BAX855 for Severe Hemophilia A

Recruiting in Palo Alto (17 mi)

+90 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Baxalta now part of Shire

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855. This includes the buildup of antibodies against FVIII which may stop BAX855 from working properly. Another aim is to learn how well BAX855 controls bleeding. In this study, the children can receive BAX855 either as preventative treatment (prophylaxis), or as needed to treat bleeding (on-demand). In case a participant develops antibodies, treatment will be provided as part of the study.

Research Team

Study Director

Principal Investigator

Takeda

Eligibility Criteria

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.

Inclusion Criteria

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

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

Participant is immune competent with a cluster of differentiation 4 (CD4+) count > 200 cells per cubic millimeter (mm^3), as confirmed by the central laboratory at screening.

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Exclusion Criteria

I have used or plan to use PEGylated drugs recently or during the study.

My blood test shows I have FVIII inhibitory antibodies.

I have been treated with a blood clotting factor other than ADVATE or BAX 855, or received ADVATE, BAX 855, or a plasma transfusion for 3 or more days before screening.

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Treatment Details

Interventions

ITI (Coagulation Factor)
PEGylated Recombinant Factor VIII (Coagulation Factor)

Trial OverviewThe 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.

Participant Groups

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.