Vortosiran for VTE Prevention
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorRibocure Pharmaceuticals AB
About this trial
Blood clots remain a serious cause of illness and death. Long-term treatment and prevention of these clots with blood thinners is often limited by bleeding risk, drug interactions, and daily dosing, leading many patients to stop treatment early. Both cancer and chemotherapy further increase the risk of clot formation.
Vortosiran is an injectable therapy that lowers Factor XI (one of the body's natural clotting helpers) production in the liver, leading to a strong and long-lasting reduction in blood clotting activity with less risk of bleeding compared to current available treatments. Studies conducted so far have shown that vortosiran is safe and shows a dose-dependent Factor XI suppression (that is, a higher dose results in a higher level of suppression) that lasts for several months, supporting testing using a limited number of injections in patients who are at higher risk of blod clot formation.
This study aims to study how safe and effective vortosiran is in patients who have had blood clots in the past and in cancer patients who are at high risk of developing blood clots.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Adult male and female participants (≥18 years) meeting the specified below criteria for Cohort A.
Postmenopausal women are eligible without additional contraceptive measures.
Age ≥55 years with ≥12 months of spontaneous amenorrhoea, or
Age <55 years with ≥12 months of spontaneous amenorrhoea and follicle-stimulating hormone in the postmenopausal range, or surgical menopause (bilateral oophorectomy, with or without hysterectomy) documented in the medical history.
Disqualifiers
Women who are pregnant, currently breastfeeding, or planning pregnancy during the trial or within the defined post-treatment period are not eligible.
Positive hepatitis B surface antigen (HBsAg), or positive total hepatitis B core antibody (anti-HBc) with detectable HBV DNA;
Positive hepatitis C virus antibody (anti-HCV) with detectable HCV RNA; or
Reactive HIV-1/2 antigen/antibody test confirmed by a positive confirmatory test.
Trial design
Design model
Parallel
Treatments tested in this trial
Vortosiran
DrugVortosiran, active drug.
Placebo
DrugPlacebo that is identical in appearance of active IMP.
Treatment groups
150 Participants
are divided into 3 treatment groupsGroup A: Low-dose groupActive comparator 1 intervention
Group B: High-dose groupActive comparator 1 intervention
Group C: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Primary objective
To evaluate the effect of two dose levels of vortosiran on coagulation Factor XI (FXI) activity.
Time frame
At Week 82
Primary endpoint
Percentage change from baseline in FXI activity, compared with placebo.
Time frame
Baseline to Week 8.Secondary outcomes
1
Secondary objective
To evaluate the safety of vortosiran at two dose levels.
Time frame
Up to week 562
Secondary objective
To assess the plasma exposure of vortosiran after dosing
Time frame
0 [pre-dose] to 24 hours after dosing.3
Secondary objective
To evaluate the effect of 2 dose levels of vortosiran on FXI antigen concentration.
Time frame
At week 164
Secondary Objective
To evaluate the effect of 2 dose levels of vortosiran on specific blood coagulation test aPTT.
Time frame
At week 16Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Ribocure Pharmaceuticals AB
Lead sponsor
Fortrea
Collaborator
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: