Clinical trial

An Open-label Extension Study of Subcutaneously Administered Fitusiran in Patients With Moderate or Severe Hemophilia A or B Who Have Participated in a Previous Clinical Study With Fitusiran

Name

LTE14762

Description

Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: * To investigate the long-term efficacy of fitusiran * To characterize the safety and efficacy of concomitantly administered Factor VIII (FVIII), Factor IX (FIX) or bypassing agents (BPA) and fitusiran for treatment of bleeding episodes * To assess changes in health-related quality of life (QOL) over time * To characterize antithrombin (AT) reduction and thrombin generation (TG) increase * To characterize the pharmacokinetics (PK) of fitusiran

Trial arms

Trial start

2015-09-18

Estimated PCD

2023-03-21

Trial end

2023-03-21

Status

Completed

Phase

Early phase I

Treatment

Fitusiran (SAR439774)

Pharmaceutical form: solution for injection Route of administration : subcutaneous (sc)

Arms:

Fitusiran

Size

Primary endpoint

Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation

Up to 7 years

Eligibility criteria

Inclusion Criteria: * Completed and tolerated study drug dosing in study TDR14767 (ALN-AT3SC-001) * Male aged ≥18 years * Moderate or severe, clinically stable hemophilia A or B as evidenced by a laboratory FVIII or FIX level ≤5% at screening. Patients with a FVIII or FIX level \>5% at screening will be eligible on provision of a historic laboratory report indicating a trough level ≤5% * Willing and able to comply with the study requirements and provide written informed consent Exclusion Criteria: * Clinically significant liver disease * Patients known to be human immunodeficiency virus seropositive and have a CD4 count \<200 cells/μL * History of venous thromboembolism * Current serious mental illness that, in the judgment of the Investigator, may compromise patient safety, ability to participate in all study assessments, or study integrity * Clinically relevant history or presence of cardiovascular, respiratory, gastrointestinal, renal, neurological, inflammatory, or other diseases that, in the judgment of the Investigator, precludes study participation

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 37, 'type': 'ACTUAL'}}

Updated at

2023-04-04

1 organization

1 product

2 indications

Organization

Product

Indication

Indication