A study of fitusiran (ALN-AT3SC) in hemophilia A and B patients without inhibitors

Phase 1

• Conditions: Hemophilia A or Hemophilia B; MedDRA version: 20.0Level: LLTClassification code 10060613Term: Hemophilia A (Factor VIII)System Organ Class: 100000011915; MedDRA version: 20.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 100000011913; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-001464-11-IE
• Lead Sponsor: Alnylam Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-07-05
• Last Update Posted: 21 May 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 90

Inclusion Criteria

Males =12 years of age.

Severe hemophilia A or B (evidenced by a central laboratory FVIII <1% or FIX level =2% at Screening) without inhibitors (evidenced by inhibitor titer of <0.6 BU/mL and supported by medical records)

AT activity =60% at Screening

A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 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: 8
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 77
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

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 factor concentrates, as determined by the frequency of bleeding episodes.;Secondary Objective: To evaluate the efficacy of fitusiran compared to on demand treatment with factor concentrates, 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 receiving 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): Annualized spontaneous bleeding rate <br><br>Annualized joint bleeding rate<br><br>Haem-A-QOL score;Timepoint(s) of evaluation of this end point: Through 9 months