Clinical trials: Demyelinating Disorders
Adrenoleukodystrophy
Newborn Screening for Adrenoleukodystrophy
To test if the routine newborn screening dried blood spots can be used to test if elevation of C26:0 lysophosphatidylcholine (C26:0-lyso-PC), a status indicating adrenoleukodystrophy (ALD)
- Trial name
- NCT02952482
- Testing
- newborns testing for ALD
- Where
- Taiwan
- Volunteers
- 45,796 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Inherited Mitochondrial Disease
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical…
- Trial name
- NCT05218655
- Testing
- Vatiquinone
- Where
- 26 sites in United States, France and 5 more
- Volunteers
- 101 people
- Sponsor
- PTC Therapeutics
Relapsing-Remitting Multiple Sclerosis
PK and PD Study of Natalizumab in Pediatric Subjects With RRMS
The primary objective of the study is to determine the pharmacokinetic (PK) profile of multiple doses of natalizumab in pediatric subjects with relapsing-remitting multiple sclerosis (RRMS).
- Trial name
- NCT01884935
- Testing
- Natalizumab
- Where
- 5 sites in Italy
- Volunteers
- 13 people
- Sponsor
- Biogen
Multiple Sclerosis, Neuromyelitis Optica
Evaluation of Neural Loss in Multiple Sclerosis and Neuromyelitis Optica Using High Resolution Oct
The purpose of this study is to determine the ability of STRATUS OCT and high resolution OCT to detect and to quantify the neural loss in multiple sclerosis and neuromyelitis optica and its subtypes.
- Trial name
- NCT01024985
- Where
- Brazil
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Neuromyelitis Optica Spectrum Disorder, Pregnancy Related
Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy
This is an observational study to monitor female participants exposed to UPLIZNA during pregnancy. This study requires voluntary reporting of pregnancies in female participants with NMOSD exposed to UPLIZNA during pregnancy or…
- Trial name
- NCT05909761
- Testing
- UPLIZNA
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Amgen
X-Linked Adrenoleukodystrophy
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
- Trial name
- NCT03196765
- Testing
- Sobetirome (NV1205)
- Where
- 13 sites in Argentina, Australia and 6 more
- Sponsor
- NeuroVia, Inc.
Myasthenia Gravis, Guillain-Barre Syndrome
Ventilator Weaning Practices in Patients Intubated for Guillain-Barré Syndrome or Myasthenia Gravis
Guillain-Barré syndrome and myasthenia gravis are two neuromuscular diseases that are commonly managed in ICUs that may require different weaning protocols.
- Trial name
- GUIMAUVE (NCT07022028)
- Where
- France
- Volunteers
- 800 people
- Sponsor
- Poitiers University Hospital
Adrenoleukodystrophy
Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the efficacy of interferon beta and thalidomide in male patients with adrenoleukodystrophy who show evidence of brain inflammatory response and are receiving concurrent glyceryl trierucate and glyceryl…
- Trial name
- NCT00004450
- Testing
- glyceryl trierucate, glyceryl trioleate, interferon beta
- Volunteers
- 60 people
- Sponsor
- FDA Office of Orphan Products Development
Neuroinflammatory Diseases, Multiple Sclerosis
FIT-ATOMIC Exercise Feasibility Trial
This multi-center, randomized controlled feasibility trial will assess a 20-week home-based exercise intervention in youth with Multiple Sclerosis (MS).
- Trial name
- FIT-ATOMIC (NCT06870968)
- Testing
- Exercise Training, Mobility and Flexibility Training
- Where
- 4 sites in United States, Canada
- Volunteers
- 40 people
- Sponsor
- The Hospital for Sick Children
Multiple Sclerosis
An Observational Study on Teriflunomide-exposed Pregnancies
Primary Objective: To evaluate any potential increase in the risk of major birth defects, in the first year of life, in teriflunomide-exposed pregnancies.
