Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

NCT04794101 | Active Not Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: MGT-RPGR-0222020-002255-372024-511411-25-00
• Study Type: interventional
• Target: 97
• Locations: 11 countries
• Sites: 25

Brief Summary

A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Conditions

X-Linked Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (4)

• Number of Participants with Ocular and Non-ocular Adverse Events

  Number of participants with ocular and non-ocular adverse events will be assessed.

  Day 1 - Month 60

• Number of Participants With Abnormalities in Laboratory Assessments

  Number of participants with abnormalities in laboratory assessments will be assessed.

  Day 1 - Month 60

• Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment

  Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.

  From Baseline - Month 60

• Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment

  Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.

  From Baseline- Month 60

Study Arms (3)

Deferred Treatment From MGT-RPGR-021 of Intermediate Dose

EXPERIMENTAL

Deferred treatment

Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose

Already Treated in MGT-RPGR-021

EXPERIMENTAL

Already treated

Biological: Genetic: AAV5-hRKp.RPGR Intermediate Dose; Biological: Genetic: AAV5-hRKp.RPGR Low Dose

Deferred Treatment From MGT-RPGR-021 Low Dose

EXPERIMENTAL

Deferred treatment

Biological: Genetic: AAV5-hRKp.RPGR Low Dose

Interventions

Genetic: AAV5-hRKp.RPGR Intermediate Dose BIOLOGICAL

Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Also known as: botaretigene sparoparvovec

Already Treated in MGT-RPGR-021; Deferred Treatment From MGT-RPGR-021 of Intermediate Dose

Genetic: AAV5-hRKp.RPGR Low Dose BIOLOGICAL

Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group

Also known as: botaretigene sparoparvovec

Already Treated in MGT-RPGR-021; Deferred Treatment From MGT-RPGR-021 Low Dose

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, 3 years of age or older, has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory.

You may not qualify if:

• None

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (25)

Shiley Eye Institute Jacobs Retina Center

La Jolla, California, 92093 0946, United States

Location

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Stanford Health Care

Palo Alto, California, 94303, United States

Location

VitreoRetinal Associates, PA

Gainesville, Florida, 32607, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, 02114, United States

Location

Univ of Michigan Medical Center

Ann Arbor, Michigan, 48105, United States

Location

Duke Eye Center

Durham, North Carolina, 27705, United States

Location

University of Pittsburgh Medical Center (UPMC)

Pittsburgh, Pennsylvania, 15213, United States

Location

Ghent University Hospital

Ghent, 9000, Belgium

Location

Hospital For Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Rigshospitalet Glostrup

Glostrup Municipality, 2600, Denmark

Location

Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts

Paris, 75012, France

Location

Hadassah Medical Center

Jerusalem, 91120, Israel

Location

Azienda Ospedaliero Universitaria Careggi

Florence, 50134, Italy

Location

Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli

Naples, 80131, Italy

Location

VUMC Amsterdam

Amsterdam, 1105AZ, Netherlands

Location

Radboudumc

Nijmegen, 6525EX, Netherlands

Location

Hosp Univ Fund Jimenez Diaz

Madrid, 28040, Spain

Location

University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical

Basel, 4031, Switzerland

Location

Universite de Lausanne, Hopital ophtalmique Jules-Gonin

Lausanne, 1004, Switzerland

Location

NHS Lothian

Edinburgh, EH3 9HA, United Kingdom

Location

Gartnavel General Hospital

Glasgow, G12 0YN, United Kingdom

Location

St James University Hospital

Leeds, LS9 7TF, United Kingdom

Location

Moorfields Eye Hospital

London, EC1V 2PD, United Kingdom

Location

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 97 participants randomized in MGT-RPGR-021. 64 participants randomized to Immediate treatment arm. 33 participants randomized to the deferred arm. These participants will be treated in MGT-RPGR-022. All participants to be followed for 60 months.

Study Record Dates

• First Submitted: December 15, 2020
• First Posted: March 11, 2021
• Study Start: December 4, 2020
• Primary Completion (Estimated): September 20, 2029
• Study Completion (Estimated): December 7, 2029
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1); US (9); CA (1); IT (2); FR (1); IL (1); NL (2); ES (1); DK (1); GB (4); CH (2)