do_not_disturb_altRecruitment Complete
Treatment of bleeding, Prophylaxis of bleeding, Hemophilia A, Children
Bayer Identifier:
21824
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
Not Available
A study to learn how safe the study treatment BAY94-9027 is and how it affects the body in previously treated children aged 7 to less than 12 years with severe hemophilia A, a genetic bleeding disorder that is caused by the lack of a protein called clotting factor 8 (FVIII) in the blood
Trial purpose
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 8 level in blood after injection called recovery, clotting factor 8 inhibitor tests and measurement of antibodies against the PEG.
The study has two parts, A and B. Part A takes 6 months and part B takes 18 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 14 times and will have 15 phone visits. In part A, visit 1 is for screening. Visits 2 to 5 take place twice a week for two weeks. Visit 6 two weeks after visit 5, visits 7 to 10 take place monthly with visit 11 six weeks after visit 10. In part B, site visits will occur on month 9, 12, 18 and 24 and phone calls every month between the site visits. 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.
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 8 level in blood after injection called recovery, clotting factor 8 inhibitor tests and measurement of antibodies against the PEG.
The study has two parts, A and B. Part A takes 6 months and part B takes 18 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 14 times and will have 15 phone visits. In part A, visit 1 is for screening. Visits 2 to 5 take place twice a week for two weeks. Visit 6 two weeks after visit 5, visits 7 to 10 take place monthly with visit 11 six weeks after visit 10. In part B, site visits will occur on month 9, 12, 18 and 24 and phone calls every month between the site visits. 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.
Key Participants Requirements
Sex
MaleAge
7 - 11 YearsTrial summary
Enrollment Goal
33Trial Dates
March 2022 - May 2025Phase
Phase 3Could I Receive a placebo
NoProducts
Jivi (Damoctocog, BAY94-9027)Accepts Healthy Volunteer
NoWhere to participate
Status | Institution | Location |
---|---|---|
Recruiting | Many Locations | Many Locations, Canada |
Not yet recruiting | Many Locations | Many Locations, Turkey |
Not yet recruiting | Many Locations | Many Locations, Brazil |
Withdrawn | UC Davis Comprehensive Cancer Center | Sacramento, 95817, United States |
Withdrawn | Rush University Medical Center | Chicago, 60612, United States |
Withdrawn | University Hospitals Cleveland Medical Center | Cleveland, 44106, United States |
Withdrawn | Rutgers Robert Wood Johnson Medical School | New Brunswick, 08901, United States |
Completed | Arnold Palmer Hospital for Children | Orlando, 32806, United States |
Withdrawn | Tulane University School of Medicine | New Orleans, 70112, United States |
Completed | McMaster Children's Hospital | Hamilton, L8N 3Z5, Canada |
Not yet recruiting | Alberta Children's Hospital - Hematology / Oncology / Blood and Marrow Transplant Clinic | Calgary, T3B 6A8, Canada |
Completed | Ege Universitesi Tip Fakultesi | Izmir, 35100, Turkey |
Withdrawn | Istanbul Universitesi Istanbul Tip Fakultesi | Istanbul, 34093, Turkey |
Completed | Acibadem Adana Hastanesi | Adana, 1130, Turkey |
Completed | Akdeniz Universitesi Tip Fakultesi Hastanesi | Antalya, 7059, Turkey |
Completed | Hacettepe Universitesi Tip Fakultesi | Ankara, 6100, Turkey |
Active, not recruiting | Ondokuz Mayis Uni Tip Fakultesi | Samsun, 55139, Turkey |
Withdrawn | Health Ministry Of Türkiye Republic Ankara Bilkent City Hospital | Ankara, 6800, Turkey |
Withdrawn | Necmettin Erbakan Universitesi Meram Tip Fakultesi Hastanesi | Konya, 42080, Turkey |
Completed | Gaziantep Universitesi Tip Fakultesi | Gaziantep, 27070, Turkey |
Completed | Irmandadade da Santa Casa de Misericordia de Sao Paulo (iSANTACASA) | São Paulo, 01223-001, Brazil |
Withdrawn | Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - HEMOES | Vitória, 29.047-105, Brazil |
Active, not recruiting | Hospital das Clínicas de Campinas - UNICAMP | Campinas, 13083-878, Brazil |
Completed | HEMORIO | Rio de Janeiro, 20211-030, Brazil |
Completed | Hosp Clínicas Facult. Med. de Ribeirão Preto / USP | Ribeirão Preto, 14051-140, Brazil |
Withdrawn | Fundação Centro de Hemoterapia e Hematologia do Pará | Belém, 66033-000, Brazil |
Completed | OUS Rikshospitalet Klinisk Forskningspost Barn | Oslo, 372, Norway |
Completed | AORN Santobono-Pausilipon | Napoli, 80122, Italy |
Completed | Ospedale Pediatrico Bambino Gesù - Oncoematologia, Trapianto Emopoietico e Terapie Cellulari | Roma (RM), 00165, Italy |
Not yet recruiting | Organizacion Medica de Investigacion (OMI) | Buenos Aires, C1015ABO, Argentina |
Completed | Instituo Hematología Arbesú | Godoy Cruz, 5501, Argentina |
Active, not recruiting | Instituto de Hematología Dr. Rubén Dávoli | Rosario, S2000CKF, Argentina |
Completed | Hospital de Niños Sor María Ludovica | La Plata, 1900, Argentina |
Primary Outcome
- AESI (hypersensitivity and loss of efficacy) associated with the first 4 exposure days leading to discontinuationAESI = adverse events of special interestdate_rangeTime Frame:Up to 6 months
Secondary Outcome
- Adverse drug reactions (ADRs)date_rangeTime Frame:Up to 6 months
- Anti-drug antibody (ADA) developmentdate_rangeTime Frame:Pre-infusion and up to 6 months
- The number of participants with confirmed Factor VIII inhibitorsdate_rangeTime Frame:Pre-infusion and up to 6 months
- Annualized bleeding rate (ABR)date_rangeTime Frame:Up to 24 months
- BAY94-9027 consumptiondate_rangeTime Frame:Up to 24 months
- Number of infusions/month and year (Annualized Infusion Rate)date_rangeTime Frame:Up to 24 months
Trial design
Trial Type
InterventionalIntervention Type
Biological/VaccineTrial Purpose
TreatmentAllocation
N/ABlinding
N/AAssignment
Single Group AssignmentTrial Arms
1