# Longitudinal Study of Neurodegenerative Disorders

> **NCT03333200** · — · RECRUITING · sponsor: **University of Pittsburgh** · enrollment: 1500 (estimated)

## 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

## Interventions

- **OTHER:** Palliative Care
- **BIOLOGICAL:** Hematopoetic Stem Cell Transplantation

## Key facts

- **NCT ID:** NCT03333200
- **Lead sponsor:** University of Pittsburgh
- **Sponsor class:** OTHER
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** RECRUITING
- **Start date:** 2012-01-11
- **Primary completion:** 2030-01
- **Final completion:** 2035-01
- **Target enrollment:** 1500 (ESTIMATED)
- **Last updated:** 2026-02-09


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03333200

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03333200, "Longitudinal Study of Neurodegenerative Disorders". Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/clinical/NCT03333200. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*