Overview

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Status:
Active, not recruiting
Trial end date:
2023-10-20
Target enrollment:
0
Participant gender:
Male
Summary
Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: - To investigate the long-term efficacy of fitusiran - To characterize the safety and efficacy of concomitantly administered Factor VIII (FVIII), Factor IX (FIX) or bypassing agents (BPA) and fitusiran for treatment of bleeding episodes - To assess changes in health-related quality of life (QOL) over time - To characterize antithrombin (AT) reduction and thrombin generation (TG) increase - To characterize the pharmacokinetics (PK) of fitusiran
Phase:
Phase 1/Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Alnylam Pharmaceuticals
Genzyme, a Sanofi Company
Treatments:
Factor VIII
Criteria
Inclusion Criteria:

- Completed and tolerated study drug dosing in study TDR14767 (ALN-AT3SC-001)

- Male aged ≥18 years

- Moderate or severe, clinically stable hemophilia A or B as evidenced by a laboratory
FVIII or FIX level ≤5% at screening. Patients with a FVIII or FIX level >5% at
screening will be eligible on provision of a historic laboratory report indicating a
trough level ≤5%

- Willing and able to comply with the study requirements and provide written informed
consent

Exclusion Criteria:

- Clinically significant liver disease

- Patients known to be human immunodeficiency virus seropositive and have a CD4 count
<200 cells/μL

- History of venous thromboembolism

- Current serious mental illness that, in the judgment of the Investigator, may
compromise patient safety, ability to participate in all study assessments, or study
integrity

- Clinically relevant history or presence of cardiovascular, respiratory,
gastrointestinal, renal, neurological, inflammatory, or other diseases that, in the
judgment of the Investigator, precludes study participation