Assessing OCU410ST for treating Stargardt Disease

A Phase 1/2 Study to Assess the Safety and Efficacy of OCU410ST for STARGARDT DISEASE

This study is testing a new treatment called OCU410ST to see if it can help improve vision in children and young adults with Stargardt Disease.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 42 (estimated)
• Ages: 6 Years to 65 Years
• Sex: All
• Sponsor: Ocugen (Industry-sponsored)
• Locations: 6 sites (Phoenix, Arizona and 5 other locations)
• Trial ID: NCT05956626 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of OCU410ST, an adeno-associated viral vector designed to treat Stargardt Disease, which causes vision loss in children and young adults. The study is conducted in two phases: Phase 1 is an open-label, dose-ranging study enrolling up to 18 subjects, while Phase 2 is a randomized, dose-expansion cohort involving 24 subjects. Participants will be assigned to treatment groups or a control group based on their age and treatment status. The trial aims to determine if OCU410ST can restore homeostasis in the eye and improve vision outcomes.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 6-65 with a clinical diagnosis of Stargardt Disease and specific genetic mutations in the ABCA4 gene.

Not a fit: Patients with other forms of vision loss or those who do not meet the genetic and clinical criteria for Stargardt Disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from Stargardt Disease, potentially improving their vision and quality of life.

How similar studies have performed: While there is limited data on similar gene therapy approaches for Stargardt Disease, the use of adeno-associated viral vectors has shown promise in other ocular conditions.

Eligibility criteria

Inclusion Criteria:

1. Are aged 18-65.
2. Have clinical evidence of a macular lesion phenotypically consistent with Stargardt Disease
3. The study eye should have at least one well-demarcated area of atrophy with a minimum diameter of 300 microns, and total lesion size \<= 18 mmE2 and a BCVA of 50 ETDRS letters or better
4. Have confirmed presence of two pathogenic mutations in the ABCA4 gene
5. Have detectable outer nuclear layer (ONL) in the macular region tomography (SD-OCT).
6. Have BCVA of 50 letters or less (using ETDRS chart)

Key Inclusion Criteria for Pediatric Subjects:

1. Are aged 6-17.
2. Have clinical diagnosis of Stargardt Disease
3. The designated primary study eye must have at least one well-demarcated area of atrophy with a minimum diameter of 300 microns and a total lesion area \<= 18 mmE2 and a BCVA of 35 ETDRS letters or better.
4. Have two (2) pathogenic mutations confirmed present, in the ABCA4 gene.

Key Exclusion Criteria for Adult Subjects:

1. Have previous treatment with a gene therapy or cell therapy product.
2. Have any concurrent retroviral therapy that would inactivate the investigational product.
3. Have any contradictions for subretinal injection and the use of anesthesia.
4. Have genes that mimic Stargardt Disease like ELOVL4, or PROM1.

Exclusion Criteria for Pediatric Subjects:

1. Have previous treatment with a gene therapy or cell therapy product.
2. Have any concurrent retroviral therapy that would inactivate the investigational product.
3. Have any intraocular surgery (including lens replacement surgery) within 6 months (prior to Screening), and any ophthalmic condition that may require surgery during the study period.
4. Have genes that mimic Stargardt Disease like ELOVL4, or PROM1.

Where this trial is running

Phoenix, Arizona and 5 other locations

• Associated Retina Consultants — Phoenix, Arizona, United States (Recruiting)
• Bascom Palmer Eye Institute — Miami, Florida, United States (Not_yet_recruiting)
• Mississippi Retina Associates — Jackson, Mississippi, United States (Recruiting)
• Duke Eye Center — Durham, North Carolina, United States (Not_yet_recruiting)
• Retina Consultants of Texas — Bellaire, Texas, United States (Recruiting)
• Retina Foundation of the Southwest — Dallas, Texas, United States (Recruiting)

Study contacts

• Study coordinator: Umair Qazi, MD, MPH

Conditions Stargardt Disease