Summary

Eligibility
for males ages 7-11 (full criteria)
Location
at UC Davis
Dates
study started
estimated completion

Description

Summary

Researchers are looking for a better way to treat hemophilia A. Hemophilia A is a genetic disorder where the body does not create enough of a protein called clotting factor 8 (FVIII) present in the blood. People with hemophilia A may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding. In severe hemophilia A (clotting factor 8 levels less than 1%) bleedings are more likely to happen. In this study researchers want to learn more about the treatment called BAY94-9027. BAY94-9027 is an injectable medicine used to replace missing clotting factor 8. In BAY94-9027 the clotting factor 8 has been pegylated (combined with a substance called polyethylene glycol (PEG)). This is to make the treatment last longer in the body so that less injections are required. BAY94-9027 is already available for the prevention and treatment of bleeding in adults and children who are 12 years and older. BAY 94-9027 is also called Jivi. BAY94-9027 is not yet available for children aged 7 to less than 12 years. One potential specific risk of pegylated drugs is that proteins in the blood called antibodies are built. These may attach to the pegylation part of the drug and this in turn may lead to allergic reactions and the drug not working as well as it should during first 4 infusions. In studies that have been done so far, this has been seen in some children younger than six years, but not in 29 children aged 6 to less than 12 years treated with BAY94-9027. Further safety information related to how the body reacts to BAY94-9027 is however still needed for this age group. The main purpose of this study is to learn how safe BAY94-9027 is (safety) and how it affects the body (tolerability) in previously treated children with severe hemophilia A who are between 7 to less than 12 years. To answer this question, the researchers will study information about two medical problems of special interest, if allergic reactions occur (also called hypersensitivity) and if the drug is not working as well as it should (also called loss of efficacy) during the first 4 infusions. Allergic reactions may range from mild local reactions to widespread effects such as shortness of breath, skin rashes and low blood pressure. Only allergic reactions related to the study treatment will be considered. The assessment if loss of efficacy occurred will be based on the occurrence of bleeding, the clotting factor VIII level in blood after injection called recovery, FVIII inhibitor tests and measurement of antibodies against the PEG. The study has two parts, A and B. Each part takes six months. In part A the participants will receive two injections of BAY94-9027 per week. In part B, the number of injections may be decreased, with up to five days between the injections. The participants in this study will visit the study site around 12 times and will have 6 phone visits. In the beginning of part A there will be 2 visits per week for two weeks. In the remainder of part A, there will be one visit every 4 weeks. In part B, there will be a phone visit per month and a study site visit every 3 months. The participants' and their caregivers will record in an electronic patient diary information about when the study treatment was given and bleeding episodes that have happened. During the study, the study doctors and their team will - take blood samples, - do physical examinations, - review the participants' electronic diary - ask questions about the participants' quality of life, - ask the participants questions about how they are feeling and what adverse events they are having An adverse event is a medical problem that happens during the study. Doctors keep track of all adverse events that happen in study, even if they do not think the adverse events might be related to the study treatments.

Official Title

A Phase 3, Single Group Treatment, Open-label, Study to Evaluate the Safety of BAY 94-9027 Infusions for Prophylaxis and Treatment of Bleeding in Previously Treated Children Aged 7 to <12 Years With Severe Hemophilia A

Keywords

Treatment of Bleeding Prophylaxis of Bleeding Hemophilia A Children Hemorrhage Factor VIII Damoctocog alfa pegol (Jivi, BAY94-9027)

Eligibility

You can join if…

Open to males ages 7-11

  • Participants with severe hemophilia A (participant's own FVIII activity [FVIII:C] <1%
  • Participants must be previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 50 exposure days (EDs) at the time of signing the informed consent
  • Participant has understood the study if appropriate for his age, informed consent must be signed by the parent, the participant can only sign the assent
  • Willingness and ability of participants and/or parents /caregivers to complete training in the use of the electronic patient diary (EPD) and to document infusions during the study

You CAN'T join if...

  • History of FVIII inhibitors
  • Current evidence of inhibitor to FVIII measured using the Nijmegen-modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory)
  • Any other inherited or acquired bleeding disorder in addition to hemophilia A (e.g. von Willebrand disease, hemophilia B)
  • Known hypersensitivity or allergic reaction to drug substance, excipients or mouse or hamster protein
  • Any other significant medical condition that the investigator feels would be a risk to the patient or would impede the study
  • Requires any pre-medication to tolerate FVIII treatment (e.g. antihistamines)
  • Planned major surgery during the study
  • Any individual who is receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (> 14 days) within the last 3 months
  • Any individual who received commercially available subcutaneous factor substitution therapy (emicizumab) within last 6 months
  • The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug within 30 days of study entry or previous participation in a clinical study with BAY94-9027

Locations

  • UC Davis Comprehensive Cancer Center not yet accepting patients
    Sacramento California 95817 United States
  • Alberta Children's Hospital not yet accepting patients
    Calgary Alberta T3B 6A8 Canada

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Bayer
ID
NCT05147662
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 33 study participants
Last Updated