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NCT03566043 (RGX-121 in 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
NCT03566043
Acronym
CAMPSIITE
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
Mucopolysaccharidosis Type II (Hunter Syndrome)
Human Patients
48
Sponsor
REGENXBIO Inc
Phase
Phase 2 Phase 3

Prediction of , index 1155

Prospective Prediction
Prediction Index
1155
Prediction Date
Commercial Prediction
partial SUCCESS (commercially sufficient clinical benefit due to no effective approved treatment)
Technical Prediction
partial SUCCESS (statistically maybe significant yet small clinical benefit compared with placebo)

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 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
Phase
Phase 2, Phase 3
Status
Active not recruiting
Enrollment
48
Lead sponsor
REGENXBIO Inc.
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
GENETIC: RGX-121
Design
Non randomized · Parallel · None
Primary purpose
Treatment
Start
2018-09-27
Primary completion
2023-11-27
Completion
2025-08
Primary outcomes
  • Part 1 Safety (24 Weeks)
  • Part 2 Biomarkers (52 Weeks)
  • Part 2 Biomarkers (104 weeks)
  • Part 2 Neurodevelopmental parameters (52 Weeks)
  • Part 2 Neurodevelopmental parameters (104 weeks)

NCT03566043 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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