of these medications and to follow live…
- Conditions
- Crohn's Disease, Rheumatoid Arthritis, Ankylosing Spondylitis, Psoriatic Arthritis
- Trial name
- NCT01797224
- Where
- United States
- Volunteers
- 925 people, healthy volunteers welcome
- Sponsor
- University of California, San Diego
Friedreich Ataxia
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA).
- Trial name
- PROFA (NCT05943002)
- Where
- 6 sites in Austria, France and 1 more
- Volunteers
- 200 people
- Sponsor
- German Center for Neurodegenerative Diseases (DZNE)
Inflammatory Bowel Diseases, Anorectal Malformations
StoMakker Mobile Application
The goal of this clinical trial is to investigate if access to the StoMakker application will significantly improve health-related quality of life in children receiving surgery resulting in an ileostomy, colostomy or continent…
- Conditions
- Inflammatory Bowel Diseases, Anorectal Malformations, Spina Bifida, Ostomy
- Trial name
- StoMakker (NCT06065995)
- Testing
- StoMakker
- Where
- Netherlands
- Volunteers
- 30 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
Duchenne Muscular Dystrophy, Duchenne Disease
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
This pilot study will investigate the potential efficacy of VM100, a nutritional supplement specifically formulated for patients with DMD, on quality of life and physical symptoms.
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD
- Trial name
- NCT07766980
- Testing
- VM100 Nutritional Supplement
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Florida
Myelomeningocele, Spina Bifida
Transcutaneous Spinal Stimulation for Children and Youth With Spina Bifida
A study to use transcutaneous spinal cord stimulation to characterize sensorimotor deficits in a pediatric population of individuals with spina bifida.
- Trial name
- NCT06918119
- Testing
- Transcutaneous stimulation
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Mayo Clinic
Neurofibromatosis 2, Vestibular Schwannoma
Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
The purpose of this study is to determine if Lapatinib has any effect on tumors found in patients with Neurofibromatosis Type 2 (NF2). NF2 is a condition that mainly affects the skin and nervous system.
- Trial name
- NCT00973739
- Testing
- Lapatinib
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- NYU Langone Health