Status:
WITHDRAWN
Safety and Efficacy of Zuretinol Acetate in Subjects With Inherited Retinal Disease
Lead Sponsor:
Retinagenix Holdings
Conditions:
Retinal Disorder
Eligibility:
All Genders
6+ years
Phase:
PHASE2
PHASE3
Brief Summary
To evaluate the efficacy of ZA oral solution in subjects with IRD caused by biallelic recessive RPE65 or LRAT gene mutations and phenotypically diagnosed as Leber's Congenital Amaurosis (LCA) or Retin...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
December 1 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2023
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04311112
Start Date
December 1 2020
End Date
September 1 2023
Last Update
February 24 2023
Active Locations (1)
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1
NY NY
New York, New York, United States, 10001