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RNAi Therapeutics

Fitusiran 80 mg Prophylaxis for Hemophilia B (ATLAS-INH Trial)

Phase 3

Waitlist Available

Research Sponsored by Genzyme, a Sanofi Company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from day 29 up to day 246 or up to the last day of bleeding follow up (any day up to day 246), whichever was the earliest

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular medication. In addition, the study assessed safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).

Eligible Conditions

Hemophilia B
Hemophilia A

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from day 29 up to day 246 or up to the last day of bleeding follow up (any day up to day 246), whichever was the earliest

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from day 29 up to day 246 or up to the last day of bleeding follow up (any day up to day 246), whichever was the earliest

This trial's timeline: 3 weeks for screening, Varies for treatment, and from day 29 up to day 246 or up to the last day of bleeding follow up (any day up to day 246), whichever was the earliest for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

Secondary study objectives

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Onset Period

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

Estimated Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

+7 more

Side effects data

From 2021 Phase 3 trial • 60 Patients • NCT03417102

Haemorrhoids

Upper Respiratory Tract Infection

Urticaria

Pharyngotonsillitis

Muscle Haemorrhage

Haematuria

Back Pain

Cystitis

Asthenia

Haemorrhage

Asymptomatic Covid-19

Tooth Fracture

Traumatic Haemorrhage

Haemarthrosis

Hypoaesthesia

Headache

Constipation

100%

80%

60%

40%

20%

Study treatment Arm

Bypassing Agents (BPA) On-demand

Fitusiran 80 mg Prophylaxis

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Fitusiran 80 mg ProphylaxisExperimental Treatment1 Intervention

Participants received Fitusiran 80 mg subcutaneously (SC) as prophylaxis once monthly from Day 1, along with the on-demand BPAs (per investigator's discretion and within bleeding dosing guidelines) for treatment of breakthrough bleeding episodes, up to a total of 9 months.

Group II: Bypassing Agents (BPA) On-demandActive Control1 Intervention

Participants received On-demand BPAs (use of these agents, as needed, for episodic bleeding episodes, and not on a regular regimen intended to prevent spontaneous bleeding) per Investigator discretion from Day 1 for treatment of breakthrough bleeding episodes, up to a total of 9 months.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

fitusiran

2018

Completed Phase 3

~180

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