Longitudinal Study of Neurodegenerative Disorders

Recruiting now

Conditions studied: MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease

In brief

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.

Key facts

Study ID

NCT03333200

Run by

University of Pittsburgh

People needed

1500

Starts

2012-01-11

Expected to finish

2035-01-01

Last updated by the study team

2026-02-09

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Any patient with a genetic neurodegenerative disorder

You may not qualify if…

• none

Where it is running

• UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)

Full record on ClinicalTrials.gov

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