Gene Therapy Trial for LCA2 OPTIRPE65

Phase 1

• Conditions: eber Congenital Amaurosis (LCA) caused by mutations in RPE65; MedDRA version: 20.0Level: PTClassification code 10070667Term: Leber's congenital amaurosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2015-003418-25-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: 2016-01-04
• Last Update Posted: 27 July 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 27

Inclusion Criteria

Inclusion in the trial will be limited to individuals who:
• Are aged 3 years or older
• Have early-onset severe retinal dystrophy consistent with RPE65 deficiency
• Have homozygous or compound heterozygous missense or null mutations in RPE65 confirmed in accredited laboratory
• Have functional or structural evidence of photoreceptor preservation as assessed by static perimetry (for visual field assessment) and SD-OCT scanning respectively
• Are able to give informed consent or assent, with the guidance of their parent/guardian where appropriate
• Are able to undertake age-appropriate clinical assessments
• If female and of child bearing potential, are willing to use an effective form of birth control (hormonal or barrier method of birth control; or abstinence) for at least 12 months following ATIMP administration
• If male, are willing to use barrier and spermicide form of contraceptive or maintain sexual abstinence for at least 12 months following ATIMP administration
• Females of childbearing potential will have a negative pregnancy test on the day of ATIMP administration. Participants are considered not of childbearing potential if they are surgically sterile (i.e. they have undergone a hysterectomy or bilateral oophorectomy) or post-menopausal
• Are willing to give consent for the use of blood and blood components collected throughout the trial for the investigation of immune responses to the 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 9
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

Individuals will be excluded who:
• Are females who are pregnant or breastfeeding
• Have contraindications for transient immune-suppression by systemic corticosteroids (including uncontrolled hypertension, diabetes mellitus, tuberculosis, renal impairment, osteoporosis, gastric ulceration, severe affective disorder) or are immunocompromised
• Have a previous history (within 5 years) of gastric or duodenal ulceration, hiatus hernia, uncontrolled gastro-oesphageal reflux or are using non-steroidal anti-inflammatory drugs on a regular basis at the time of screening
• Have a known allergy to any of the non-investigational drugs to be used in the trial
• Have participated in another research study involving an investigational medicinal 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
• Have had intraocular 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
• 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