Clinical trials: Spinal Deformities
Chiari Malformation, Type 1
Identification of Genetic Mutations Involved in Chiari Type I Malformations
Although most cases of Chiari malformation type I (CM1) are sporadic, familial cases of CM1, with or without syringomyelia, suggest a genetic cause in the pathogenesis of these malformations.
- Trial name
- ChiariGene (NCT05165030)
- Testing
- Blood Sample
- Where
- France
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile
Physical Activity, Motor Competence, Pulmonary Function, and Quality of Life in Children With Severe Spinal Disease
Observation study about physical activity, motor competence, pulmonary function, and health related quality of life in children, surgically treated for early onset scoliosis
- Conditions
- Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile, Scoliosis Neuromuscular, Scoliosis in Neurofibromatosis
- Trial name
- NCT03494829
- Where
- Norway
- Volunteers
- 50 people
- Sponsor
- Oslo University Hospital
Spina Bifida
Establishing the Preliminary Utility of a Novel Pediatric Manual Mobile Standing Wheelchair
To establish the preliminary utility of a novel pediatric manual mobile standing wheelchair (PedMMSWC).
- Trial name
- NCT07290556
- Testing
- PedMMSWC Wheelchair User Testing, PedMMSWC Simulated Home, School, Community Environment
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Optimal Mobility, Inc
Metastatic Disease, Spinal Cord Injury
Negative Pressure Wound Therapy vs. Sterile Dressing for Patients Undergoing Thoracolumbar Spine Surgery
The purpose of this study is to determine if the use of the Prevena™ System decreases the rate of subcutaneous seroma, superficial wound dehiscence and infection.
- Conditions
- Metastatic Disease, Spinal Cord Injury, Spinal Degeneration, Spinal Deformity
- Trial name
- NCT03632005
- Testing
- Vacuum Assisted Closure, Sterile dressing
- Where
- Canada
- Volunteers
- 550 people
- Sponsor
- John Street
Friedreich Ataxia
A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia
To test the variability of specific ribonucleic acid (RNA) and proteins as well as frataxin levels in samples of blood and buccal cells taken directly from patients with Friedreich's ataxia (FRDA) in order to confirm potential…
- Trial name
- NCT04255680
- Testing
- Buccal Swabs and Blood Draws
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Larimar Therapeutics, Inc.
Friedreich Ataxia
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with…
- Trial name
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- IRCCS Eugenio Medea
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Body Weight Support Harness System in Spinal Muscular Atrophy
The goal of this pilot interventional study is to learn about the use of an in-home harness system in children who have been treated for spinal muscular atrophy. The main questions it aims to answer are: 1.
- Trial name
- NCT05715749
- Testing
- In-home body weight support harness system
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Nationwide Children's Hospital
Cervical Radiculopathy, Cervical Myelomalacia
Cervical Spinal Cord Signal Abnormalities in Patients With Cervical Radicular Pain
This prospective observational study aims to determine the prevalence of cervical spinal cord signal abnormalities, including myelomalacia, among adult patients presenting to a tertiary pain clinic with cervical radicular pain.
- Trial name
- CSCS (NCT07823426)
- Testing
- Clinical and Cervical MRI Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 400 people
- Sponsor
- Ankara City Hospital Bilkent
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
Duchenne Muscular Dystrophy, Spinal Muscular Atrophy
Optimum Insufflation Capacity in NMD
Patients with underlying neuromuscular disorder (NMD) often suffer from weakness in the inspiratory and expiratory muscles. Consequently they do not have the strength to generate the minimum flow of 160 to 300 liters/minute for…
- Conditions
- Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Chronic Respiratory Insufficiency
- Trial name
- NCT01981915
- Testing
- IPPB, LIAM
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Universität Duisburg-Essen
Friedreich Ataxia
Characterization of the Interruptions of the GAA Expansion and Study of Their Influence on the Severity of Friedreich's Ataxia
Friedreich's ataxia (FA) is the most frequent recessive genetic ataxia with an estimated prevalence of 1/50 000. The first symptoms appear around the age of 10 years with a progressive course and the need for an armchair 10- 15…
- Trial name
- INTREP-AF (NCT04346238)
- Testing
- Reuse of existing data from patients' medical records
- Where
- France
- Volunteers
- 120 people
- Sponsor
- University Hospital, Montpellier
Tethered Cord, Tethered Cord Syndrome
Investigation of Surgical Sectioning of the Filum Terminale in Treating Occult Tethered Cord Syndrome Patients
The investigators hypothesize that surgical release of the filum terminale (strand of fibrous tissue at the end of the spinal cord) is a more efficacious treatment option for symptomatic relief than medical management in subjects…
- Conditions
- Tethered Cord, Tethered Cord Syndrome, Occult Spina Bifida, Spina Bifida Occulta
- Trial name
- OCCULT (NCT05163899)
- Testing
- Release of filum terminale
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Weill Medical College of Cornell University
Spinal Muscular Atrophy Type I
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy type I; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE 02 (NCT00439218)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Westat
Friedreich's Ataxia
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
- Trial name
- PROVE-FA (NCT07681713)
- Testing
- Vatiquinone
- Where
- 9 sites in United States, Belgium and 4 more
- Volunteers
- 120 people
- Sponsor
- PTC Therapeutics
Friedreich Ataxia
Idebenone to Treat Friedreich's Ataxia
This study will determine whether a drug called idebenone is safe and effective in reducing the level of oxidants that are believed to damage the nervous system and hearts in patients with Friedreich's ataxia.
- Trial name
- NCT00229632
- Testing
- Idebenone
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Spine Deformity
Perioperative Nutritional Status and Nutritional Support in Patients With Spinal Deformity
To investigate the nutritional status, nutritional support and postoperative complications of patients undergoing spinal orthopedic surgery during the perioperative period.
- Trial name
- NCT04968483
- Testing
- Data collection
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Peking University Third Hospital
Neurogenic Bladder Disorder, Spina Bifida
Complications of Bladder Augmentation in Children
The goal of this observational study is to describe the complications in participants who underwent enterocystoplasty in childhood.
- Conditions
- Neurogenic Bladder Disorder, Spina Bifida, Complications of Surgical Procedures or Medical Care, Child, Only
- Trial name
- AVPED (NCT06733194)
- Where
- France
- Volunteers
- 87 people
- Sponsor
- University Hospital, Clermont-Ferrand
Friedreich's Ataxia
A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
- Trial name
- NCT00530127
- Testing
- deferiprone
- Where
- 6 sites in Australia, Belgium and 4 more
- Volunteers
- 80 people
- Sponsor
- ApoPharma
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy
Prospective Evaluation of Infants With Spinal Muscular Atrophy:
SPOT SMA is a prospective NIH-supported clinical study targeting pre-symptomatic or recently diagnosed infants and children with Spinal Muscular Atrophy (SMA) types 1, 2, or 3 and their healthy control siblings less than 36…
- Trial name
- SPOTSMA (NCT02831296)
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital