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

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)


Lead Sponsor: MeiraGTx UK II Ltd

Collaborator: Syne Qua Non Limited
Bionical Emas

Source MeiraGTx UK II Ltd
Brief Summary

A clinical trial of AAV2/5 vector for patients with X-linked retinitis pigmentosa (XLRP)

Detailed Description

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

Overall Status Active, not recruiting
Start Date July 14, 2017
Completion Date November 2020
Primary Completion Date November 2020
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Incidence of Adverse Events related to the sub retinal administration of AAV2-RPGR 18 months
Secondary Outcome
Measure Time Frame
Improvement in visual function 18 months
Improvement in retinal function 18 months
Improvement in Quality of Life 18 months
Enrollment 46

Intervention Type: Genetic

Intervention Name: AAV2/5-RPGR

Description: Open label, non-randomised, dose-escalation intervention followed by randomised dose confirmation against a control arm



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: Male

Minimum Age: 5 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
James Bainbridge, Prof Principal Investigator University College, London
Massachusetts Eye and Ear Institute | Boston, Massachusetts, 02114, United States
Kellogg Eye Center | Ann Arbor, Michigan, 48105, United States
UPMC Eye Center | Pittsburgh, Pennsylvania, 15213, United States
Leeds Teaching Hospitals NHS Trust | Leeds, LS9 7TF, United Kingdom
Moorfields Eye Hospital NHS Foundation Trust | London, United Kingdom
Location Countries

United Kingdom

United States

Verification Date

March 2020

Responsible Party

Type: Sponsor

Condition Browse
Number Of Arms 3
Arm Group

Label: Low dose AAV2/5-RPGR

Type: Experimental

Description: Single, subretinal administration of low dose AAV2/5-RPGR

Label: Intermediate dose AAV2/5-RPGR

Type: Experimental

Description: Single, subretinal administration of intermediate dose AAV2/5-RPGR

Label: High dose AAV2/5-RPGR

Type: Experimental

Description: Single, subretinal administration of high dose AAV2/5-RPGR

Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Single Group Assignment

Intervention Model Description: Biological

Primary Purpose: Treatment

Masking: None (Open Label)