Natural History of Infantile Neuroaxonal Dystrophy

Completed

Conditions studied: Infantile Neuroaxonal Dystrophy, INAD

In brief

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

Key facts

Study ID

NCT03999814

Run by

Biojiva LLC

People needed

13

Starts

2018-07-30

Expected to finish

2020-02-27

Last updated by the study team

2020-06-29

Who can join

Age: 2 and older, up to 10. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Diagnosis of atypical NAD (ANAD)
• Unwilling or unable to allow medical record review

Where it is running

• Sarah Endemann — Los Altos, California, United States

Full record on ClinicalTrials.gov

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