A Study of Fitusiran in Hemophilia A and B Patients with Inhibitors

Phase 1

• Conditions: Hemophilia A or Hemophilia B; MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850; MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-001463-36-GB
• Lead Sponsor: Genzyme Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-05-31
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 54

Inclusion Criteria

Males =12 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.

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 46
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

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