Health Condition 1: D67- Hereditary factor IX deficiency Health Condition 2: D66- Hereditary factor VIII deficiency
Conditions
Interventions
Intervention1: Fitusiran: A fixed dose of 80 mg fitusiran, subcutaneous administration, once-monthly for a duration of 48 months.
Control Intervention1: NIL: NIL
Sponsors
Genzyme Corporation
PPD Pharmaceuticals
Eligibility
Inclusion criteria
Inclusion criteria: Age I 01. Participant must be at least 12 years of age inclusive, at the time of signing the informed consent Type of participant and disease characteristics I 02. Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial Sex I 03. Male Informed Consent I 04. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participantâ??s legally authorized representative.
Exclusion criteria
Exclusion criteria: Medical conditions E 01. Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis Prior/concomitant therapy E 02. Current participation in ITI E 03. Current use of factor concentrates or BPAs as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes E 04. Use of compounds other than factor concentrates or BPAs for hemophilia treatment Prior/concurrent clinical study experience E 05. Received an investigational drug or device, other than fitusiran, within 30 days of anticipated IMP administration or 5 half-lives of the IMP, whichever is longer E 06. Current or prior participation in a gene therapy trial Diagnostic assessments E 07. ALT and/or AST >1.5� upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST >5� ULN for patients who were in the fitusiran arm in the parent study Other exclusions E 08. Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized E 09. Any country-related specific regulation that would prevent the participant from entering the study (country-specific requirements) E 10. Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures E 11. Participants who are dependent on the Sponsor or Investigator (as defined in section 1.61 of the International Council for Harmonisation (ICH)-Good Clinical Practice (GCP) E6) E 12. Participants are employees of the clinical study site or other individuals directly involved in the conduct of the study, or immediate family members of such individuals E 13. Any specific situation during study implementation/course that may rise ethics considerations E 14. Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence, severity, relatedness, and seriousness of AEs, and laboratory assessmentsTimepoint: 55 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Annualized bleeding rate in the treatment periodTimepoint: 55 months;Annualized joint bleeding rate in the treatment periodTimepoint: 55 months;Annualized spontaneous bleeding rate in the treatment periodTimepoint: 55 months;Change in haemophilia quality of life questionnaire for adults physical health score and total score in the treatment period (in participants â?¥17 years of age)Timepoint: 55 months | — |
Countries
Australia, Bulgaria, Canada, China, Denmark, France, Germany, Hungary, India, Ireland, Israel, Italy, Japan, Malaysia, Portugal, Republic of Korea, Russian Federation, South Africa, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States of America
Contacts
Public ContactRashmi Chitgupi
PPD Pharmaceutical Development India Private Limited
Outcome results
None listed