Gene Therapy for Color Blindness

Recruiting in Palo Alto (17 mi)

+1 other location

Age: Any Age

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: MeiraGTx UK II Ltd

No Placebo Group

Trial Summary

What is the purpose of this trial?This study is a longer-term follow-up study for patients who participated in one of the clinical trials: AAV - CNGB3 retinal gene therapy for patients with achromatopsia, or AAV - CNGA3 retinal gene therapy for patients with achromatopsia.

Eligibility Criteria

This trial is for individuals who have a type of color blindness called achromatopsia and were previously treated with gene therapies AAV - CNGB3 or AAV - CNGA3. Participants must be able to consent, follow the study's long-term procedures, and have received one of these treatments in earlier Phase I/II trials.

Inclusion Criteria

I received a specific gene therapy in my eye as part of a previous study.

Participant Groups

The study is observing the long-term effects of two gene therapy interventions: AAV - CNGB3 and AAV - CNGA3. These therapies were administered in previous clinical trials to treat achromatopsia, a form of color blindness.

3Treatment groups

Experimental Treatment

Group I: Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3Experimental Treatment1 Intervention

Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3

Group II: Biological-high dose of either AAV - CNGB3 or AAV - CNGA3Experimental Treatment1 Intervention

Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3

Group III: Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3Experimental Treatment1 Intervention

Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3