Longitudinal Study of Neurodegenerative Disorders

  • End date
    Jan 13, 2035
  • participants needed
  • sponsor
    University of Pittsburgh
Updated on 13 November 2021
stem cell transplantation
cell transplantation
genetic disorder
palliative care
neurodegenerative disorders
genetic disorders


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.


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.

Condition S-Adenosylhomocysteine Hydrolase Deficiency, Adrenoleukodystrophy, mucopolysaccharidosis type i, Metachromatic leukodystrophy, Batten Disease, Hunter's Syndrome, hurler syndrome, GM1 Gangliosidoses, Hallervorden-Spatz Disease, niemann pick disease, Pelizaeus-Merzbacher Disease, Metachromatic Leukodystrophy - MLD, Tay-Sachs Disease, Giant Axonal Neuropathy, gaucher's disease, dysostosis multiplex, Hunter Syndrome (MPS II), tay sachs disease, Neuronal Ceroid Lipofuscinoses (NCL), GM3 Gangliosidosis, Multiple Sulfatase Deficiency, krabbe disease, Osteopetrosis, Infantile Neuronal Ceroid Lipofuscinosis, Gaucher Disease, Sandhoff Disease, Hurler's Syndrome, Neuronal ceroid lipofuscinosis, Krabbe's Disease, Sanfilippo's Syndrome, mps iii, Lysosomal Storage Disease, NP Deficiency, Purine Nucleoside Phosphorylase Deficiency, Niemann-Pick Disease, alpha-Mannosidosis, Leukodystrophy, Osteochondrodystrophy, Vanishing White Matter Disease
Treatment Palliative Care, Hematopoetic Stem Cell Transplantation
Clinical Study IdentifierNCT03333200
SponsorUniversity of Pittsburgh
Last Modified on13 November 2021


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Inclusion Criteria

Any patient with a genetic neurodegenerative disorder

Exclusion Criteria

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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