NCT03333200View on ClinicalTrials.gov

Longitudinal Study of Neurodegenerative Disorders

RecruitingOBSERVATIONAL
Enrollment

1,500

Participants

Timeline

Start Date

January 11, 2012

Primary Completion Date

January 31, 2030

Study Completion Date

January 31, 2035

Conditions
MLDKrabbe DiseaseALDMPS IMPS IIMPS IIIVanishing White Matter DiseaseGM3 GangliosidosisPKANTay-Sachs DiseaseNP DeficiencyOsteopetrosisAlpha-MannosidosisSandhoff DiseaseNiemann-Pick DiseasesMPS IVGaucher DiseaseGANGM1 GangliosidosesMorquio DiseaseS-Adenosylhomocysteine Hydrolase DeficiencyBatten DiseasePelizaeus-Merzbacher DiseaseLeukodystrophyLysosomal Storage DiseasesPurine Nucleoside Phosphorylase DeficiencyMultiple Sulfatase Deficiency Disease
Interventions
OTHER

Palliative Care

Collecting information about the natural progression of these diseases

BIOLOGICAL

Hematopoetic Stem Cell Transplantation

Following patients who have received HSCT as part of their clinical care.

Trial Locations (1)

15224

RECRUITING

UPMC Children's Hospital of Pittsburgh, Pittsburgh

All Listed Sponsors
lead

University of Pittsburgh

OTHER

NCT03333200 - Longitudinal Study of Neurodegenerative Disorders | Biotech Hunter | Biotech Hunter