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

Primary Purpose

X-Linked Retinitis Pigmentosa

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
AAV2/5-RPGR
Sponsored by
MeiraGTx UK II Ltd
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for X-Linked Retinitis Pigmentosa focused on measuring XLRP RPGR

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Massachusetts Eye and Ear Institute
  • Kellogg Eye Center
  • UPMC Eye Center
  • Leeds Teaching Hospitals NHS Trust
  • Moorfields Eye Hospital NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Phase 1 (Part 1, Dose Escalation)

Phase 2 (Part 2; Expansion)

Arm Description

Participants receive one of three doses of AAV2/5-RPGR

Participants receive one of two doses of AAV2/5-RPGR

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
August 1, 2017
Last Updated
December 2, 2022
Sponsor
MeiraGTx UK II Ltd
Collaborators
Syne Qua Non Limited, Bionical Emas
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1. Study Identification

Unique Protocol Identification Number
NCT03252847
Brief Title
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
July 14, 2017 (Actual)
Primary Completion Date
November 18, 2021 (Actual)
Study Completion Date
November 18, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MeiraGTx UK II Ltd
Collaborators
Syne Qua Non Limited, Bionical Emas

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
X-Linked Retinitis Pigmentosa
Keywords
XLRP RPGR

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Biological
Masking
None (Open Label)
Allocation
Randomized
Enrollment
49 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Phase 1 (Part 1, Dose Escalation)
Arm Type
Experimental
Arm Description
Participants receive one of three doses of AAV2/5-RPGR
Arm Title
Phase 2 (Part 2; Expansion)
Arm Type
Experimental
Arm Description
Participants receive one of two doses of AAV2/5-RPGR
Intervention Type
Genetic
Intervention Name(s)
AAV2/5-RPGR
Intervention Description
Single, subretinal administration of AAV2/5-RPGR
Primary Outcome Measure Information:
Title
Incidence of Adverse Events related to the sub retinal administration of AAV2-RPGR
Description
Safety is defined as the absence of advanced therapy medicinal product (ATIMP)-related safety events
Time Frame
18 months
Secondary Outcome Measure Information:
Title
Improvement in visual function
Description
Improvements in visual function as assessed by ocular examination
Time Frame
18 months
Title
Improvement in retinal function
Description
Improvements in retinal function as assessed by retinal assessement
Time Frame
18 months
Title
Improvement in Quality of Life
Description
•Quality of life will be measured by QoL questionnaire
Time Frame
18 months

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James Bainbridge, Prof
Organizational Affiliation
University College, London
Official's Role
Principal Investigator
Facility Information:
Facility Name
Massachusetts Eye and Ear Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Kellogg Eye Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Facility Name
UPMC Eye Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Leeds Teaching Hospitals NHS Trust
City
Leeds
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Facility Name
Moorfields Eye Hospital NHS Foundation Trust
City
London
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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