A study of fitusiran (ALN-AT3SC) in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis
- Conditions
- Emofilia A, Emofilia BMedDRA version: 20.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 100000004850MedDRA version: 20.0Level: LLTClassification code 10060613Term: Hemophilia A (Factor VIII)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2016-004087-19-IT
- Lead Sponsor
- ALNYLAM PHARMACEUTICALS, INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Male
- Target Recruitment
- 90
- Males =12 years of age.
- Severe hemophilia A or B (evidenced by a central laboratory FVIII <1% or FIX level =2% at Screening) with or without inhibitors
- At least 1 bleeding episode in the last 12 months
- AT activity =60% at Screening
- Documented details of prophylactic treatment with factor concentrates or bypassing agents for the treatment of hemophilia A or B for at least 6 months
- Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of patients under the age of legal consent
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 78
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5
- Patients with known co-existing bleeding disorders other than hemophilia A or B
- Patients with clinically significant liver disease
- Patients known to be HIV positive and have a CD4 count <200 cells/µL
- Patients with a history of arterial or venous thromboembolism
- Estimated glomerular filtration rate =45 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula)
- Patients with a co-existing thrombophilic disorder
- Patients with a history of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc
- Patients with a history of intolerance to SC injection(s)
- Patients with an anticipated or planned need for surgery during the study
- 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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To characterize the frequency of bleeding episodes while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor or bypassing agent prophylaxis.;Secondary Objective: To characterize the frequency of spontaneous bleeding episodes, the frequency of joint bleeding episodes in patients, and health related quality of life (HRQOL) in patients while receiving fitusiran treatment, relative to receiving factor or bypassing agent prophylaxis.;Primary end point(s): Annualized bleeding rate (ABR);Timepoint(s) of evaluation of this end point: through 13 months
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Annualized spontaneous bleeding rate<br>- Annualized joint bleeding rate<br>- Haem-A-QOL score;Timepoint(s) of evaluation of this end point: through 13 months