- Trial name
- NCT03198351
- Testing
- Teriflunomide (HMR1726)
- Where
- 2 sites in United States, Canada
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- Sanofi
Multiple Sclerosis
Safety Study in Patients With Multiple Sclerosis Treated Fingolimod or Other Approved Disease-modifying Therapies
The purpose of this world-wide prospective parallel-cohort study in patients with relapsing forms of MS, either newly treated with fingolimod or receiving another disease-modifying therapy, is to further explore the incidence of…
- Trial name
- PASSAGE (NCT01442194)
- Testing
- other disease-modifying therapy, Fingolimod
- Where
- 238 sites in United States, Argentina and 5 more
- Volunteers
- 3,076 people
- Sponsor
- Novartis Pharmaceuticals
Clinically Isolated Syndrome (CIS), Multiple Sclerosis (MS)
Centralized Reading Assessment of Chronic CerebroSpinal Venous Insufficiency (CCSVI) in Patients With Multiple Sclerosis and Other Neurological Diseases
This a retrospective, blinded trial in which collaborators in Italy will review the doppler findings from the Combined Transcranial and Extracranial Venous Doppler (CTEVD) trial in an attempt to measure reproducibility.
- Conditions
- Clinically Isolated Syndrome (CIS), Multiple Sclerosis (MS), Other Neurological Disorders (OND), Healthy (HC)
- Trial name
- NCT02308579
- Volunteers
- 499 people
- Sponsor
- University at Buffalo
Multiple Sclerosis, Relapsing-Remitting
Extension Study of BG00012 in Pediatric Subjects With Relapsing Remitting Multiple Sclerosis (RRMS)
The primary objective of the study is to evaluate the long-term safety of BG00012 in subjects who completed Study 109MS202 (NCT02410200).
- Trial name
- NCT02555215
- Testing
- dimethyl fumarate
- Where
- 12 sites in United States, Belgium and 8 more
- Volunteers
- 20 people
- Sponsor
- Biogen
Pain, Traumatic Brain Injury
The NRC Dataset. Developing a Minimum Dataset for Multidisciplinary Rehabilitation: a Modified E-Delphi Study
Several rehabilitation datasets are presently in use for specific patient groups in England. The 'National Clinical Audit of Specialist Rehabilitation for patients with complex needs following major injury' (NCASRI) evaluated…
- Conditions
- Pain, Traumatic Brain Injury, Major Trauma, Multiple Sclerosis
- Trial name
- NCT06726135
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- ThomasCorbett
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Multiple Sclerosis
Treadmill Training in Chronic MS: Efficacy and Cost-effectiveness
The purpose of the study is to determine whether treadmill training is safe and beneficial in patients with walking difficulty because of multiple sclerosis.
- Trial name
- NCT00334100
- Testing
- Exercise
- Where
- United States
- Sponsor
- US Department of Veterans Affairs
Lysosomal Storage Disorders, Sandhoff Disease
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
- Conditions
- Lysosomal Storage Disorders, Sandhoff Disease, Krabbe Disease, CLN2
- Trial name
- NCT07740512
- Testing
- PLX-200
- Volunteers
- 24 people
- Sponsor
- Polaryx Therapeutics, Inc.
Multiple Sclerosis, Cognitive Disorders
Effect of Transcutaneous Electrical Nerve Stimulation on Cognition in Multiple Sclerosis Patients
The aim of this study is to evaluate whether the Transcutaneous Electrical Nerve Stimulation (TENS) is effective on improvement of cognitive disorders in Multiple sclerosis patients.
- Trial name
- NCT02876536
- Testing
- TENS
- Where
- Iran
- Volunteers
- 20 people
- Sponsor
- Isfahan University of Medical Sciences
Devic's Disease
Hematopoietic Stem Cell Transplant in Devic's Disease
This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with rabbit antithymocyte globulin (rATG)/rituximab (drugs which reduce the function of the immune system)…
- Trial name
- NCT00787722
- Testing
- Hematopoietic Stem Cell Transplantation, Cyclophosphamide, G-CSF
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Northwestern University
Multiple Sclerosis, NMO Spectrum Disorder
China National Registry of Neuro-Inflammatory Diseases
Central nervous system (CNS) idiopathic inflammatory demyelinating diseases (IDD) are mainly diseases caused by autoimmune factors that result in CNS demyelination damage and loss.
- Conditions
- Multiple Sclerosis, NMO Spectrum Disorder, Clinically Isolated Syndrome, CNS Demyelinating Autoimmune Diseases
- Trial name
- CNRID (NCT05154370)
- Testing
- Intravenous steroid
- Where
- China
- Volunteers
- 10,000 people
- Sponsor
- Beijing Tiantan Hospital