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NCT05629559 (4D-310 in Fabry Disease): BVCT ex-ante prediction vs. reported outcome

On 2 Dec 2023, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 12 Feb 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: more likely to be statistically insignificant and commercially insufficient.

Clinical Trial

NCTID
NCT05629559
Acronym
INGLAXA
Drug Name
4D-310
Drug MoA
AAV GLA gene therapy
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Fabry Disease
Human Patients
18
Sponsor
4D Molecular Therapeutics
Phase
Phase 1 Phase 2

Prediction of , index 1006

Prospective Prediction
Prediction Index
1006
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit)
Technical Prediction
FAILURE (statistically insignificant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
72 days in advance
Readout Data Interpretation
weak efficacy in cardiac endpoints (minimal clinically important difference (MCID) no key renal endpoints with previous clinical hold due to toxicity.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
more likely to be 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
An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement
Phase
Phase 1, Phase 2
Status
Recruiting
Enrollment
18
Lead sponsor
4D Molecular Therapeutics
Conditions
Fabry Disease
Interventions
BIOLOGICAL: 4D-310
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2022-10-16
Primary completion
2026-06
Completion
2030-06
Primary outcomes
  • Incidence and severity of adverse events (1 year)

NCT05629559 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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