Title

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
  • Phase

    Phase 1/Phase 2
  • Study Type

    Interventional
  • Status

    Active, not recruiting
  • Study Participants

    34
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.
The follow up study is designed to collect data on longer-term safety and efficacy.
Study Started
Jun 29
2017
Primary Completion
Jan 15
2026
Anticipated
Study Completion
Jan 15
2026
Anticipated
Last Update
Jan 14
2022

Biological either AAV - CNGB3 or AAV - CNGA3

Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3

Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3 Experimental

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

Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3 Experimental

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

Biological-high dose of either AAV - CNGB3 or AAV - CNGA3 Experimental

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

Criteria

Inclusion in the study will be limited to individuals who:

Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s) where appropriate
Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)
Are willing to adhere to the protocol and long-term follow-up

Individuals will be excluded who:

Are unwilling or unable to meet with the requirements of the study
No Results Posted