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NCT04571970 (RGX-121 in paediatric Mucopolysaccharidosis Type II): BVCT ex-ante prediction vs. reported outcome

On 9 Jul 2023, BioinvestGPT's BVCT predicted technical partial success and commercial partial success for this trial. The 9 Feb 2026 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially insufficient (small clinical benefit).

Clinical Trial

NCTID
NCT04571970
Drug Name
RGX-121
Drug MoA
gene therapy that delivers IDS
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
paediatric Mucopolysaccharidosis Type II (Hunter Syndrome)
Human Patients
6
Sponsor
REGENXBIO Inc
Phase
Phase 1 Phase 2

Prediction of , index 1154

Prospective Prediction
Prediction Index
1154
Prediction Date
Commercial Prediction
partial SUCCESS (commercially sufficient clinical benefit due to no effective approved treatment)
Technical Prediction
partial SUCCESS (commercially sufficient clinical benefit due to no effective approved treatment)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
946 days in advance
Readout Data Interpretation
FDA issued a CRL for RGX-121 indicating that the clinical benefit is small and insufficient
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially insufficient (small clinical benefit)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Children 5 Years of Age and Older With MPS II (Hunter Syndrome)
Phase
Phase 1, Phase 2
Status
Completed
Enrollment
6
Lead sponsor
REGENXBIO Inc.
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
GENETIC: RGX-121
Design
Single group · None
Primary purpose
Treatment
Start
2021-03-11
Primary completion
2023-05-09
Completion
2024-05-23
Primary outcomes
  • Number of participants with treatment-related adverse events and serious adverse events (24 Weeks)

NCT04571970 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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