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
Interventions
Product Name: Fitusiran 80mg
Product Code: SAR439774
Pharmaceutical Form: Solution for injection in vial
INN or Proposed INN: fitusiran
CAS Number: 1609016-97-8
Current Sponsor code: SAR439774
Concen
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the study only if all of the following criteria apply: Age 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 02. Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial Sex 03. Male A) There are no contraceptive requirements for this study except where required by local regulations. Informed Consent 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 participant’s legally authorized representative. Are the trial subjects under 18? yes Number of subjects for this age range: 22 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 210 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 12
Exclusion criteria
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: Medical conditions 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 02. Current participation in ITI 03. Current use of factor concentrates or BPAs as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes. However, participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period, per Investigator's judgement in agreement with the study Medical Monitor may be permitted to be enrolled. 04. Use of compounds other than factor concentrates or BPAs for hemophilia treatment, including use of emicizumab (Hemlibra®) within 6 months prior to screening. Prior/concurrent clinical study experience 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 06. Current or prior participation in a gene therapy trial Diagnostic assessments 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 08. Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized 09. Any country-related specific regulation that would prevent the participant from entering the study 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 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) 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 13. Any specific situation during study implementation/course that may rise ethics considerations 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 |
|---|---|
| Main Objective: To characterize the long-term safety and tolerability of fitusiran.;Secondary Objective: To characterize the efficacy and the long-term efficacy of fitusiran as assessed by the frequency of: - Bleeding episodes, - Spontaneous bleeding episodes, - Joint bleeding episodes. To characterize the effects of fitusiran on health-related quality of life measures in participants =17 years of age.;Primary end point(s): Incidence, severity, relatedness, and seriousness of AEs, and laboratory assessments.;Timepoint(s) of evaluation of this end point: From study baseline (day 1) up to maximum 88 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Annualized bleeding rate in the treatment period. Annualized spontaneous bleeding rate in the treatment period. Annualized joint bleeding rate in the treatment period. Change in haemophilia quality of life questionnaire for adults (Haem-A-QoL) physical health score and total score in the treatment period (in participants =17 years of age).;Timepoint(s) of evaluation of this end point: From first ever dose of fitusiran (day 1) up to maximum 78 months | — |
Countries
Australia, 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