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Genetic: AAV5-hRKp.RPGR

Phase 3

X-Linked Retinitis Pigmentosa | Monoclonal antibody | Ophthalmology |Johnson & Johnson|Last Updated: Mar 16, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment105
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04671433Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GenePHASE3 COMPLETED 105 — — Dec 4, 2020Sep 30, 2024Mar 16, 202628 United States, Belgium +9
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Study Endpoints
Primary Endpoints
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
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 Endpoints
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
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
From Baseline to Week 52
Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
From Baseline to Week 52
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Experimental - Immediate TreatmentEXPERIMENTALIntermediate dose.
Deferred TreatmentOTHERDeferred Treatment
Experimental Immediate TreatmentEXPERIMENTALLow dose.
Interventions
NameTypeDescription
Genetic: AAV5-hRKp.RPGRBIOLOGICALBilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
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Eligibility Criteria
Age Range3 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites28

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 anti...

Countries:United StatesBelgiumCanadaDenmarkFranceIsraelItalyNetherlandsSpainSwitzerlandUnited Kingdom
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Recent Changes (Last 90 Days)
MEDIUMMay 21, 2026NCT04671433TRIAL_REMOVED: changed
MEDIUMMay 21, 2026NCT04671433TRIAL_REMOVED: changed