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RECRUITING
NCT07313618
EARLY_PHASE1

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

Sponsor: West China Hospital

View on ClinicalTrials.gov

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.

Key Details

Gender

All

Age Range

5 Years - 12 Years

Study Type

INTERVENTIONAL

Enrollment

18

Start Date

2025-12-22

Completion Date

2030-12-31

Last Updated

2026-01-12

Healthy Volunteers

No

Interventions

GENETIC

JWK010 gene therapy

JWK010: AAV vector containing a coding sequence for tyrosinase.

Locations (1)

West China Hospital, Sichuan University

Chengdu, Sichuan, China