Randomized, Double blind, Phase II Trial of Vortosiran to Evaluate Safety, Tolerability, and Pharmacodynamic Efficacy in Participants with Prior Venous Thromboembolism After Completion of Initial Anticoagulant Therapy (Cohort A) and in Cancer Patients at High Risk of Venous Thromboembolism (Cohort B)

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2026-525479-15-00

Enrollment

Registered

2026-09-04

Start date

Unknown

Completion date

Unknown

Last updated

2026-09-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Venous Thromboembolism

Brief summary

Percentage change from baseline in FXI activity, compared with placebo at Week 8.

Detailed description

Frequency, intensity, and seriousness of AEs during the trial, as well as clinically significant changes in laboratory parameters, vital signs, physical examinations, and 12-lead ECGs, Plasma concentrations of vortosiran, summarized with descriptive statistics by dose and sampling timepoint, Change from baseline and percentage change from baseline in FXI activity after dosing of vortosiran, compared with placebo, over the trial period, Change from baseline and percentage change from baseline in aPTT, PT/INR and FXI antigen levels after dosing of vortosiran, compared with placebo, over the trial period.

Interventions

DRUGThe corresponding placebo consist of the 25 mm phosphate buffer solution with 2 µg of vitamin b2 added for colouring purposes only

DRUGRBD4059 injection

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Percentage change from baseline in FXI activity, compared with placebo at Week 8.	—

Secondary

Measure	Time frame
Frequency, intensity, and seriousness of AEs during the trial, as well as clinically significant changes in laboratory parameters, vital signs, physical examinations, and 12-lead ECGs, Plasma concentrations of vortosiran, summarized with descriptive statistics by dose and sampling timepoint, Change from baseline and percentage change from baseline in FXI activity after dosing of vortosiran, compared with placebo, over the trial period, Change from baseline and percentage change from baseline in aPTT, PT/INR and FXI antigen levels after dosing of vortosiran, compared with placebo, over the trial period.	—

Measure

Time frame

—

Outcome results

None listed

Source: EU CTIS · Data processed: Sep 5, 2026