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Coagulation Factor

BAX855 for Severe Hemophilia A

Phase 3

Waitlist Available

Research Sponsored by Baxalta now part of Shire

Eligibility Criteria Checklist

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

Must have

Participant has severe hemophilia A (Factor VIII (FVIII) <1%) as determined by the central laboratory, or a historical FVIII level <1% as determined at any local laboratory, optionally supported by an additional FVIII gene mutation consistent with severe hemophilia A.

Participant is <6 years old at the time of screening.

Must not have

Participant has detectable FVIII inhibitory antibodies (>=0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.

Spontaneous disappearance of the inhibitor prior to ITI.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up throughout part a and part b of the study, approximately 7 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is for young children with severe hemophilia A who have not been treated with BAX855 or other FVIII concentrates. The goal is to check for side effects and learn how well BAX855 controls bleeding.

Who is the study for?

This trial is for children under 6 with severe hemophilia A who haven't been treated with BAX855 or similar treatments. They must have had less than three exposures to certain medications and no history of severe allergic reactions to specific proteins, PEG, or Tween 80. Their liver and kidney functions should be normal, and they shouldn't be on other PEGylated drugs or certain immune-modifying drugs.

What is being tested?

The study tests BAX855 in young children with hemophilia A for side effects, including the development of antibodies that may reduce its effectiveness. The drug can be used as a regular preventive treatment or given when bleeding occurs. If antibodies develop, additional treatment will be provided within the study.

What are the potential side effects?

Possible side effects include allergic reactions to ingredients in BAX855 like mouse or hamster proteins, PEG, or Tween 80. There's also a risk of developing inhibitors (antibodies) against Factor VIII which could make the treatment less effective.

Eligibility Criteria

Inclusion Criteria

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

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I have severe hemophilia A with Factor VIII less than 1%.

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I am under 6 years old.

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I have had less than 3 treatments with ADVATE, BAX 855, or plasma transfusions.

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I have a confirmed positive inhibitor for my condition, with bleeding not controlled by usual treatments.

Exclusion Criteria

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

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My blood test shows I have FVIII inhibitory antibodies.

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My inhibitor has disappeared on its own before treatment.

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I have severe liver problems, with very high liver enzyme levels or blood clotting issues.

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My kidney function is severely impaired.

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I have a bleeding disorder that is not hemophilia A.

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My weight is expected to be under 5 kg at the start of the study.

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I will be taking a strong immune system affecting drug during the study.

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I have received more than two doses of ADVATE before joining the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ throughout part a and part b of the study, approximately 7 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~throughout part a and part b of the study, approximately 7 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and throughout part a and part b of the study, approximately 7 years 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 FVIII Inhibitor Development

Success Rate of Immune Tolerance Induction (ITI)

Secondary study objectives

Annualized Bleeding Rate (ABR) for Prophylactic and On-demand Treatment

Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies

Blood Loss Per Participant in Case of Surgery

+16 more

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

1Treatment groups

Experimental Treatment

Group I: Previously untreated patients (PUPs)Experimental Treatment2 Interventions

Part A (Main Study): Participants age \<3 years and not experienced two joint bleeds will receive on- demand treatment of 10-80 international unit per kilogram (IU/kg) of BAX 855 intravenously depending on the severity of the bleeding episode and age \<3 years or after a maximum of two joint bleeds will receive prophylaxis treatment with dose of 25-80 IU/kg of BAX855 IV (based on investigator discretion) once weekly for up to 100 EDs. Part A (Surgery): In participants, the administration of BAX 855 will be individualized based on the participants IR and half-life. For major surgery to achieve the target level of 80-100% FVIII in plasma of normal FVIII level and for minor surgery \>=30-60% FVIII levels for dental or other invasive surgery. Part B (Immune tolerance induction \[ITI\]): Participants will receive prophylaxis treatment of 100-200 IU/kg BAX 855 IV daily or 50 IU/kg three time in a week and will be reduced to twice weekly to maintain FVIII trough level of 1% for further 3 months.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Rurioctocog alfa pegol

FDA approved

ITI

2015

Completed Phase 3

~120

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