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: 100000004850; MedDRA version: 20.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-001464-11-PT
• Lead Sponsor: Genzyme Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-08-30
• Study Completion: 2021-07-14
• Last Update Posted: 25 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 120

Inclusion Criteria

Males =12 years of age.

Severe hemophilia A or B without 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 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

A minimum of 6 bleeding episodes requiring factor concentrate 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: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 105
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