An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02554773
• Other study ID #: LTE14762
• Secondary ID: ALN-AT3SC-002201
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: September 18, 2015
• Est. completion date: March 21, 2023

Study information

• Verified date: April 2023
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

It is anticipated that patients in this study will receive treatment with open label fitusiran for approximately 7 years or until fitusiran becomes commercially available, whichever occurs first.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 37
• Est. completion date: March 21, 2023
• Est. primary completion date: March 21, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Drug:
• Fitusiran (SAR439774)
Pharmaceutical form: solution for injection Route of administration : subcutaneous (sc)

Locations

Country	Name	City	State
Bulgaria	Clinical Trial Site	Plovdiv
Bulgaria	Clinical Trial Site	Sofia
Russian Federation	Clinical Trial Site	Kirov
Russian Federation	Clinical Trial Site	Moscow
Switzerland	Clinical Trial Site	Zurich
United Kingdom	Clinical Trial Site	Basingstoke
United Kingdom	Clinical Trial Site	Glasgow
United Kingdom	Clinical Trial Site	London
United Kingdom	Clinical Trial Site	London
United Kingdom	Clinical Trial Site	Truro
United States	Clinical Trial Site	Ann Arbor	Michigan
United States	Clinical Trial Site	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  Bulgaria,  Russian Federation,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation	Incidence of treatment-emergent adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation	Up to 7 years
Secondary	Annualized bleed rate (ABR)		Up to 7 years
Secondary	Time intervals between bleeding episodes		Up to 7 years
Secondary	Weight-adjusted consumption of FVIII, FIX, or BPA	BPA Assessment of concomitantly administered factor VIII (FVIII), factor IX (FIX), or bypassing agents (BPA) and fitusiran for treatment of bleeding	Up to 7 years
Secondary	Changes in health-related quality of life (QOL) with long-term dosing of fitusiran	QOL assessed by an EQ-5D questionnaire and HAEM-A-QoL	Up to 7 years
Secondary	Pharmacokinetics (PK) of fitusiran: Cmax		Day 1, Month 12, Month 24
Secondary	Pharmacokinetics (PK) of fitusiran: AUC		Day 1, Month 12, Month 24
Secondary	Pharmacokinetics (PK) of fitusiran: t1/2		Day 1, Month 12, Month 24
Secondary	Pharmacokinetics (PK) of fitusiran: CL/F		Day 1, Month 12, Month 24
Secondary	Pharmacokinetics (PK) of fitusiran: V/F		Day 1, Month 12, Month 24
Secondary	The effect of fitusiran on plasma levels of antithrombin (AT) and thrombin generation (TG)		Up to 7 years