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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04671433
Recruitment Status : Active, not recruiting
First Posted : December 17, 2020
Last Update Posted : April 24, 2024
Sponsor:
Information provided by (Responsible Party):
Janssen Research & Development, LLC

Study Description
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Brief Summary:
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Condition or disease Intervention/treatment Phase
X-Linked Retinitis Pigmentosa Biological: Genetic: AAV5-hRKp.RPGR Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 97 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
Masking: Single (Outcomes Assessor)
Masking Description: No intervention - deferred treatment group (Bilateral, subretinal administration of AAV5-RPGR to be administered in the follow-up study)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : December 4, 2020
Estimated Primary Completion Date : September 20, 2024
Estimated Study Completion Date : September 20, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Experimental - Immediate Treatment
Intermediate dose.
 Biological: Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Other Name: botaretigene sparoparvovec
Deferred Treatment
Deferred Treatment
 Biological: Genetic: AAV5-hRKp.RPGR
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)
Experimental: Experimental Immediate Treatment
Low dose.
 Biological: Genetic: AAV5-hRKp.RPGR
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Other Name: botaretigene sparoparvovec


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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.


Eligibility Criteria
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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04671433


Locations
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Sponsors and Collaborators
Janssen Research & Development, LLC
Investigators
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Study Director: Janssen Research & Development, LLC Clinical Trial Janssen Research & Development, LLC
More Information
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Responsible Party: Janssen Research & Development, LLC
ClinicalTrials.gov Identifier: NCT04671433    
Other Study ID Numbers: CR109258
MGT-RPGR-021 ( Other Identifier: Janssen Research & Development, LLC )
2020-002873-88 ( EudraCT Number )
First Posted: December 17, 2020    Key Record Dates
Last Update Posted: April 24, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn