Trials / Recruiting
RecruitingNCT03333200
Longitudinal Study of Neurodegenerative Disorders
- Status
- Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 1,500 (estimated)
- Sponsor
- University of Pittsburgh · Academic / Other
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
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.
Detailed description
Patients would be evaluated by a multidisciplinary team at specific time points every 3 months the first year, every 6 months the second year and once a year thereafter.
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
| Type | Name | Description |
|---|---|---|
| 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. |
Timeline
- Start date
- 2012-01-11
- Primary completion
- 2030-01-01
- Completion
- 2035-01-01
- First posted
- 2017-11-06
- Last updated
- 2026-02-09
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT03333200. Inclusion in this directory is not an endorsement.