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

NCT ID: NCT04312672

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

42 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-07-31

Study Completion Date

2026-11-18

Brief Summary

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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

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Conditions

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X-Linked Retinitis Pigmentosa

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Follow up cohort

no intervention follow up study

AAV5-hRKp.RPGR

Intervention Type BIOLOGICAL

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

Interventions

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AAV5-hRKp.RPGR

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Received AAV5-hRKp.RPGR in the MGT009 Study

Exclusion Criteria

* Unwilling or unable to meet with the requirements of the study
Minimum Eligible Age

5 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Janssen Research & Development, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Janssen Research & Development, LLC Clinical Trial

Role: STUDY_DIRECTOR

Janssen Research & Development, LLC

Locations

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Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, United States

Site Status

Univ of Michigan Medical Center

Ann Arbor, Michigan, United States

Site Status

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status

Leeds Teaching Hospitals NHS Trust

Leeds, , United Kingdom

Site Status

Moorfields Eye Hospital

London, , United Kingdom

Site Status

Countries

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United States United Kingdom

Other Identifiers

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MGT010

Identifier Type: OTHER

Identifier Source: secondary_id

2018-000425-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MGT010

Identifier Type: -

Identifier Source: org_study_id