Home › Leukodystrophies › NCT03333200
Longitudinal Study of Neurodegenerative Disorders
RecruitingObservational study
Who can join
All ages · All sexes
Full eligibility criteria
Inclusion Criteria: * Any patient with a genetic neurodegenerative disorder Exclusion Criteria: * none
About the study
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 understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.
What is being tested
- Palliative Care (other)
- Hematopoetic Stem Cell Transplantation (biological)
Sponsor: University of Pittsburgh · Participants: 1,500 · Started: Jan 11, 2012
Contact the study team
- Deepa Rajan, MD · Phone: 412-692-8388
Official record on ClinicalTrials.gov — NCT03333200
Locations in the U.S.
| Pennsylvania | UPMC Children's Hospital of Pittsburgh, Pittsburgh |
Conditions
- Leukodystrophies
- Sudden Infant Death Syndrome
- Leukoencephalopathies
- alpha-Mannosidosis
- Mucopolysaccharidosis IV
- Gaucher Disease
- Neuronal Ceroid-Lipofuscinoses
- Pelizaeus-Merzbacher Disease
- Language
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.