Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06679946 | A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy | PHASE3 | ENROLLING BY_INVITATION | 700 | — | — | Dec 3, 2024 | Mar 31, 2028 | Feb 20, 2026 | 87 | United States, Argentina +15 |
| NCT04153149 | HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy | PHASE3 | ACTIVE NOT_RECRUITING | 655 | — | — | Nov 26, 2019 | Dec 2, 2026 | Jan 12, 2026 | 119 | United States, Argentina +30 |
An AE is any untoward medical occurrence in a patient or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) were compared between treatment groups using a modified Andersen-Gill model with a robust variance. All-cause mortality included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits. The number of participants with at least one CV event or all-cause mortality event have been reported here. No imputation was done for participants who dropped out early for the primary analysis of this composite outcome endpoint.
All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) were compared between treatment groups using a modified Andersen-Gill model with a robust variance. All-cause mortality included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits. The number of participants with at least one CV event or all-cause mortality event have been reported here. No imputation was done for participants who dropped out early for the primary analysis of this composite outcome endpoint.
| Arm | Type | Description |
|---|---|---|
| Vutrisiran 25 mg | EXPERIMENTAL | Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M). |
| Placebo | PLACEBO_COMPARATOR | Participants received vutrisiran matching placebo administered SC q3M during the 36-month DB period. After the DB period, participants receive vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the OLE period. |
| Name | Type | Description |
|---|---|---|
| Vutrisiran | DRUG | Vutrisiran administered SC q3M |
| Sterile Normal Saline (0.9% NaCl) | DRUG | Sterile normal saline (0.9% NaCl) will be administered by SC injection. |
Inclusion Criteria: * Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003 Exclusion Criteria: * Has permanently discontinued study drug administration while parti...