Safety and Efficacy of Zuretinol Acetate in Subjects With Inherited Retinal Disease

NCT ID: NCT04311112

Last Updated: 2023-02-24

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2/PHASE3
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-31
• Study Completion Date: 2023-09-30

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).

Conditions

Retinal Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

ZA placebo

Group Type: PLACEBO_COMPARATOR

Placebos

Intervention Type: DRUG

Placebo

ZA low dose

Group Type: ACTIVE_COMPARATOR

ZA Low dose

Intervention Type: DRUG

ZA low dose

ZA high dose

Group Type: ACTIVE_COMPARATOR

ZA high dose

Intervention Type: DRUG

ZA high dose

Interventions

Placebos

Placebo

Intervention Type: DRUG

ZA Low dose

ZA low dose

Intervention Type: DRUG

ZA high dose

ZA high dose

Intervention Type: DRUG

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.

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Retinagenix Holdings

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

NY NY

New York, New York, United States

Countries

United States

Other Identifiers

RG201

Identifier Type: -

Identifier Source: org_study_id