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NCT Number: NCT04794101

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

X-Linked Retinitis Pigmentosa

Age range

3 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Ghent University Hospital, Ghent, Belgium

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Genetic: AAV5-hRKp.RPGR Intermediate Dose

Biological

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

Other names: botaretigene sparoparvovec

Genetic: AAV5-hRKp.RPGR Low Dose

Biological

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

Other names: botaretigene sparoparvovec

Primary outcomes

  1. Number of Participants with Ocular and Non-ocular Adverse Events

    Time frame: Day 1 - Month 60

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

  2. Number of Participants With Abnormalities in Laboratory Assessments

    Time frame: Day 1 - Month 60

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

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

    Time frame: From Baseline - Month 60

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

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

    Time frame: From Baseline- Month 60

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

Sponsors and collaborators

Lead sponsor

Janssen Research & Development, LLC

Industry

Registry information

Official study title

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

Important dates

Study start
2020
Primary completion
2029
Study completion
2029
First posted
Mar 11, 2021
Registry last updated
Jul 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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