NCT03549871

A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis

ATLAS-PPX: an Open-label, Multinational, Switching Study to Describe the Efficacy and Safety of Fitusiran Prophylaxis in Patients With Hemophilia A and B Previously Receiving Factor or Bypassing Agent Prophylaxis.

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 80 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA) prophylaxis. Secondary Objectives: * To characterize the following while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis: * the frequency of spontaneous bleeding episodes * the frequency of joint bleeding episodes * health related quality of life (HRQOL) in participants greater than or equal to (\>=) 17 years of age * To characterize the frequency of bleeding episodes during the onset and treatment periods in participants receiving fitusiran. * To characterize the safety and tolerability of fitusiran. * To characterize the annualized weight-adjusted consumption of factor/BPA while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis.

Detailed description

The estimated total time on study, inclusive of Screening, for each participant was up to 15 months for participants who were enrolled in the extension study except for participants in the subgroup of Cohort A, for whom it was up to 9 months. The estimated total time on study was up to 21 months (up to 15 months in participants in the subgroup of Cohort A) in participants who did not enroll in the extension study due to the requirement for an additional up to 6 months of follow-up for monitoring of antithrombin (AT) levels.

Conditions

• Hemophilia

Interventions

Type	Name	Description
DRUG	Fitusiran	Pharmaceutical form: solution for injection Route of administration: subcutaneous
DRUG	BPA prophylaxis	Pharmaceutical form: solution for injection Route of administration: Intravenous
DRUG	Factor (FVIII or FIX) prophylaxis	Pharmaceutical form: solution for injection Route of administration: Intravenous

Timeline

• Start date: 2018-07-25
• Primary completion: 2022-01-20
• Completion: 2022-03-25
• First posted: 2018-06-08
• Last updated: 2023-02-06
• Results posted: 2023-02-06

Locations

35 sites across 15 countries: United States, Australia, China, Denmark, France, Ireland, Israel, Italy, Japan, Malaysia, Mexico, South Korea, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study