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
Castration-resistant Prostate Cancer, Metastatic Cancer
pTVG-HP DNA Vaccine With or Without pTVG-AR DNA Vaccine and Pembrolizumab in Patients With Castration-Resistant, Metastatic Prostate Cancer
This trial will evaluate the use of one versus two DNA vaccines, delivered concurrently with PD-1 blockade using pembrolizumab followed by treatment with pembrolizumab alone, and delivered over a prolonged period of time (for a…
- Conditions
- Castration-resistant Prostate Cancer, Metastatic Cancer, Prostate Cancer
- Trial name
- NCT04090528
- Testing
- pTVG-HP, pTVG-AR, Pembrolizumab
- Where
- 2 sites in United States
- Volunteers
- 60 people
- Sponsor
- University of Wisconsin, Madison
Fetal Congenital Abnormalities, Fetoscopy
Fetoscopic Robotic Open Spina Bifida Treatment
Fetal spina bifida is a common birth defect that results in hydrocephalus, motor-, bowel-, bladder- and sexual dysfunction in the child. The condition is progressive in utero.
- Conditions
- Fetal Congenital Abnormalities, Fetoscopy, Fetal Surgery, Robotic Surgical Procedure
- Trial name
- FROST (NCT06907732)
- Testing
- Fetoscopic robotic open spina bifida closure
- Volunteers
- 15 people
- Sponsor
- Mount Sinai Hospital, Canada
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
Lumbar Spinal Stenosis, Cardiac Amyloidosis
Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis
Primary objective: To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modifying therapies are most effective.
- Conditions
- Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation
- Trial name
- NCT06034405
- Testing
- Tc99-PYP or Tc99-HDMP Scan
- Where
- 6 sites in United States
- Volunteers
- 1,663 people, healthy volunteers welcome
- Sponsor
- Columbia University
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
Spinocerebellar Ataxia Type 3
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3).
- Trial name
- NCT05160558
- Testing
- BIIB132
- Where
- 20 sites in United States, Germany and 4 more
- Volunteers
- 8 people
- Sponsor
- Biogen
Posterior Tibial Tendon Dysfunction, Pes Planus
Frequency of Pes Planus and Posterior Tibial Tendon Dysfunction in Patients With Ankylosing Spondylitis
This investigation aims to investigate the frequency of pes planus and posterior tibial tendon dysfunction in patients with ankylosing spondylitis.
- Conditions
- Posterior Tibial Tendon Dysfunction, Pes Planus, Spondylitis, Ankylosing
- Trial name
- NCT04810715
- Testing
- Patients with Ankylosing Spondylitis
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- Afyonkarahisar Health Sciences University
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
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
Amyotrophic Lateral Sclerosis, Kennedy Disease
Cell Signaling, Reinnervation and Metabolism in Kennedy Disease and Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS), is a rapidly progressive neurodegenerative disorder, usually leading to death from respiratory failure in 3-5 years.
- Trial name
- CERMALS (NCT05107349)
- Testing
- Muscle biopsy
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Machado-Joseph Disease
Weight in Lower Limbs Improves Gait Ataxia of in Machado-Joseph Disease Patients
Few studies evaluated the efficacy of adding weights on the lower limbs in patients with ataxic disorders. There is no current evidence on which would be the most appropriate weight for use in this context.
- Trial name
- NCT02906046
- Testing
- Weight in Lower Limbs
- Volunteers
- 20 people
- Sponsor
- Federal University of Health Science of Porto Alegre
Degeneration Spine, Spine Deformity
Reasons and Risk Factors for Unplanned Spinal Re-operation
Unplanned re-operation is one of the common negative indicators reflecting the quality and safety of surgery in the medical industry and has become one of the ten goals for the improvement of national medical quality and safety…
- Conditions
- Degeneration Spine, Spine Deformity, Spine Tumor, Spine Fracture
- Trial name
- NCT05995327
- Where
- 5 sites in China
- Volunteers
- 70,000 people
- Sponsor
- Peking University Third Hospital
Spinal Dysraphism
Uronephrological Complications Risk Factors in Spinal Dysraphism
Spinal dysraphism consist of congenital malformations resulting of abnormalities in the formation of neural tube and/or surrounding structures during embryogenesis.
- Trial name
- RUD (NCT05718440)
- Testing
- Individualisation of uronephrological complications risk factors
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
BRAF V600E, Colorectal Cancer
A Phase II Study of Sintilimab Combined With Ipilimumab N01, Cetuximab and Dabrafenib in Patients With Microsatellite-Stable, BRAF V600E-Mutated Metastatic Colorectal Cancer
Colorectal cancer (CRC) is the second leading cause of cancer-related death globally. BRAF V600E mutations occur in approximately 12% of metastatic CRC (mCRC) patients, conferring an extremely poor prognosis with a median overall…
- Conditions
- BRAF V600E, Colorectal Cancer, Sintilimab, MSS (Microsatellite Stable)
- Trial name
- NCT07506109
- Testing
- Ipilimumab N01, Sintilimab, Cetuximab
- Where
- 4 sites in China
- Volunteers
- 49 people
- Sponsor
- Tianjin Medical University Cancer Institute and Hospital