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NCT04671433 (botaretigene sparoparvovec in X-linked retinitis pigmentosa): BVCT ex-ante prediction vs. reported outcome

On 3 Dec 2024, BioinvestGPT's BVCT predicted technical partial success and commercial partial success for this trial. The 5 May 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially insufficient.

Clinical Trial

NCTID
NCT04671433
Acronym
LUMEOS
Drug Name
botaretigene sparoparvovec (bota-vec)
Drug MoA
AAV5 gene therapy delivering RPGR transgene
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Eye Diseases
Indication
X-linked retinitis pigmentosa (XLRP)
Human Patients
97
Sponsor
Johnson & Johnson
Phase
Phase 3

Prediction of , index 1850

Prospective Prediction
Prediction Index
1850
Prediction Date
Commercial Prediction
partial SUCCESS (commercially maybe sufficient clinical benefit in VMA/BCVA superior to 4D-125; given that no gene therapy has been approved for XLRP and Elevidys's label expansion has been approved for DMD despite very weak efficacy data)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak clinical benefit in VMA/BCVA against placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
153 days in advance
Readout Data Interpretation
bota-vec failed to achieve statistically significant improvement in vision-guided mobility compared with placebo
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

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
Phase
Phase 3
Status
Completed
Enrollment
105
Lead sponsor
MeiraGTx Ocular UK Ltd
Conditions
X-Linked Retinitis Pigmentosa
Interventions
BIOLOGICAL: Genetic: AAV5-hRKp.RPGR; BIOLOGICAL: Genetic: AAV5-hRKp.RPGR
Design
Randomized · Parallel · Single
Primary purpose
Treatment
Start
2020-12-04
Primary completion
2024-09-30
Completion
2024-09-30
Primary outcomes
  • 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)

NCT04671433 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials