NCT03278873

Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3

Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3 and AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 or CNGA3

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 34 (actual)
• Sponsor: MeiraGTx UK II Ltd · Industry
• Sex: All
• Age: 3 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia associated with defects in CNGB3 who participated in a clinical trial in which they received AAV-CNGB3 retinal gene therapy.

Detailed description

The follow-up study is designed to collect data on the longer-term safety and efficacy of AAV-CNGA3 retinal gene therapy and AAV-CNGB3 retinal gene therapy.

Conditions

• Achromatopsia

Interventions

Type	Name	Description
BIOLOGICAL	Prior exposure to AAV-CNGA3 or AAV-CNGB3	Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively.

Timeline

• Start date: 2017-06-29
• Primary completion: 2024-04-04
• Completion: 2024-04-04
• First posted: 2017-09-12
• Last updated: 2025-06-11
• Results posted: 2025-06-11

Locations

2 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study