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Sponsor: Genzyme, a Sanofi Company
Conditions: Hemophilia A, Hemophilia B
Interventions: fitusiran, factor concentrates
Countries: United States, Australia, China, Denmark, France, Germany, Hungary, India
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes. Secondary Objectives: * 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. * Health-related quality of life (HRQOL) in participants \>=17 years of age. * To determine the frequency of bleeding episodes during the onset period. * To determine the safety and tolerability of fitusiran.
Sex: MALE
Age: 12 Years to —
Healthy volunteers: No
Study type: INTERVENTIONAL
Inclusion Criteria:
* Males, \>=12 years of age.
* Severe hemophilia A or B without inhibitors.
* Severity confirmed by a central laboratory where FVIII level was less than (\<) 1 percent (%) or Factor IX (FIX) level was less than or equal to (\<=) 2% at Screening.
* On-demand use of factor concentrate to manage bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following criterion:
* Nijmegen modified Bethesda assay inhibitor titer of \<0.6 Bethesda units per milliliter (BU/mL) at Screening.
* No use of Bypassing agents to treat bleeding episodes for at least the last 6 months prior to Screening.
* No history of immune tolerance induction therapy within the last 3 years prior to Screening.
* A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening.
* Willing and complied with the study requirements and to provide written informed consent and assent.
Exclusion Criteria:
* Known co-existing bleeding disorders other than hemophilia A or B, i.e., Von Willebrand's disease, additional factor deficiencies, or platelet disorders.
* Antithrombin (AT) activity \<60% at Screening.
* Co-existing thrombophilic disorder.
* Clinically significant liver disease.
* Active hepatitis C virus infection.
* HIV positive with a cluster of differentiation-4 count of \<200 cells/microliter.
* History of arterial or venous thromboembolism.
* Inadequate renal function.
* History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc).
* History of intolerance to SC injection(s).
* Any other conditions or comorbidities that would make the participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment.- Little Rock, Arkansas, United States
- Gainesville, Florida, United States
- Tampa, Florida, United States
- Chicago, Illinois, United States
- New Orleans, Louisiana, United States
- Ann Arbor, Michigan, United States
- Las Vegas, Nevada, United States
- Akron, Ohio, United States
- Camperdown, Australia
- Murdoch, Australia
- Prahran, Australia
- Beijing, China
- Guangzhou, China
- Hangzhou, China
- Shanghai, China
- Tianjin, China
- Copenhagen, Denmark
- Lyon, France
- Paris, France
- Rouen, France
- Berlin, Germany
- Frankfurt am Main, Germany
- Leipzig, Germany
- Budapest, Hungary
- Bangalore, India
- Jaipur, India
- Lucknow, India
- Mumbai, India
- Mumbai, India
- Mumbai, India
- + 34 more on CT.gov