A Study of fitusiran (ALN-AT3SC) in hemophilia A and B patients with inhibitors
- Conditions
- Hemophilia A or Hemophilia BMedDRA version: 20.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]MedDRA version: 20.0Level: LLTClassification code 10060613Term: Hemophilia A (Factor VIII)System Organ Class: 100000004850
- Registration Number
- EUCTR2016-001463-36-DK
- Lead Sponsor
- Genzyme Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 54
Males =12 years of age. In Denmark only males = 18 years of age.
Severe hemophilia A or B with inhibitors evidenced by:
a.A central laboratory measurement or documented medical record
evidence of FVIII <1% or FIX level =2% at Screening.
b.On-demand use of BPAs to manage bleeding episodes for at least the
last 6 months prior to Screening, and meet one of the following
Nijmegen-modified Bethesda assay results criteria:
Inhibitor titer of =0.6 BU/mL at Screening, or
Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence
of 2 consecutive titers =0.6 BU/mL, or Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence
of anamnestic response
A minimum of 6 bleeding episodes requiring BPA 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 in the case of patients under the
age of legal consent, per local and national requirements. Assent is N/A in Denmark as only patients = 18 years of age will be included.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 44
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 0
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 evaluate the efficacy of fitusiran compared to on-demand treatment with BPAs (bypassing agents), as determined by the frequency of bleeding episodes;Secondary Objective: To evaluate the efficacy of fitusiran compared to on-demand treatment with BPAs, as determined by the frequency of spontaneous bleeding episodes, the frequency of joint bleeding episodes in patients, and health related quality of life (HRQOL) in patients = 17 years of age. In Denmark only in patients = 18 years of age<br><br>To determine the frequency of bleeding episodes during the onset period<br><br>To determine the safety and tolerability of fitusiran;Primary end point(s): Annualized bleeding rate (ABR) ;Timepoint(s) of evaluation of this end point: Through 9 months
- Secondary Outcome Measures
Name Time Method Secondary end point(s): ABR in the treatment period<br>Annualized spontaneous bleeding rate in the efficacy period<br>Annualized joint bleeding rate in the efficacy period<br>Change in Haem-A-QOL physical health score and total score in the treatment period<br>ABR in the onset period;Timepoint(s) of evaluation of this end point: Through 9 months