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

X-Linked Retinitis Pigmentosa Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03252847
• Other study ID #: MGT009
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: July 14, 2017
• Est. completion date: November 18, 2021

Study information

• Verified date: November 2022
• Source: MeiraGTx UK II Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 49
• Est. completion date: November 18, 2021
• Est. primary completion date: November 18, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa
• X-Linked Retinitis Pigmentosa

Intervention

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

Locations

Country	Name	City	State
United Kingdom	Leeds Teaching Hospitals NHS Trust	Leeds
United Kingdom	Moorfields Eye Hospital NHS Foundation Trust	London
United States	Kellogg Eye Center	Ann Arbor	Michigan
United States	Massachusetts Eye and Ear Institute	Boston	Massachusetts
United States	UPMC Eye Center	Pittsburgh	Pennsylvania

Sponsors (3)

Lead Sponsor	Collaborator
MeiraGTx UK II Ltd	Bionical Emas, Syne Qua Non Limited

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Adverse Events related to the sub retinal administration of AAV2-RPGR	Safety is defined as the absence of advanced therapy medicinal product (ATIMP)-related safety events	18 months
Secondary	Improvement in visual function	Improvements in visual function as assessed by ocular examination	18 months
Secondary	Improvement in retinal function	Improvements in retinal function as assessed by retinal assessement	18 months
Secondary	Improvement in Quality of Life	•Quality of life will be measured by QoL questionnaire	18 months