Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) – Retinitis Pigmentosa GTPase Regulator (RPGR)

Overview

The Phase 1 part of the study is a dose escalation of subretinal administration of AAV2/5 vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR. The Phase 2 part of the study is a cohort expansion of subretinal administration of AAV2/5 vector to assess the safety and efficacy of this vector in participants with XLRP caused by mutations in RPGR.

Full Title of Study: “An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2-.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: November 18, 2021

Detailed Description

This is an open-label phase 1/2 dose-escalation and cohort expansion trial to determine the safety and efficacy of subretinal administration of AAV2/5 vector in participants with XLRP caused by mutations in RPGR.

Interventions

  • Genetic: AAV2/5-RPGR
    • Single, subretinal administration of AAV2/5-RPGR

Arms, Groups and Cohorts

  • Experimental: Phase 1 (Part 1, Dose Escalation)
    • Participants receive one of three doses of AAV2/5-RPGR
  • Experimental: Phase 2 (Part 2; Expansion)
    • Participants receive one of two doses of AAV2/5-RPGR

Clinical Trial Outcome Measures

Primary Measures

  • Incidence of Adverse Events related to the sub retinal administration of AAV2-RPGR
    • Time Frame: 18 months
    • Safety is defined as the absence of advanced therapy medicinal product (ATIMP)-related safety events

Secondary Measures

  • Improvement in visual function
    • Time Frame: 18 months
    • Improvements in visual function as assessed by ocular examination
  • Improvement in retinal function
    • Time Frame: 18 months
    • Improvements in retinal function as assessed by retinal assessement
  • Improvement in Quality of Life
    • Time Frame: 18 months
    • •Quality of life will be measured by QoL questionnaire

Participating in This Clinical Trial

Key inclusion Criteria:

  • Are aged 5 years or older male – Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI) Key exclusion Criteria:

• Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months

Gender Eligibility: Male

Minimum Age: 5 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • MeiraGTx UK II Ltd
  • Collaborator
    • Syne Qua Non Limited
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • James Bainbridge, Prof, Principal Investigator, University College, London

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