CompletedNot Applicable

A Natural History Study of Infantile Neuroaxonal Dystrophy

China, Egypt, India43 participantsStarted 2019-06-17

Plain-language summary

This study is a longitudinal and prospective study of the natural history of infantile neuroaxonal dystrophy (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 or compound heterozygous PLA2G6 variants * Signed informed consent form (ICF) prior to entry into the registry Exclusion Criteria: * Diagnosis of atypical NAD (ANAD) * Additional underlying diagnosis with features that overlap with INAD * Unwilling or unable to allow medical record review * Unwilling or unable to participate in serial assessments every 6 months (including deceased patients)

What they're measuring

INAD Mortality

Timeframe: 1-2 years of follow-up is planned

Trial details

NCT IDNCT04027816

SponsorBiojiva LLC

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2022-02-14

View on ClinicalTrials.gov More Neuroaxonal Dystrophy, Infantile trials