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

NCT02554773

CompletedPhase 1, Phase 2

Sponsor: Genzyme, a Sanofi Company

Conditions: Hemophilia A, Hemophilia B

Interventions: Fitusiran (SAR439774)

Countries: United States, Bulgaria, Russia, Switzerland, United Kingdom

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

Eligibility overview

Sex: MALE

Age: 18 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility 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
Locations (12)
  • Ann Arbor, Michigan, United States
  • Pittsburgh, Pennsylvania, United States
  • Plovdiv, Bulgaria
  • Sofia, Bulgaria
  • Kirov, Russia
  • Moscow, Russia
  • Zurich, Switzerland
  • Basingstoke, United Kingdom
  • Glasgow, United Kingdom
  • London, United Kingdom
  • London, United Kingdom
  • Truro, United Kingdom