Longitudinal Study of Neurodegenerative Disorders

Last updated: March 4, 2025
Sponsor: University of Pittsburgh
Overall Status: Active - Recruiting

Phase

N/A

Condition

Neuronal Ceroid Lipofuscinoses (Ncl)

Multiple Sclerosis

Gaucher Disease

Treatment

Hematopoetic Stem Cell Transplantation

Palliative Care

Clinical Study ID

NCT03333200
STUDY19020318
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Any patient with a genetic neurodegenerative disorder

Exclusion

Exclusion Criteria:

  • none

Study Design

Total Participants: 1500
Treatment Group(s): 2
Primary Treatment: Hematopoetic Stem Cell Transplantation
Phase:
Study Start date:
January 11, 2012
Estimated Completion Date:
January 31, 2035

Study 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.

Connect with a study center

  • The Program for the Study of Neurodevelopment in Rare Disorders, Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania 15224
    United States

    Active - Recruiting

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania 15224
    United States

    Active - Recruiting

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