A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
- Conditions
- Hemophilia A, Hemophilia B
- Registration Number
- JPRN-jRCT2080224129
- Lead Sponsor
- Sanofi K.K.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- Male
- Target Recruitment
- 120
Inclusion Criteria:
1. Males, >=12 years of age
2. Severe hemophilia A or B without inhibitors
- Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is <=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 BU/mL at Screening
* No use of BPAs 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
3. A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening.
4. 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, ie, 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 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 judgment
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<br>period: 1 month onset period + efficacy period of 8 months)]<br>efficacy<br>2. spontaneous bleeding rate<br>[Time Frame: 8 months (efficacy period: day 29 to Month 9)]<br><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 0-100 with higher scores representing greater impairment.<br>[Time Frame: 9 months (treatment period: 1 month onset<br>period + efficacy period of 8 months)]<br>efficacy<br>5. Number of participants reported with treatment-emergent<br>adverse events<br>[Time Frame: 15 months (9 months treatment + 6 months<br>follow up)]