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.
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03333200
- Recruitment status
- Recruiting
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 1,500 participants
Conditions and interventions
Conditions
- 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
- Palliative Care Other
- Hematopoetic Stem Cell Transplantation Biological
Other · Biological
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 Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03333200 live on ClinicalTrials.gov.