CompletedNot Applicable

Natural History of Infantile Neuroaxonal Dystrophy

United States13 participantsStarted 2018-07-30

Plain-language summary

This is a retrospective and cross-sectional review of the natural history of INAD.

Who can participate

Age range18 Months – 10 Years

SexALL

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

Inclusion Criteria: * Male or female 18 months to 10 years of age * Medical history consistent with the symptoms of classic INAD (onset of symptoms between the ages of 6 months and 3 years) * Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes) * Signed informed consent form (ICF) prior to entry into the registry Exclusion Criteria: * Diagnosis of atypical NAD (ANAD) * Unwilling or unable to allow medical record review

What they're measuring

To describe the natural history of infantile neuroaxonal dystrophy (INAD).

Timeframe: Birth to time of enrollment.

Trial details

NCT IDNCT03999814

SponsorBiojiva LLC

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2020-02-27

View on ClinicalTrials.gov More Infantile Neuroaxonal Dystrophy trials