Long-term Follow-up Gene Therapy Study for RPGR- XLRP

Long-term Follow-up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Study Overview

• Status: Active, not recruiting
• Conditions: X-Linked Retinitis Pigmentosa
• Intervention / Treatment: Biological: AAV5-hRKp.RPGR

• Study Type: Observational
• Enrollment (Actual): 55

Contacts and Locations

• Study Contact: Name: Ocular Information; Phone Number: +44203866 4320; Email: ocularinfo@meiragtx.com

Study Locations

• United Kingdom
  • Leeds, United Kingdom, LS9 7TF
    • Leeds Teaching Hospitals NHS Trust
  • London, United Kingdom, EC1V 9EL
    • Moorfields Eye Hospital
• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts Eye and Ear Infirmary
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Univ of Michigan Medical Center
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • University of Pittsburgh Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Males receiving AAV5-hRKp.RPGR in the MGT009 study

Description

Inclusion Criteria:

• Received AAV5-hRKp.RPGR in the MGT009 Study

Exclusion Criteria:

• Unwilling or unable to meet with the requirements of the study

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Follow up cohort; no intervention follow up study	Biological: AAV5-hRKp.RPGR; Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants	Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported.	60 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)	Change in functional vision of walk time after treatment administration in VMA (Version 1.0) will be reported.	Up to 60 Months
Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)	Change in functional vision of LLQ domain scores in PRO will be reported. The instrument is scored by domain, computed by scaling individual items from 0 to 100 and then averaging the individual items for each domain. A higher score reflects higher functional level.	Up to 60 Months
Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score	The longer-term efficacy of AAV5-hRKp.RPGR in change in BCVA using ETDRS chart letter score will be reported.	Up to 60 Months
Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions	Change in LLVA as assessed by ETDRS chart under low luminance conditions will be reported.	Up to 60 Months
Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry	Change in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported.	Up to 60 months
Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field	Change in retinal sensitivity by pointwise comparison of data in static perimetry within the full visual field will be reported.	Up to 60 months
Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field	Change in retinal sensitivity by pointwise comparison of data in static perimetry within the Central 30 Degrees visual field will be reported.	Up to 60 months
Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry	Change in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be reported.	Up to 60 months

Collaborators and Investigators

• Sponsor: Janssen Research & Development, LLC
• Investigators: Study Director: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Study record dates

Study Major Dates

• Study Start (Actual): July 31, 2017
• Primary Completion (Estimated): November 18, 2026
• Study Completion (Estimated): November 18, 2026

Study Registration Dates

• First Submitted: July 15, 2019
• First Submitted That Met QC Criteria: March 12, 2020
• First Posted (Actual): March 18, 2020

Study Record Updates

• Last Update Posted (Actual): March 27, 2024
• Last Update Submitted That Met QC Criteria: March 25, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Retinal Dystrophies; Retinitis; Retinitis Pigmentosa
• Other Study ID Numbers: MGT010 (Other Identifier: Janssen Research & Development, LLC); 2018-000425-31 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No