NCT04312672

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
42

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jul 2017

Longer than P75 for all trials

Geographic Reach
2 countries

5 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 31, 2017

Completed
2 years until next milestone

First Submitted

Initial submission to the registry

July 15, 2019

Completed
8 months until next milestone

First Posted

Study publicly available on registry

March 18, 2020

Completed
5.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 17, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 17, 2025

Completed
Last Updated

January 21, 2026

Status Verified

January 1, 2026

Enrollment Period

8.1 years

First QC Date

July 15, 2019

Last Update Submit

January 16, 2026

Conditions

X-Linked Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (8)

  • Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)

    Up to 60 Months

  • Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)

    Up to 60 Months

  • Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score

    Up to 60 Months

  • Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions

    Up to 60 Months

  • Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry

    Up to 60 months

  • +3 more secondary outcomes

Study Arms (1)

Follow up cohort

no intervention follow up study

Biological: AAV5-hRKp.RPGR

Interventions

AAV5-hRKp.RPGRBIOLOGICAL

Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up.

Follow up cohort

Eligibility Criteria

Age5 Years+
Sexmale(Gender-based eligibility)
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Males receiving AAV5-hRKp.RPGR in the MGT009 study

You may qualify if:

  • Received AAV5-hRKp.RPGR in the MGT009 Study

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, 02114, United States

Location

Univ of Michigan Medical Center

Ann Arbor, Michigan, 48105, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Leeds Teaching Hospitals NHS Trust

Leeds, LS9 7TF, United Kingdom

Location

Moorfields Eye Hospital

London, EC1V 9EL, United Kingdom

Location

Study Officials

  • Janssen Research & Development, LLC Clinical Trial

    Janssen Research & Development, LLC

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 15, 2019

First Posted

March 18, 2020

Study Start

July 31, 2017

Primary Completion

September 17, 2025

Study Completion

September 17, 2025

Last Updated

January 21, 2026

Record last verified: 2026-01

Locations

GB(2)US(3)