Study to assess the safety, tolerability, and effects of vortosiran in two groups: participants who previously had a blood clot and completed initial blood-thinner treatment, and participants with cancer who are at high risk of blood clots

Ki vehet részt

• Cohort A and B: Adult male and female participants (≥18 years)
• Cohort B: Initiation of a new course of systemic chemotherapy with a minimum intended duration of ≥3 months
• Cohort B: Intermediate–high risk of VTE, defined as a Khorana score ≥2 at screening; 2.Cohort A and B: Women of childbearing potential must agree to use highly effective contraception; 3.Cohort A: Objectively confirmed symptomatic index DVT of the lower limbs at the popliteal vein or more proximal, and/or symptomatic PE, documented by objective imaging modalities
• Cohort A: Completed 3 to 12 months of standard therapeutic anticoagulant therapy for the index VTE episode at least 2 weeks before randomization
• Cohort A: No recurrent VTE during the initial 3 to 12 months anticoagulant therapy treatment period
• Cohort A: Clinical equipoise for extended anticoagulation, and discontinuation of anticoagulation is consistent with local standard of care for this patient profile. Specifically, clinical equipoise for extended anticoagulation
• Cohort A: Hemodynamically stable at screening and, in the investigator’s judgment, suitable for outpatient follow-up
• Cohort B: Eastern Cooperative Oncology Group performance status 0-2 during screening
• Cohort B: Newly diagnosed cancer site or progression of malignant disease

Ki nem vehet részt

• Cohort B: Planned stem cell transplantation
• Cohort A and B: Planned surgery that may change VTE/bleeding risk during the trial period and no recent surgery (≤2 weeks prior screening)
• Cohort A: Concomitant antithrombotic therapy that would confound assessment or unacceptably increase bleeding risk
• Cohort A: Active clinically significant bleeding or conditions associated with unacceptably high bleeding risk
• Cohort A: Severe uncontrolled hypertension
• Cohort B: High bleeding risk
• Cohort B: Hepatic disease with coagulopathy
• Cohort B: Cancer types not eligible as the sole diagnosis: Basal cell or squamous cell carcinoma of the skin only Acute leukemia Myeloproliferative neoplasm
• Cohort A and B: Evidence of active hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) infection at screening
• Cohort A and B: Any serious medical, psychiatric, or social condition that, in the investigator’s opinion, would interfere with trial participation, compliance, or interpretation of trial results
• Cohort A: Any absolute contraindication to continuation of anticoagulation (eg, recent life-threatening bleeding, intracranial hemorrhage, major spinal/eye surgery with unresolved risk)
• Cohort A and B: The estimated glomerular filtration rate (eGFR) <30 mL/min
• Cohort A: Relevant baseline laboratory abnormalities at screening: Hemoglobin <10 g/dL Platelet count <100,000/109 cells/L Alanine Aminotransferase (ALT) or AST (Aspartate Aminotransferase) > 1.5 × Upper Limit of Normal (ULN) Total bilirubin >1.5 × ULN
• Cohort A and B: Pregnancy or breastfeeding
• Cohort A: Clear indication for indefinite/extended anticoagulation for any reason