NCT03417245

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 120 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes. Secondary Objectives: * To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by: * The frequency of spontaneous bleeding episodes. * The frequency of joint bleeding episodes. * Health-related quality of life (HRQOL) in participants \>=17 years of age. * To determine the frequency of bleeding episodes during the onset period. * To determine the safety and tolerability of fitusiran.

Detailed description

The duration of treatment with fitusiran was 9 months. The estimated total time on study, inclusive of screening, was up to 11 months for all participants in the factor on-demand arm and for participants in the fitusiran arm who enrolled in the extension study (LTE15174). The estimated total time on the study was up to 17 months for participants in the fitusiran treatment arm who did not enroll in the extension study due to the requirement for up to an additional 6 months of follow-up monitoring for antithrombin levels. Participants who completed the study will be eligible for an open-label extension study LTE15174 (NCT03754790).

Conditions

• Hemophilia A
• Hemophilia B

Interventions

Type	Name	Description
DRUG	fitusiran	by SC injection
DRUG	factor concentrates	by intravenous (IV) injection

Timeline

• Start date: 2018-03-01
• Primary completion: 2021-01-26
• Completion: 2021-07-14
• First posted: 2018-01-31
• Last updated: 2022-03-28
• Results posted: 2022-02-04

Locations

64 sites across 19 countries: United States, Australia, China, Denmark, France, Germany, Hungary, India, Israel, Italy, Japan, Malaysia, South Africa, South Korea, Spain, Taiwan, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study