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

Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04794101

Enrollment

Registered

2021-03-11

Start date

2020-12-04

Completion date

2029-12-07

Last updated

2026-08-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

X-Linked Retinitis Pigmentosa

Brief summary

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

Interventions

BIOLOGICALGenetic: AAV5-hRKp.RPGR Intermediate Dose

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

BIOLOGICALGenetic: AAV5-hRKp.RPGR Low Dose

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

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

SINGLE (Outcomes Assessor)

Intervention model description

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.

Eligibility

Sex/Gender

ALL

Age

3 Years to No maximum

Healthy volunteers

Inclusion criteria

* 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.

Exclusion criteria

* None

Design outcomes

Primary

Measure	Time frame	Description
Number of Participants with Ocular and Non-ocular Adverse Events	Day 1 - Month 60	Number of participants with ocular and non-ocular adverse events will be assessed.
Number of Participants With Abnormalities in Laboratory Assessments	Day 1 - Month 60	Number of participants with abnormalities in laboratory assessments will be assessed.
Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment	From Baseline - Month 60	Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment	From Baseline- Month 60	Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.

Countries

Belgium, Canada, Denmark, France, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom, United States

Contacts

STUDY_DIRECTORJanssen Research & Development, LLC Clinical Trial

Janssen Research & Development, LLC

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Aug 29, 2026