Gene Therapy Trial for People with Achromatopsia (unable to see colours) due to a gene defect

Phase 1

• Conditions: Therapeutic area: Diseases [C] - Eye Diseases [C11]; Achromatopsia caused by mutations in the CNGB3 gene; MedDRA version: 20.0 Level: LLT Classification code 10000454 Term: Achromatopsia System Organ Class: 100000004850

• Registration Number: EUCTR2016-002290-35-GB
• Lead Sponsor: MeiraGTx UK II Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-11-17
• Last Update Posted: 16 December 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 27

Inclusion Criteria

Inclusion in the trial will be limited to individuals who:
• Are aged 3 years or older (children will be included only once the maximal tolerated dose has been determined)
• Have Achromatopsia caused by mutations in CNGB3
• Present evidence of preservation of photoreceptors at the macula
• Are able to undertake age-appropriate clinical assessments
• Are willing to give consent for the use of blood and blood components collected throughout the trial for the investigation of immune response to ATIMP

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 who:
• Are females who are pregnant or breastfeeding
• Had intra-ocular surgery within 6 months of screening
• Have an ocular or systemic disorder that may preclude subretinal surgery and/or interfere with interpretation of the study results.
• Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months
• Have any other condition that the PI considers makes them inappropriate for entry into the trial, inclusive of but not limited to a history of the following: hypertension, diabetes mellitus, tuberculosis, renal impairment, immunocompromised
state, osteoporosis, gastric ulceration or severe affective disorder
• Are unwilling to consider the possibility of entry into a subsequent longer term follow up study

Study & Design

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