White Matter Disease Clinical Trials

3 recruiting

White Matter Disease Trials at a Glance

7 actively recruiting trials for white matter disease are listed on ClinicalTrialsFinder across 6 cities in 4 countries. The largest study group is Phase 1 with 2 trials, with the heaviest enrollment activity in Philadelphia, Salt Lake City, and Baltimore. Lead sponsors running white matter disease studies include Children's Hospital of Philadelphia, Hugo W. Moser Research Institute at Kennedy Krieger, Inc., and Calico Life Sciences LLC.

Browse white matter disease trials by phase

Treatments under study

About White Matter Disease Clinical Trials

Looking for clinical trials for White Matter Disease? There are currently 3 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new White Matter Disease trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about White Matter Disease clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 17 of 7 trials

Recruiting

Longitudinal Study of Neurodegenerative Disorders

Gaucher DiseaseBatten DiseaseLeukodystrophy+24 more
University of Pittsburgh1,500 enrolled1 locationNCT03333200
Recruiting
Phase 1Phase 2

An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

Vanishing White Matter Disease
Calico Life Sciences LLC50 enrolled5 locationsNCT05757141
Recruiting

The Myelin Disorders Biorepository Project

AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369
Recruiting

Diffusion Magnetic Resonance Imaging (dMRI) in the Early Evaluation of Brain White Matter Diseases

Diffusion Magnetic Resonance ImagingBrain White Matter Diseases
The General Authority for Teaching Hospitals and Institutes150 enrolled1 locationNCT07108712
Recruiting
Phase 1Phase 2

Retina is a Marker for Cerebrovascular Heath

Cerebral Small Vessel DiseasesCerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and LeukoencephalopathyCerebral Microbleeding+1 more
Mayo Clinic100 enrolled1 locationNCT04753970
Recruiting

Natural History Study of Patients with HPDL Mutations

Neonatal EncephalopathyGenetic DiseaseMutation+4 more
University of California, San Diego50 enrolled1 locationNCT05848271
Recruiting

Natural History Study of Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation (LBSL)

Genetic DiseaseAtaxia, CerebellarWhite Matter Disease+3 more
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.100 enrolled1 locationNCT03624374