Longitudinal Study of Neurodegenerative Disorders
Public ClinicalTrials.gov record NCT03333200. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Study identification
- NCT ID
- NCT03333200
- Recruitment status
- Recruiting
- Study type
- Observational
- Phase
- Not listed
- Lead sponsor
- University of Pittsburgh
- Other
- Enrollment
- 1,500 participants
Conditions and interventions
Conditions
- ALD
- Alpha-Mannosidosis
- Batten Disease
- GAN
- GM1 Gangliosidoses
- GM3 Gangliosidosis
- Gaucher Disease
- Krabbe Disease
- Leukodystrophy
- Lysosomal Storage Diseases
- MLD
- MPS I
- MPS II
- MPS III
- MPS IV
- Morquio Disease
- Multiple Sulfatase Deficiency Disease
- NP Deficiency
- Niemann-Pick Diseases
- Osteopetrosis
- PKAN
- Pelizaeus-Merzbacher Disease
- Purine Nucleoside Phosphorylase Deficiency
- S-Adenosylhomocysteine Hydrolase Deficiency
- Sandhoff Disease
- Tay-Sachs Disease
- Vanishing White Matter Disease
Interventions
- Hematopoetic Stem Cell Transplantation Biological
- Palliative Care Other
Biological · Other
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 10, 2012
- Primary completion
- Dec 31, 2029
- Completion
- Dec 31, 2034
- Last update posted
- Feb 8, 2026
2012 – 2035
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03333200, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 8, 2026 · Synced May 20, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03333200 live on ClinicalTrials.gov.