Safety and Efficacy of Zuretinol Acetate in Subjects With... | NCT04311112

NCT04311112

Protocol Section

Identification Module

Results Section

No data available

No data is available for this block.

Annotation Section

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No data is available for this block.

Document Section

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No data is available for this block.

Derived Section

Miscellaneous Info Module

Status Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Label	Type	Intervention Names
ZA placebo	Placebo Comparator	Drug: Placebos
ZA low dose	Active Comparator	Drug: ZA Low dose
ZA high dose	Active Comparator	Drug: ZA high dose

Name	Type	Description	Arm Group Labels	Other Names
Placebos	Drug	Placebo	ZA placebo
ZA Low dose

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Change in functional vision at Week 12 using a visual navigation course at different ambient illumination levels	The primary efficacy endpoint (PEE) will be a measure of functional vision assessed at Week 12 employing mobility testing (Ora Inc, Andover, MA) via their Visual Navigation Challenge (VNC) course. The VNC measures functional vision by evaluating the subject's ability to navigate a maze accurately and successfully at different levels of ambient illumination. The PEE is a comparison between groups in their mean change from randomization to week 12, that is, the difference in VNC score from baseline performance to week 12 performance. Performance on the VNC is assessed using each eye individually and both eyes together at 1 or more levels of illumination that range from 0.35 lux to 500 lux. A subject's VNC score is the lowest level of luminance at which the subject can navigate and correctly pass through the maze.	week 12

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Have read, understood and signed the informed consent form (ICF).
Be aged 6 years or older.
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.
Be naïve to gene therapy, surgical implantation of prosthetic retinal chips, or subretinal injections.
If previously administered ZA , have at least > 3 years since last administration of ZA.
Pregnancy testing and contraception before study treatment: Women of childbearing potential must not be pregnant or lactating.

Exclusion Criteria:

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.
Have had ocular surgery within 3 months of Screening, including cataract or laser procedures.
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.
Have taken any supplements containing ≥ 10,000 IU vitamin A within 60 days of Screening.
Have taken any medication that affects bone metabolism within 6 months of Screening.
Have circulating 25-hydroxy vitamin D < 20 ng/mL.
Use of medications that may interact with a retinoid, including tetracycline, ketoconazole and methotrexate within 60 days of Screening.
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.
Have a known and documented allergy to soy.