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Completed

NCT Number: NCT04671433

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

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

Completed

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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

UZ Gent, 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

  • Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
  • Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
  • Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery

Treatment and study plan

Genetic: AAV5-hRKp.RPGR

Biological

Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group

Other names: botaretigene sparoparvovec

Primary outcomes

  1. Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze

    Time frame: From Baseline to 52 Weeks

    Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze.

Secondary outcomes

  1. Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52

    Time frame: From Baseline to Week 52

    Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed.

  2. Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52

    Time frame: From Baseline to Week 52

    Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 will be assessed.

  3. Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52

    Time frame: From Baseline to Week 52

    Pointwise response in full visual field at Week 52 will be assessed.

  4. Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52

    Time frame: From Baseline to Week 52

    Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed.

  5. Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52

    Time frame: From Baseline to Week 52

    Pointwise response in the central 30 degrees visual field at Week 52 will be assessed.

  6. Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52

    Time frame: From Baseline to Week 52

    Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed.

  7. Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52

    Time frame: From Baseline to Week 52

    Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed.

  8. Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52

    Time frame: From Baseline to Week 52

    Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52.

  9. Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52

    Time frame: From Baseline to Week 52

    Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52.

  10. Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52

    Time frame: From Baseline to Week 52

    Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52.

  11. Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52

    Time frame: From Baseline to Week 52

    Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52.

  12. Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52

    Time frame: From Baseline to Week 52

    Change from baseline in visual function as assessed by low luminance visual acuity using the ETDRS chart letter score in worse-seeing eye at Week 52.

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

    Time frame: Day 1 - Week 52

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

  14. Number of Participants With Abnormalities in Laboratory Assessments

    Time frame: Day 1 - 52 Weeks

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

Sponsors and collaborators

Lead sponsor

Janssen Research & Development, LLC

Industry

Registry information

Official study title

Phase 3 Randomized, Controlled 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
2024
Study completion
2024
First posted
Dec 17, 2020
Registry last updated
Mar 16, 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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