Longitudinal Study of Neurodegenerative Disorders

Part of paid clinical trials in Pittsburgh, Pennsylvania.

Sponsor
University of Pittsburgh
Study ID
NCT03333200
Status
Recruiting
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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

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Palliative Care — OTHER
    Collecting information about the natural progression of these diseases
  • Hematopoetic Stem Cell Transplantation — BIOLOGICAL
    Following patients who have received HSCT as part of their clinical care.

Study Details

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 Dates

Start date
Jan 11, 2012
Status verified
Feb 2026
Primary completion
Jan 31, 2030
Completion
Jan 31, 2035

Study Design

Enrollment
1,500 participants (estimated)

Primary Outcome Measure

Cognitive development [ Time Frame: 15 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224
Deepa Rajan, MD
[email protected]
412-692-8388

Find similar trials in Pittsburgh, PA

By research site
UPMC Children's Hospital of Pittsburgh· Pittsburgh, PA

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