CompletedNot Applicable

A Natural History Study of Canavan Disease

United States, Germany21 participantsStarted 2016-05

Plain-language summary

The purpose of this study is to learn more about the natural history of Canavan disease

Who can participate

Age range99 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * The patient must have a confirmed diagnosis of Canavan Disease as defined by elevated NAA levels, decreased ASPA activity, or mutations in the ASPA gene. Exclusion Criteria: * The PI will assess whether it is in the best interest of the patient to exclude them from the study for their own comfort and well being. In cases where the PI deems it appropriate, severely affected patients will be excluded

What they're measuring

Change in Gross Motor Function Measure over time

Timeframe: baseline, 6 months, 1 year, 18 months, 2 years

Trial details

NCT IDNCT02851563

SponsorNYU Langone Health

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2019-04

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