Clinical trials: Spinal Deformity
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Friedreich Ataxia
Micronised Resveratrol as a Treatment for Friedreich Ataxia
The aim of this study is to assess the efficacy of micronised resveratrol as a treatment for FRDA, in terms of reducing the severity of ataxia symptoms at 24 weeks, through a randomised blinded, placebo controlled crossover trial.
- Trial name
- NCT03933163
- Testing
- Resveratrol
- Where
- 4 sites in Australia
- Volunteers
- 25 people
- Sponsor
- Murdoch Childrens Research Institute
Juvenile and Adolescent Idiopathic Scoliosis
3-D Correction of Adolescent Idiopathic Scoliosis (AIS) With Differential Metals
This study is designed to evaluate the coronal, sagittal, and axial correction of deformity secondary to adolescent idiopathic scoliosis utilizing a technique employing a posterior spinal fusion construct utilizing rods of…
- Trial name
- AIS (NCT05631821)
- Testing
- differential rod bending with rod contours (MESA Spinal System)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Rush University Medical Center
Scoliosis, Kyphosis
Postoperative and Chronic Pain Genetic Spine and Pectus Surgery Study
This will be an open label, prospective study to determine the association between specific genotypes, epigenetics and behavioral factors, with the phenotypes, defined by pain perception, postoperative pain, analgesic and side…
- Conditions
- Scoliosis, Kyphosis, Kyphoscoliosis, Pectus Excavatum
- Trial name
- NCT02998138
- Where
- 5 sites in United States
- Volunteers
- 880 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic 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
Spinal Cord Injuries, Cerebral Palsy
Mobile Health Self-Management and Support System for Chronic and Complex Health Conditions
This study will assess the benefits of using mobile health system designed for individuals with chronic and complex health conditions (such as those with Spinal Cord Injury,Cerebral Palsy, Spina Bifida, and Traumatic Brain…
- Conditions
- Spinal Cord Injuries, Cerebral Palsy, Spinal Dysraphism, Brain Injuries
- Trial name
- NCT02592291
- Testing
- MHealth
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Spinal Muscular Atrophy Type I, Scoliosis
The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a serious neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive proximal muscle atrophy and denervation.
- Conditions
- Spinal Muscular Atrophy Type I, Scoliosis, Chest Deformities, Spinal Orthosis
- Trial name
- NCT05878418
- Testing
- Thoracolumbosacral Spinal Orthosis (TLSO), Trunk Exercise (TE), Pulmonary Rehabilitation(PR) and Pulmonary Care (PC)
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Medipol University
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
Spina Bifida, Neurogenic Bowel
Bowel Continence Across the Lifespan in People With Spina Bifida
The goal of this observational study is to learn how different enema programs affect bowel control in children and adults with spina bifida.
- Conditions
- Spina Bifida, Neurogenic Bowel, Bowel Incontinence
- Trial name
- BCALS (NCT07390318)
- Testing
- Retrograde Enema, Antegrade Enema
- Where
- 24 sites in United States
- Volunteers
- 943 people
- Sponsor
- David Chu
Friedreich's Ataxia
(+) Epicatechin to Treat Friedreich's Ataxia
This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.
- Trial name
- NCT02660112
- Testing
- (+)-Epicatechin
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Ralitza Gavrilova
Psoriasis, Pregnancy
Stelara and Tremfya Pregnancy Exposure Registry OTIS Autoimmune Diseases in Pregnancy Project
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
- Psoriasis, Pregnancy, Psoriatic Arthritis, Crohn Disease
- Trial name
- NCT02103361
- Testing
- Ustekinumab, Guselkumab
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- University of California, San Diego
SMA II
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
- Trial name
- NCT05901987
- Testing
- GC101
- Where
- 5 sites in China
- Volunteers
- 33 people
- Sponsor
- GeneCradle Inc
Ataxia, Spinocerebellar, Ataxias, Hereditary
Functional Trunk Training in Ataxia Patients
The study is to examine the effect of functional trunk training on trunk control and upper extremity functions in patients with autosomal recessive ataxia.
- Trial name
- NCT04740359
- Testing
- Functional training, Routine training
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Hasan Kalyoncu University
Spina Bifida, Neurogenic Bladder
CIC Behavioral Economics in Children With Spina Bifida
The purpose of the study is to develop a comprehensive program to increase early initiation of self-catheterization in children with Spina Bifida. This will be a prospective open label randomized control trial.
- Trial name
- NCT05562713
- Testing
- Present Bias without Loss Aversion, Present Bias with Loss Aversion
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Duke University
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
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.
Spinal Muscular Atrophy 1
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
- Trial name
- NCT06191354
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 12 people
- Sponsor
- Kun Sun
Friedreich's Ataxia
GV101 for Friedreich's Ataxia (FA) Treatment
This is a dose-ranging, randomized, double-blind, placebo-controlled 12-week Phase 2a trial with an up to two week loading dose period.
- Trial name
- NCT07870590
- Testing
- GV101
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Graviton Bioscience Corporation
Neurofibromatosis
Natural History Study of Patients With Neurofibromatosis Type 2
Objective With this prospective natural experiment trial on neurofibromatosis type 2 (NF2) study, we hope to understand the factors leading to tumor progression and neurological disease burden in NF2.
- Trial name
- NCT00598351
- Where
- United States
- Volunteers
- 269 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)