Crick
HomeTrialsGenesDrugs23andMeGraphBlogContact
  • Home
  • Trials
  • Genes
  • Drugs
  • 23andMe
  • Graph
  • Blog
  • Contact
Crick

An open-source clinical intelligence platform. Explore clinical trials, gene networks, and molecular structures using public data sources.

Data Sources

  • ClinicalTrials.gov
  • OpenTargets
  • ClinVar
  • PubChem

Links

  • Contact
  • About
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Hereditary Central Nervous System Demyelinating Diseases”

172 trials

Filters

Phase
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Status
Recruiting
Active, Not Recruiting
Not Yet Recruiting
Completed
Enrolling by Invitation

Showing 20 of 172 results

Large-scale testing (Phase 3)Ended earlyNCT04378075
What this trial is testing

Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Who this might be right for
Mitochondrial DiseasesDrug Resistant EpilepsyLeigh Disease+5 more
PTC Therapeutics 68
Early research (Phase 1)Looking for participantsNCT06150716
What this trial is testing

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Who this might be right for
Pelizaeus-Merzbacher Disease
Ionis Pharmaceuticals, Inc. 24
Not applicableLooking for participantsNCT06796920
What this trial is testing

Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy

Who this might be right for
Adrenomyeloneuropathy Without Cerebral Involvement
Third Military Medical University 10
Not applicableTemporarily pausedNCT04031469
What this trial is testing

A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Disease

Who this might be right for
Gut MicrobiomeGastrointestinal MicrobiomeAutism+77 more
ProgenaBiome 1,000
Not applicableWithdrawnNCT01093105
What this trial is testing

An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)

Who this might be right for
Leukodystrophy, Globoid Cell
Shire
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Not applicableEnded earlyNCT02204904
What this trial is testing

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
X-Linked Adrenoleukodystrophy (X-ALD)Cerebral Adrenoleukodystrophy (CALD)Adrenoleukodystrophy (ALD)
Genetix Biotherapeutics Inc. 59
Not applicableUnknownNCT00005900
What this trial is testing

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
I Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more
Fairview University Medical Center 10
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableStudy completedNCT01592552
What this trial is testing

A Biospecimen and Clinical Data Study on Patients for Drug & Biomarker Discovery

Who this might be right for
Alzheimer's DiseaseMultiple SclerosisParkinson's Disease+2 more
Sanguine Biosciences 20
Testing effectiveness (Phase 2)WithdrawnNCT05200104
What this trial is testing

Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

Who this might be right for
ALD (Adrenoleukodystrophy)
Poxel SA
Not applicableStudy completedNCT01626092
What this trial is testing

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Who this might be right for
Lysosomal Storage DiseasePeroxisomal Disorder
Masonic Cancer Center, University of Minnesota 3
Not applicableActive Not RecruitingNCT04126005
What this trial is testing

Natural History Study of Patients With Canavan Disease (CANinform Study)

Who this might be right for
Canavan Disease
Aspa Therapeutics 70
Not applicableNo Longer AvailableNCT03639844
What this trial is testing

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Who this might be right for
Hurler SyndromeInherited Metabolic DisorderLysosomal Storage Disorder+2 more
Bellicum Pharmaceuticals
Early research (Phase 1)Study completedNCT02595489
What this trial is testing

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Stanford University 21
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Not applicableEnded earlyNCT01963650
What this trial is testing

Natural History Study of Children With Metachromatic Leukodystrophy

Who this might be right for
Lipid Metabolism DisordersMetachromatic Leukodystrophy (MLD)Nervous System Diseases+11 more
Shire 1
Not applicableStudy completedNCT03653390
What this trial is testing

A Community Wellness Program for Adults Living With Long-term Physical Disability

Who this might be right for
Physical Disability
University of Washington 516
Testing effectiveness (Phase 2)Looking for participantsNCT04998396
What this trial is testing

AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

Who this might be right for
Canavan Disease
Aspa Therapeutics 26
Not applicableStudy completedNCT00006057
What this trial is testing

Diagnostic and Screening Study of Genetic Disorders

Who this might be right for
Tay-Sachs DiseasePorphyria, ErythropoieticLeukodystrophy, Globoid Cell+1 more
National Center for Research Resources (NCRR) 50
Load More Results