Clinical trials: Spinal Deformities, open to volunteers
Spina Bifida, Fecal Incontinence
Enema Device for Children With Spina Bifida
The International Center for Colorectal and Urogenital Care at Children's Hospital Colorado treats patients who suffer from many different colorectal issues such as anorectal malformation, Hirschsprung disease, and severe…
- Trial name
- NCT05253196
- Testing
- Use of the foley catheter assistive device
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Colorado, Denver
Postural Kyphosis, Spine
Reliability of PHAS in Postural Hyperkyphosis
This observational, cross-sectional study aims to evaluate the reliability of the Postural Habits and Awareness Scale (PHAS) in patients with postural hyperkyphosis.
- Conditions
- Postural Kyphosis, Spine, Assessment, Self
- Trial name
- NCT06907901
- Testing
- PHAS Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 128 people
- Sponsor
- Istanbul University - Cerrahpasa
Gait Impairment, Cerebral Palsy (CP)
Clinical Investigation to Validate the Safety and Performance of the ABLE Kids Device in Pediatric Patients With Neurological Gait Disorders in a Clinical Setting
The primary objective of the study is to validate the safety and clinical performance of the ABLE Kids device in pediatric patients with neurological gait disorders or muscular weakness during a 10-session gait training program…
- Conditions
- Gait Impairment, Cerebral Palsy (CP), Neuromuscular Diseases (NMD), Spinal Cord Injuries (SCI)
- Trial name
- NCT07745712
- Testing
- ABLE Kids
- Where
- Spain
- Volunteers
- 15 people
- Sponsor
- ABLE Human Motion S.L.
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
Spina Bifida
QUALAS Validation in Dutch
Rationale Patients with spina bifida often experience a decreased quality of life compared to patients without spina bifida. The Quality of Life Assessment for Spina Bifida (QUALAS) is a validated questionnaire to assess the…
- Trial name
- NCT06723951
- Testing
- QUALAS survey
- Where
- 2 sites in Netherlands
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Erasmus Medical Center
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
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
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)
Spinal Muscular Atrophy Type 3
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
- Trial name
- NCT06421831
- Testing
- GC101
- Where
- China
- Volunteers
- 21 people
- Sponsor
- GeneCradle Inc
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Friedreich Ataxia, Cardiomyopathies
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA).
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
- Trial name
- NCT05302271
- Testing
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Weill Medical College of Cornell University
Spina Bifida, Myelomeningocele
tSCS in Children With Spina Bifida
A single-center, open-label, investigational pilot trial to explore potential effects of transcutaneous spinal cord stimulation on leg muscle strength and walking in children with myelomeningocele.
- Trial name
- NCT07615686
- Testing
- Transcutaneous stimulator
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Bailey Petersen
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
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
Spinal Muscular Atrophy 1
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
- Trial name
- NCT07554924
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 11 people
- Sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.
Friedreich Ataxia
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological…
- Trial name
- UNIFAI (NCT06016946)
- Where
- 34 sites in United States, Australia and 15 more
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Friedreich's Ataxia Research Alliance
Idiopathic Toe Walking, Cerebral Palsy
The Effect of Targeting the Plantaris Muscle-tendon in Surgical Correction of Ankle Equinus in Children
Tight ankle muscles can produce ankle equinus (limited ability to pull the foot upward) and occur often in children, significantly impacting their ability to walk.
- Conditions
- Idiopathic Toe Walking, Cerebral Palsy, Hereditary Spastic Paraparesis, Traumatic Brain Injury
- Trial name
- Plantaris Sx (NCT07090057)
- Testing
- Tendoachilles (TA) lengthening or gastrocnemius (GN) recession, then Plantaris tenotomy, Plantaris tenotomy, then Tendoachilles (TA) lengthening or gastrocnemius (GN) recession
- Where
- Canada
- Volunteers
- 42 people
- Sponsor
- University of Alberta
Adolescent Idiopathic Scoliosis (AIS)
Clinical and Biological Parameters in Adolescent Idiopathic Scoliosis
This single-center, observational, cross-sectional study aims to investigate the association between fecal calprotectin levels and clinical characteristics in adolescents with idiopathic scoliosis.
- Trial name
- NCT07900126
- Volunteers
- 83 people
- Sponsor
- Gaziler Physical Medicine and Rehabilitation Education and Research Hospital
Hereditary Autoinflammatory Diseases, Schnitzler Syndrome
AutoInflammatory Disease Alliance Registry (AIDA)
Autoinflammatory diseases (AID) are clinical entities characterized by recurrent inflammatory attacks in absence of infection, neoplasm or deregulation of the adaptive immune system.
- Conditions
- Hereditary Autoinflammatory Diseases, Schnitzler Syndrome, Behcet Syndrome, PFAPA Syndrome
- Trial name
- AIDA (NCT05200715)
- Testing
- No intervention is foreseen by the protocol.
- Where
- 112 sites in Algeria, Australia and 20 more
- Volunteers
- 3,500 people
- Sponsor
- University of Siena
Post-Acute COVID-19 Syndrome, ME/CFS
Unhide® Project: A Digital Health Platform to Collect Lifestyle Data for Brain Inflammation Research
The unhide® Project is a non-interventional, longitudinal research study designed to establish a secure data repository of demographic, health, and lifestyle information from individuals with brain inflammation and related…
- Conditions
- Post-Acute COVID-19 Syndrome, ME/CFS, Rheumatic Arthritis, Juvenile Rheumatoid Arthritis (JRA)
- Trial name
- NCT04806620
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Brain Inflammation Collaborative