| Eligibility |
Inclusion Criteria:
1. Have read, understood and signed the informed consent form (ICF).
2. Be aged 6 years or older.
3. Have a diagnosis of IRD phenotypically diagnosed as LCA or RP by an ocular geneticist
or ophthalmologist and caused by pathologic biallelic autosomal recessive mutation in
RPE65 or LRAT as determined by a fully accredited certified central genotyping
laboratory.
4. Be naïve to gene therapy, surgical implantation of prosthetic retinal chips, or
subretinal injections.
5. If previously administered ZA , have at least > 3 years since last administration of
ZA.
6. Pregnancy testing and contraception before study treatment: Women of childbearing
potential must not be pregnant or lactating.
Exclusion Criteria:
1. Have a presence of concurrent ocular disease that in the opinion of the Investigator
would put the subject at greater risk during the study or significantly affect study
results.
2. Have had ocular surgery within 3 months of Screening, including cataract or laser
procedures.
3. Have taken any prescription or investigational oral retinoid medication (e.g.,
isotretinoin or acitretin) within 6 months of Screening; subjects who did not tolerate
their previous oral retinoid medication will be excluded regardless of the time of
last exposure.
4. Have taken any supplements containing = 10,000 IU vitamin A within 60 days of
Screening.
5. Have taken any medication that affects bone metabolism within 6 months of Screening.
6. Have circulating 25-hydroxy vitamin D < 20 ng/mL.
7. Use of medications that may interact with a retinoid, including tetracycline,
ketoconazole and methotrexate within 60 days of Screening.
8. Use of intraocular or periocular corticosteroids within 90 days of Screening; use of
corticosteroid implants within 3 years of Screening; use of systemic corticosteroids
unless these are at a steady low dose with low dose level in effect prior to or at
Screening; or use of intraocular or periocular anti-vascular endothelial growth factor
agents within 2 months of Screening.
9. Have a known and documented allergy to soy.
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