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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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up throughout part a and part b of the study, approximately 7 years
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
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.
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
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of Participants With FVIII Inhibitor Development
Success Rate of Immune Tolerance Induction (ITI)
Secondary outcome measures
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 moreTrial 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
Find a Location
Who is running the clinical trial?
Baxalta now part of ShireLead Sponsor
110 Previous Clinical Trials
8,984 Total Patients Enrolled
52 Trials studying Hemophilia A
3,921 Patients Enrolled for Hemophilia A
Takeda Development Center Americas, Inc.Industry Sponsor
56 Previous Clinical Trials
11,910 Total Patients Enrolled
2 Trials studying Hemophilia A
71 Patients Enrolled for Hemophilia A
Study DirectorStudy DirectorTakeda
1,207 Previous Clinical Trials
489,560 Total Patients Enrolled
64 Trials studying Hemophilia A
4,899 Patients Enrolled for Hemophilia A
Media Library
Eligibility Criteria:
This trial includes the following eligibility 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.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.My FVIII inhibitor level has not been confirmed to be 0.6 BU or higher with a second test.I am under 6 years old.I have had less than 3 treatments with ADVATE, BAX 855, or plasma transfusions.My inhibitor has disappeared on its own before treatment.I have severe liver problems, with very high liver enzyme levels or blood clotting issues.My kidney function is severely impaired.I have a bleeding disorder that is not hemophilia A.My weight is expected to be under 5 kg at the start of the study.I will be taking a strong immune system affecting drug during the study.I have received more than two doses of ADVATE before joining the study.
Research Study Groups:
This trial has the following groups:- Group 1: Previously untreated patients (PUPs)
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Where are the research sites for this project?
"This particular trial is based in three hospitals - Cincinnati Children's, Univ Florida College of Medicine, and Primary Children's - but is also running at 22 other sites."
Answered by AI
Is this research groundbreaking in any way?
"ITI has been the focus of 2 clinical trials in the past 5 years. These trials were conducted across 38 cities and 24 countries. The first trial was sponsored by Baxalta and completed Phase 3 in 2015. 7 studies have been conducted in total."
Answered by AI
Could you please cite any other research that has used ITI?
"Right now, there are 2 on-going clinical trials evaluating ITI. One of these trials is in Phase 3. The majority of the research being conducted is based in Dallas, Texas; however, there are 93 different locations running similar studies."
Answered by AI
What have been the most common side effects associated with ITI?
"ITI's safety is reassuringly supported by both efficacy data and multiple rounds of safety trials, so it received a score of 3."
Answered by AI
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