Clinical trial NCT04311112: Safety and Efficacy of Zuretinol Acetate in Subjects With Inherited Retinal Disease
Official title: Safety and Efficacy of Zuretinol Acetate Oral Solution in Subjects With Inherited Retinal Disease Caused by Mutations in Retinal Pigment Epithelium Protein 65 or Lecithin:Retinol Acyltransferase
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
PHASE3
Conditions: Retinal Disorder
Lead sponsor: Retinagenix Holdings
Sponsor class: INDUSTRY
First posted: 2020-03-17
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 Retinitis Pigmentosa (RP).


Eligibility criteria: 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.

Primary outcomes:
measure: Change in functional vision at Week 12 using a visual navigation course at different ambient illumination levels
timeFrame: week 12
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: NY NY
state: New York
Study officials:
References: