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NCT04046224 (ST920 in Fabry Disease): BVCT ex-ante prediction vs. reported outcome

On 19 Jan 2025, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 24 Jun 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically maybe insignificant and commercially TBD.

Clinical Trial

NCTID
NCT04046224
Acronym
STAAR
Drug Name
ST920
Drug MoA
AAV2/6-delivered GLA gene therapy
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Fabry Disease
Human Patients
33
Sponsor
Sangamo Therapeutics
Phase
Phase 1 Phase 2

Prediction of , index 1875

Prospective Prediction
Prediction Index
1875
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in eGFR inferior to pegunigalsidase alfa)
Technical Prediction
FAILURE (statistically maybe significant yet very weak clinical benefit in eGFR compared with placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
156 days in advance
Readout Data Interpretation
ST920 achieved a positive yet statistically insignificant eGFR slope at week 52
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically maybe insignificant and commercially TBD
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, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease (STAAR)
Phase
Phase 1, Phase 2
Status
Completed
Enrollment
36
Lead sponsor
Sangamo Therapeutics
Conditions
Fabry Disease
Interventions
BIOLOGICAL: ST-920
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2019-07-23
Primary completion
2025-04-10
Completion
2025-04-10
Results first posted
2026-04-14
Primary outcomes
  • Incidence of Treatment-emergent Adverse Events (TEAEs) - All (Up to 12 months after the ST-920 infusion)
  • Incidence of Treatment-emergent Adverse Events (TEAEs) - Related to ST-920 (Up to 12 months post ST-920 infusion)
  • Incidence of Treatment-emergent Adverse Events (TEAEs) - Serious (Up to 12 month post ST-920 infusion)
  • Incidence of Treatment-emergent Adverse Events (TEAEs) - Any TEAEs Leading to Study Discontinuation or Withdrawal (Up to 12 month post ST-920 infusion)

NCT04046224 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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