Trial Condition(s):

Treatment of bleeding, Prophylaxis of bleeding, Hemophilia A, Children

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 (Alfa-PROTECT)

Bayer Identifier:

21824

ClinicalTrials.gov Identifier:

NCT05147662

EudraCT Number:

2021-004858-30

Recruiting

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.

Inclusion Criteria
- 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
Exclusion Criteria
- 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 the 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

Trial Summary

Enrollment Goal
33
Trial Dates
black-arrow
Phase
3
Could I receive a placebo?
No
Products
Jivi (Damoctocog, BAY94-9027)
Accepts Healthy Volunteers
No

Where to Participate

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Locations
Status
LocationsStatus
Locations

Many Locations

Many Locations, Canada

Status
Recruiting
 
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Many Locations

Many Locations, Turkey

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Not yet recruiting
 
Locations

Many Locations

Many Locations, Brazil

Status
Not yet recruiting
 
Locations

UC Davis Comprehensive Cancer Center

Sacramento, United States, 95817

Status
Not yet recruiting
 
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Rush University Medical Center

Chicago, United States, 60612

Status
Not yet recruiting
 
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University Hospitals Cleveland Medical Center

Cleveland, United States, 44106

Status
Not yet recruiting
 
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Rutgers Robert Wood Johnson Medical School

New Brunswick, United States, 08901

Status
Not yet recruiting
 
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Arnold Palmer Hospital for Children

Orlando, United States, 32806

Status
Recruiting
 
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Tulane University School of Medicine

New Orleans, United States, 70112

Status
Not yet recruiting
 
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McMaster Children's Hospital

Hamilton, Canada, L8N 3Z5

Status
Recruiting
 
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Ege Universitesi Tip Fakultesi

Izmir, Turkey, 35100

Status
Recruiting
 
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Istanbul Universitesi Istanbul Tip Fakultesi

Istanbul, Turkey, 34093

Status
Not yet recruiting
 
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Acibadem Adana Hastanesi

Adana, Turkey, 01130

Status
Recruiting
 
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Akdeniz Universitesi Tip Fakultesi Hastanesi

Antalya, Turkey, 07059

Status
Not yet recruiting
 
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Hacettepe Universitesi Tip Fakultesi

Ankara, Turkey, 06100

Status
Not yet recruiting
 
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Ondokuz Mayis Uni Tip Fakultesi

Samsun, Turkey, 55139

Status
Not yet recruiting
 
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Ankara Sehir Hastanesi

Ankara, Turkey, 06800

Status
Not yet recruiting
 
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Necmettin Erbakan Universitesi Meram Tip Fakultesi Hastanesi

Konya, Turkey, 42080

Status
Not yet recruiting
 
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Gaziantep Universitesi Tip Fakultesi

Gaziantep, Turkey, 27070

Status
Not yet recruiting
 
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Instituto de Pesquisa e Inovação Tecnologica (IPITEC)

São Paulo, Brazil, 01223-001

Status
Not yet recruiting
 
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Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - HEMOES

Vitória, Brazil, 29.047-105

Status
Not yet recruiting
 
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Hospital das Clínicas de Campinas - UNICAMP

Campinas, Brazil, 13083-878

Status
Not yet recruiting
 
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HEMORIO

Rio de Janeiro, Brazil, 20211030

Status
Not yet recruiting
 
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Hosp Clínicas Facult. Med. de Ribeirão Preto / USP

Ribeirão Preto, Brazil, 14051-140

Status
Recruiting
 
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Fundação Centro de Hemoterapia e Hematologia do Pará

Belém, Brazil, 66033-000

Status
Not yet recruiting
 
Locations

OUS Rikshospitalet Klinisk Forskningspost Barn

Oslo, Norway, 0372

Status
Recruiting
 
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AORN Santobono-Pausilipon

Napoli, Italy, 80122

Status
Not yet recruiting
 
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IRCCS Ospedale Pediatrico Bambino Gesù

Roma, Italy, 00165

Status
Not yet recruiting
 
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Organizacion Medica de Investigacion (OMI)

Buenos Aires, Argentina, C1015ABO

Status
Not yet recruiting
 
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Instituo Hematología Arbesú

Godoy Cruz Mendoza, Argentina, 5501

Status
Recruiting
 
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Instituto de Hematología Dr. Rubén Dávoli

Rosario, Argentina, S2000CKF

Status
Not yet recruiting
 
Locations

Hospital de Niños Sor María Ludovica

La Plata, Argentina, 1900

Status
Not yet recruiting
 

Trial Design