NCT07313618

Safety and Efficacy of a Single Suprachoroidal Injection of JWK010 Gene Therapy in Subjects With Oculocutaneous Albinism Type 1 (OCA1)

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: West China Hospital · Academic / Other
• Sex: All
• Age: 5 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

Oculocutaneous albinism (OCA) is the most common type of albinism. People with OCA have little or no pigment (melanin) in their eyes, skin, and hair. This often leads to symptoms such as sensitivity to light, crossed or misaligned eyes, reduced vision, and involuntary eye movements. OCA type 1 is caused by changes in the tyrosinase gene, which results in a lack or reduced function of the tyrosinase enzyme. This enzyme is essential for producing melanin, so people with OCA1 cannot make enough of it. JWK010 is a gene therapy product developed specifically for patients with OCA1. It is designed to help the cells produce functional tyrosinase protein, with the goal of restoring pigment in the retina and improving retinal structure and function.

Conditions

• Oculocutaneous Albinism (OCA)

Interventions

Type	Name	Description
GENETIC	JWK010 gene therapy	JWK010: AAV vector containing a coding sequence for tyrosinase.

Timeline

• Start date: 2025-12-22
• Primary completion: 2030-12-31
• Completion: 2030-12-31
• First posted: 2026-01-02
• Last updated: 2026-01-12

Locations

1 site across 1 country: China