A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
- Conditions
- Hemophilia A, Hemophilia B
- Registration Number
- JPRN-jRCT2080224128
- Lead Sponsor
- Sanofi K.K.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- Male
- Target Recruitment
- 54
Inclusion Criteria:
- Males, >=12 years of age
- Severe hemophilia A or B with inhibitors
(Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is <= 2% at Screening; Inhibitors defined as inhibitor titer of >= 0.6 BU/mL or as evidenced by medical records)
- A minimum of 6 bleeding episodes requiring bypassing agent treatment within the last 6 months prior to Screening
- Willing and able to comply 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
- Antithrombin (AT) activity <60% at Screening
- Co-existing thrombophilic disorder
- Clinically significant liver disease
- Active HCV infection
- HIV positive with a CD4 count of <200 cells/microL
- 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 patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method efficacy<br>1.Annualized bleeding rate(ABR) <br>[Time Frame: 8 months (efficacy period: day 29 to Month 9)]
- Secondary Outcome Measures
Name Time Method efficacy<br>1.Annualized bleeding rate (ABR)<br>[Time Frame: At 1 month (onset period), 9 months (treatment period:<br>1 month onset period + efficacy period of 8 months)]<br>efficacy<br>2.Annualized spontaneous bleeding rate <br>[Time Frame: 8 months (efficacy period: day 29 to Month 9)]<br>efficacy<br>3.Annualized joint bleeding rate<br>[Time Frame: 8 months (efficacy period: day 29 to Month 9)] <br>efficacy<br>4.Quality of Life (QOL) as measured by Haem-A-QOL Questionnaire score on a scale of 1-100 with higher scores representing greater impairment. <br>[Time Frame: 9 months (treatment period: 1 month onset period + efficacy period of 8 months)]<br>safety<br>5. Number of participants reported with treatment-emergent adverse<br>events<br>[Time Frame: 15 months (9 months treatment + 6 months follow up)]