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RNAi Therapeutic

Fitusiran for Hemophilia (ATLAS-OLE 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
Participant must be at least 12 years of age inclusive, at the time of signing the informed consent
Male
Must not have
Use of compounds other than factor concentrates or BPAs for hemophilia treatment
Clinically significant liver disease
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from study baseline (day 1) up to maximum 88 months
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial will study the safety and effectiveness of an experimental drug called fitusiran in people with a bleeding disorder.

Who is the study for?
This trial is for males aged 12 or older with severe hemophilia A or B who have finished a Phase 3 fitusiran study. They must be able to consent and follow the study rules. It's not for those on certain other treatments, with liver issues, history of blood clots, recent surgery, or in gene therapy trials.
What is being tested?
The trial tests the long-term safety and effectiveness of Fitusiran in managing bleeding episodes in hemophilia patients. It looks at how often bleeding occurs and its impact on quality of life over time.
What are the potential side effects?
While specific side effects are not listed here, the trial aims to monitor the long-term safety and tolerability of Fitusiran which may include tracking any adverse reactions related to bleeding management.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 12 years old or older.
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I am male.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I use treatments other than factor concentrates or BPAs for my hemophilia.
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I have a serious liver condition.
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I had surgery within the last 14 days or am receiving treatment for bleeding after surgery.
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I have had a blood clot in an artery or vein.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from study baseline (day 1) up to maximum 88 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and from study baseline (day 1) up to maximum 88 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of participants with treatment emergent adverse events (TEAEs)
Secondary study objectives
Annualized bleeding rate (ABR)
Annualized joint bleeding rate
Annualized spontaneous bleeding rate
+1 more

Side effects data

From 2022 Phase 3 trial • 80 Patients • NCT03549871
19%
Upper Respiratory Tract Infection
14%
Alanine Aminotransferase Increased
14%
Fibrin D Dimer Increased
10%
Cough
10%
Cholelithiasis
10%
Hepatic Steatosis
10%
Injection Site Pain
10%
Injection Site Erythema
10%
Abdominal Pain
10%
Cholecystitis
5%
Nasopharyngitis
5%
Toothache
5%
Arthropod Bite
5%
Vomiting
5%
Synovitis
5%
Haemophilic Arthropathy
5%
Central Venous Catheter Removal
5%
Aspartate Aminotransferase Increased
5%
Headache
5%
Influenza
5%
Pancreatitis Acute
5%
Arthralgia
5%
Fall
5%
Femur Fracture
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cohort A: SAS 1 - Fitusiran 80 mg QM
Overall: SAS 1 - Fitusiran 80 mg QM
Cohort A: SAS 2 - Fitusiran 50 mg Q2M
Overall: SAS 1 - Factor/BPA Prophylaxis
Cohort A: SAS 1 - BPA Prophylaxis
Cohort B: SAS 1 - Factor Prophylaxis
Cohort B: SAS 1 - Fitusiran 80 mg QM
Cohort A: SAS 2 - BPA Prophylaxis

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: FitusiranExperimental Treatment1 Intervention
Participants will be administered fitusiran as a subcutaneous injection once monthly or every other month for up to 48 months post initiation of modified IMP dose/frequency or until fitusiran becomes commercially available, whichever comes first.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fitusiran
2018
Completed Phase 3
~80

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor
527 Previous Clinical Trials
185,452 Total Patients Enrolled
Clinical Sciences & OperationsStudy DirectorSanofi
871 Previous Clinical Trials
2,020,080 Total Patients Enrolled

Media Library

Fitusiran (RNAi Therapeutic) Clinical Trial Eligibility Overview. Trial Name: NCT03754790 — Phase 3
Bleeding disorder Research Study Groups: Fitusiran
Bleeding disorder Clinical Trial 2023: Fitusiran Highlights & Side Effects. Trial Name: NCT03754790 — Phase 3
Fitusiran (RNAi Therapeutic) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03754790 — Phase 3
~69 spots leftby Nov 2026
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