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NCT05139316 (DTX401 Placebo Oral corticosteroids… in Glycogen Storage Disease Type IA): BVCT ex-ante prediction vs. reported outcome

On 3 Jan 2024, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. The 30 May 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially sufficient (no drug approved for this indication).

Clinical Trial

NCTID
NCT05139316
Acronym
GlucoGene
Drug Name
DTX401 Placebo Oral corticosteroids Placebo for oral corticosteroids
Drug MoA
intravenous AAV8 G6PC gene therapy
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Congenital; Hereditary; and Neonatal Diseases and Abnormalities
Indication
Glycogen Storage Disease Type IA (GSDIa)
Human Patients
46
Sponsor
Ultragenyx Pharmaceutical
Phase
Phase 3

Prediction of , index 1041

Prospective Prediction
Prediction Index
1041
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient clinical benefit)
Technical Prediction
SUCCESS (statistically significant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
148 days in advance
Readout Data Interpretation
31% placebo-controlled reduction in daily cornstarch intake.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially sufficient (no drug approved for this indication)
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia
Phase
Phase 3
Status
Completed
Enrollment
49
Lead sponsor
Ultragenyx Pharmaceutical Inc
Conditions
Glycogen Storage Disease Type IA
Interventions
GENETIC: DTX401; OTHER: Placebo; DRUG: Oral prednisolone; DRUG: Placebo for oral prednisolone
Design
Randomized · Crossover · Quadruple
Primary purpose
Treatment
Start
2021-11-08
Primary completion
2024-02-20
Completion
2026-02-20
Primary outcomes
  • Percent Change from Baseline to Week 48 in Daily Cornstarch Intake (Baseline, Week 48)

NCT05139316 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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