Hemophilia A or Hemophilia B MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850
Conditions
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Males =12 years of age. Severe hemophilia A or B (evidenced by a central laboratory FVIII =65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: 1. Known co-existing bleeding disorders other than hemophilia A or B, ie, Von Willebrand’s disease, additional factor deficiencies, or platelet disorders. 2. Current use of factor concentrates as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes. 3. AT activity 1.2; b. ALT and/or AST >1.5× upper limit of normal reference range (ULN); c. Total bilirubin >ULN (>1.5 ULN in patients with Gilbert’s Syndrome); d. History of portal hypertension, esophageal varices, or hepatic encephalopathy; e. Presence of ascites by physical exam 5. Hepatitis C virus antibody positive, except patients with a history of HCV infection who meet both conditions a. and b.: a. Completed curative treatment at least 12 weeks prior to enrollment and attained sustained virologic response as documented by a negative HCV RNA at screening, or they have spontaneously cleared infection as documented by negative HCV RNA at Screening. b. No evidence of cirrhosis according to one of the following assessments: ? FibroScan <12.5 kPa (where available), or ? FibroTest score <0.75 and APRI <2 (if FibroScan unavailable) 6. Presence of acute hepatitis, ie, hepatitis A, hepatitis E. 7. Presence of acute or chronic hepatitis B infection (IgM anti-HBc antibody positive or HBsAg positive). 8. Platelet count =100,000/µL. 9. Presence of acute infection at Screening. 10. Known to be HIV positive with CD4 count <200 cells/µL. 11. Estimated glomerular filtration rate =45 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula). 12. Co-existing thrombophilic disorder, as determined by presence of any of the below as identified at central laboratory (or via historical results, where available): a. FV Leiden (homozygous or heterozygous) b. Protein S deficiency c. Protein C deficiency d. Prothrombin mutation (G20210A; homozygous or heterozygous) 13. History of antiphospholipid antibody syndrome. 14. History of arterial or venous thromboembolism, atrial fibrillation, significant valvular disease, myocardial infarction, angina, transient ischemic attack, or stroke. Patients who have experienced thrombosis associated with indwelling venous access may be enrolled. 15. Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin that has been successfully treated. 16. Any condition (eg, medical concern), which in the opinion of the Investigator, would make the patient unsuitable for dosing on Day 1 or which could interfere with the study compliance, the patient’s safety and/or the patient’s participation in the completion of the treatment period of the study. This includes significant active and poorly controlled (unstable) cardiovascular, neurologic, gastrointestinal, endocrine, renal or psychiatric disorders unrelated to hemophilia identified by key laboratory abnormalities or medical history. 17. At Screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study. 18. Completion of a surgical procedure within 14 days prior to Screening, or currently receiving additional factor infusion for postoperative hemostasis. 19. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc. 20. Inadequate venous ac
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of fitusiran compared to on demand treatment with factor concentrates, as determined by the frequency of bleeding episodes.;Secondary Objective: To evaluate the efficacy of fitusiran compared to on demand treatment with factor concentrates, as determined by the frequency of spontaneous bleeding episodes, the frequency of joint bleeding episodes in patients, and health related quality of life (HRQOL) in patients receiving fitusiran.;Primary end point(s): Annualized bleeding rate (ABR) ;Timepoint(s) of evaluation of this end point: Through 9 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): ABR in the treatment period Annualized spontaneous bleeding rate in the efficacy period Annualized joint bleeding rate in the efficacy period Change in Haem-A-QOL physical health score and total score in the treatment period ABR in the onset period;Timepoint(s) of evaluation of this end point: Through 9 months | — |
Countries
Australia, Brazil, Bulgaria, Canada, China, Denmark, France, Germany, Hungary, India, Ireland, Israel, Italy, Japan, Korea, Republic of, Malaysia, Netherlands, Portugal, Russian Federation, South Africa, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States
Contacts
Public ContactProject Management
PPD Global
Outcome results
None listed