Clinical trials: Demyelinating Disorder
Inherited Metabolic Disorders (IMD)
A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456
- Trial name
- IMD-002 (NCT04008849)
- Testing
- Safety and efficacy assessments
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Magenta Therapeutics, Inc.
Metachromatic Leukodystrophy (MLD)
Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to transduce patient-derived hematopoietic stem…
- Trial name
- NCT07046338
- Testing
- Lentiviral TYF-ARSA correction of patient's autologous HSCs
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Healthy, Multiple Sclerosis
Normal Chinese Lifespan Brain Charts Initiative (NCLBCI)
Major brain diseases (including neuroimmune diseases, neurodegenerative diseases, and cerebrovascular diseases) are leading causes of death and disability in humans.
- Conditions
- Healthy, Multiple Sclerosis, Neuromyelitis Optica Spectrum Disorders, Neurodegenerative Diseases
- Trial name
- NCT06643858
- Testing
- No intervention
- Where
- China
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Beijing Tiantan Hospital
Neurogenic Detrusor Overactivity
Open-label, Long-term Safety, Efficacy, and Pharmacokinetics Study of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age With NDO and on CIC
The purpose of this study is to evaluate the safety, efficacy, and PK of Vibegron in pediatric participants with NDO who are regularly using CIC
- Trial name
- KANGUROO (NCT05491525)
- Testing
- Vibegron
- Where
- 38 sites in United States, Belgium and 14 more
- Volunteers
- 71 people
- Sponsor
- Urovant Sciences GmbH
Pediatric Onset Multiple Sclerosis (POMS)
Impact of Physical Activity on Cognitive Outcomes in Youth With Pediatric-Onset Multiple Sclerosis (POMS)
The purpose of this study is to to determine how implementing a home-based virtual reality video (VR) game exercise program in young people with Multiple Sclerosis(MS) can improve disability outcomes by measuring its impact on…
- Trial name
- NCT03933020
- Testing
- VR active video game intervention, Educational session, Standard Management of Physical Activity
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- The University of Texas Health Science Center, Houston