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

Active, not recruiting

• Conditions: X-Linked Retinitis Pigmentosa
• Interventions: Biological: AAV5-hRKp.RPGR

• Registration Number: NCT04312672
• Lead Sponsor: Janssen Research & Development, LLC

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-07-31
• Study First Submitted: 2019-07-15
• Study First Submitted QC: 2020-03-12
• Study First Posted: 2020-03-18
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-08
• Last Update Posted: 2025-01-09
• Primary Completion: 2026-11-18
• Study Completion: 2026-11-18

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 42

Inclusion Criteria

• Received AAV5-hRKp.RPGR in the MGT009 Study

Read More

Exclusion Criteria

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

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Follow up cohort	AAV5-hRKp.RPGR	no intervention follow up study

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (5)

Massachusetts Eye and Ear Infirmary; 🇺🇸 Boston, Massachusetts, United States

University of Pittsburgh Medical Center; 🇺🇸 Pittsburgh, Pennsylvania, United States

Leeds Teaching Hospitals NHS Trust; 🇬🇧 Leeds, United Kingdom

Univ of Michigan Medical Center; 🇺🇸 Ann Arbor, Michigan, United States

Moorfields Eye Hospital; 🇬🇧 London, United Kingdom