An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with HemophiliaA or B.
- Conditions
- Health Condition 1: D67- Hereditary factor IX deficiencyHealth Condition 2: D66- Hereditary factor VIII deficiency
- Registration Number
- CTRI/2019/07/020239
- Lead Sponsor
- Genzyme Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Open to Recruitment
- Sex
- Not specified
- Target Recruitment
- 0
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.
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence, severity, relatedness, and seriousness of AEs, and laboratory assessmentsTimepoint: 55 months
- Secondary Outcome Measures
Name Time Method 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