Health TrialsbyHealthPanel

Longitudinal Study of Neurodegenerative Disorders

ConditionsMLDKrabbe DiseaseALDMPS IMPS IIMPS IIIVanishing White Matter DiseaseGM3 GangliosidosisPKANTay-Sachs DiseaseNP DeficiencyOsteopetrosisAlpha-MannosidosisSandhoff DiseaseNiemann-Pick DiseasesMPS IVGaucher DiseaseGANGM1 GangliosidosesMorquio DiseaseS-Adenosylhomocysteine Hydrolase DeficiencyBatten DiseasePelizaeus-Merzbacher DiseaseLeukodystrophyLysosomal Storage DiseasesPurine Nucleoside Phosphorylase DeficiencyMultiple Sulfatase Deficiency Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorUniversity of Pittsburgh

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

Any patient with a genetic neurodegenerative disorder

Disqualifiers

none

Trial population

Persons with genetic neurodegenerative diseases.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Palliative Care

    Other intervention

    Collecting information about the natural progression of these diseases

  • Hematopoetic Stem Cell Transplantation

    Biological/Vaccine

    Following patients who have received HSCT as part of their clinical care.

Treatment groups

No treatment groups listed

Trial outcomes

Primary outcomes

1

Cognitive development

Repeated standardized age equivalent scores.

Time frame
15 years
2

Language development

Repeated standardized age equivalent scores.

Time frame
15 years
3

Gross Motor development .

Repeated standardized age equivalent scores.

Time frame
15 years
4

Fine Motor development

Repeated standardized age equivalent scores.

Time frame
15 years

Secondary outcomes

1

Exploratory biomarkers

Blood, CSF and urine

Time frame
15 years
2

Neurodegeneration of the brain as measured by MRI diffusion tensor imaging from birth to 5 years of age

Specialized technique to use DTI data to measure brain degeneration over time

Time frame
5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Pittsburgh

Lead sponsor

Source ClinicalTrials.gov
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