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RNAi Therapeutics

Fitusiran for Pediatric Hemophilia (ATLAS-PEDS 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

Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL

Weight requirements at the time of enrollment: 8 to <45 kg

Must not have

Co-existing thrombophilic disorder

Acute or chronic Hepatitis B virus infection

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of at follow up)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a new drug for hemophilia in children. The goal is to see if it is safe and effective.

Who is the study for?

This trial is for male children aged 1 to less than 12 with severe hemophilia A or B. They must have a history of inhibitor antibodies and meet specific blood test criteria. Participants need good vein access for blood draws, weigh between 8 to <45 kg, and be able to follow the study plan with parental consent.

What is being tested?

The trial is testing appropriate dose levels of Fitusiran in young boys with severe hemophilia A or B. It aims to confirm dosing, assess safety and tolerability, and measure how much drug stays in the bloodstream over time.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions at injection sites, possible liver issues due to altered clotting factor production, allergic responses, or other unforeseen complications related to Fitusiran.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My latest inhibitor test result is below 0.6 BU/mL, but I have records showing two previous results of 0.6 BU/mL or higher.

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My weight is between 8 and 45 kg.

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I have antibodies against clotting factors and meet specific test criteria.

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I am a boy aged between 1 and 11 years.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a blood clotting disorder.

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I have Hepatitis B.

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I currently have acute Hepatitis A or E.

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I am HIV positive with a CD4 count below 400.

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I have a catheter and have had serious issues with it that required hospital care or blood thinners.

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I have a serious liver condition.

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I have not used Hemlibra® in the last 6 months.

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My kidney function is not normal.

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I have had blood clots in my arteries or veins not caused by a vein catheter.

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I have a bleeding disorder, but it's not hemophilia A or B.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of at follow up)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of at follow up)

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of at follow up) for reporting.