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NCT04153149 (Vutrisiran Sterile Normal Saline in Transthyretin Amyloidosis With Cardiomyopathy): BVCT ex-ante prediction vs. reported outcome

On 22 Dec 2023, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 30 Aug 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially insufficient inferior to both Acoramidis and tafamidis.

Clinical Trial

NCTID
NCT04153149
Acronym
HELIOS-B
Drug Name
Vutrisiran Sterile Normal Saline (0.9% NaCl)
Drug MoA
anti-TTR siRNA
Drug Modality
small interfering RNA (siRNA) antisense oligonucleotide
Drug Class
First-In-Class
Therapeutic Area
Nutritional and Metabolic Diseases
Indication
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Human Patients
655
Sponsor
Alnylam Pharmaceuticals
Phase
Phase 3

Prediction of , index 1377

Prospective Prediction
Prediction Index
1377
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit inferior to both tafamidis and acoramidis)
Technical Prediction
FAILURE (statistically maybe significant yet weak clinical benefit for the composite endpoint)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
252 days in advance
Readout Data Interpretation
composite primary endpoint met (1.2% ARR and 13.8% RRR at month 30); inferior to 6.4% ARR and 25% RRR at month 30 p<0.0001 for Acoramidis in ATTRIBUTE-cm study; and inferior to 13.4% ARR and 31.2% RRR at month 30 p=0.0006 for tafamidis in ATTR-ACT study); secondary endpoint 6MWD met p<0.025 (vs p<0.0001 for both Acoramidis and tafamidis); all cause modality met with p<0.05.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially insufficient inferior to both Acoramidis and tafamidis
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Phase
Phase 3
Status
Active not recruiting
Enrollment
655
Lead sponsor
Alnylam Pharmaceuticals
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
DRUG: Vutrisiran; DRUG: Sterile Normal Saline (0.9% NaCl)
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2019-11-26
Primary completion
2024-05-08
Completion
2026-12-02
Results first posted
2025-10-21
Primary outcomes
  • Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population (Up to Month 36)
  • Composite Endpoint of All-Cause Mortality and Recurrent CV Events (CV Hospitalizations and Urgent HF Visits) in the Vutrisiran Monotherapy Subgroup (Up to Month 36)

NCT04153149 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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