ong-term follow-up gene therapy study for Achromatopsia (CNGB3)

Phase 1

• Conditions: Achromatopsia caused by mutations in the CNGB3 gene; MedDRA version: 19.1 Level: LLT Classification code 10000454 Term: Achromatopsia System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2016-003856-59-GB
• Lead Sponsor: MeiraGTX UK II Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-02-28
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 27

Inclusion Criteria

- Are able to give informed consent or assent, with or without the guidance of their parent/guardian where appropriate
- Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving intraocular administration of AAV2/8-hCARp.hCNGB3
- Are willing to adhere to the protocol and long-term follow-up
Are the trial subjects under 18? yes
Number of subjects for this age range: 9
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified