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6 recruiting studies matching your filters

A Phase 1b/2 Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy Following Fosigotifator Administration in Adult and Pediatric Subjects With Vanishing White Matter Disease

Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants. This…

Vanishing White Matter Disease

Estimated

~$10 – $8,750

Calico Life Sciences LLCNCT05757141

Phase 1

Retinal Vasoreactivity is a Marker for Cerebral Small Vessel Disease Progression

Cerebral small vessel disease (SVD), present in 80-94% of adults over age 65 years, increases the risk of stroke by 2-fold, and dementia by 2.3-fold. There is …

Cerebral Small Vessel DiseasesCerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and LeukoencephalopathyCerebral Microbleeding+1 more

Estimated

~$10 – $6,250

Mayo ClinicNCT04753970

Characterization of the Natural History of Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation

In this study, we will conduct retrospective chart and imaging reviews and prospective longitudinal virtual assessments of individuals with LBSL.

LeukoencephalopathiesLBSLLeukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation+3 more

Estimated

~$10 – $1,050

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.NCT03624374

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…

LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more

Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandi…

MLDKrabbe DiseaseALD+24 more

Estimated

~$10 – $6,250

University of PittsburghNCT03333200

A Patient Registry and Natural History Study of Patients with Biallelic HPDL Mutations

This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations

Mitochondrial EncephalomyopathiesHereditary Spastic ParaplegiaSpastic Paraplegia+4 more

Estimated

~$10 – $750

University of California, San DiegoNCT05848271