Safety Study of Damoctocog Alfa Pegol for Children Aged 7 to 11 with Severe Hemophilia A

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What is this study about?

This clinical trial is focused on studying severe hemophilia A, a condition where the blood does not clot properly due to a lack of a protein called Factor VIII. The study involves a treatment known as BAY 94-9027, which is a form of Factor VIII designed to help manage bleeding episodes in children who have been previously treated for this condition. The treatment is administered as a solution for injection, and it is intended to be used both for regular prevention of bleeding and for treating bleeding episodes when they occur.

The purpose of the study is to evaluate the safety of BAY 94-9027 in children aged 7 to under 12 years with severe hemophilia A. Participants will receive the treatment over a period of time, and the study will monitor for any side effects or reactions to the medication. The study will also track how often bleeding occurs and how much of the medication is used. This information will help determine how well the treatment works and how safe it is for children in this age group.

Throughout the study, participants will be asked to document their experiences using an electronic diary. This will include recording any infusions of the medication and any bleeding episodes. The study aims to provide valuable information on the use of BAY 94-9027 in managing severe hemophilia A in children, contributing to better treatment options for this condition.

1 joining the study

The study involves children aged 7 to less than 12 years with severe hemophilia A. The condition is characterized by very low levels of a blood clotting protein called factor VIII.

Participants must have been previously treated with factor VIII concentrates for at least 50 exposure days.

2 treatment administration

The medication used in the study is called BAY 94-9027, also known as damoctocog alfa pegol. It is administered as a solution for injection.

The medication is given through an intravenous route, meaning it is injected directly into a vein.

3 treatment schedule

The study involves regular infusions of the medication to prevent and treat bleeding episodes.

Participants and their caregivers will be trained to use an electronic patient diary to document each infusion.

4 monitoring and safety assessment

The main objective of the study is to assess the safety and tolerability of the treatment.

Participants will be monitored for any adverse reactions, including hypersensitivity and loss of efficacy during the first four exposure days.

5 study duration

The study is expected to continue until July 2025.

Participants will be involved in the study for the duration necessary to gather sufficient safety and efficacy data.

Who Can Join the Study?

The participant must be between 7 and less than 12 years old when the parent or guardian signs the informed consent.
The participant must have a known medical history of severe hemophilia A. This means the participant’s own FVIII activity (a measure of blood clotting) is less than 1%. This should be based on reliable past medical records. If no reliable records are available, the FVIII activity must be measured during screening after a 48-72 hour period without treatment, depending on the previous product used.
The participant must be male.
The participant must have been previously treated with FVIII concentrate(s) (a type of treatment for hemophilia) for at least 50 exposure days. This means the participant has received this treatment on at least 50 different days.
The participant must understand the study and, if appropriate for his age, sign an informed assent. The parent(s) or guardian(s) must be able to give signed informed consent and comply with the requirements and restrictions in the informed consent form and the study protocol.
The participant and/or parents/caregivers must be willing and able to complete training in using the electronic patient diary and document infusions during the study.

Who Cannot Join the Study?

Patients who have not been previously treated for severe hemophilia A. This means they must have received treatment for this condition before.
Patients who are not within the age range of 7 to less than 12 years old.
Female patients, as the study is only for male patients.
Patients who are considered part of a vulnerable population, which means they might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Oslo Universitetssykehus HF	Oslo	Norway

Other Sites

Site Name	City	Country	Status
Ospedale Pediatrico Bambino Gesu’	Rome	Italy

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	23.03.2022
Norway	Not recruiting	23.03.2022

Trial locations

Investigated drugs:

Damoctocog Alfa Pegol

BAY 94-9027 is a medication used in this clinical trial to help manage severe hemophilia A in children aged 7 to under 12 years. It is designed to replace a missing protein in the blood that helps with clotting, which is crucial for preventing and controlling bleeding episodes. The study aims to evaluate the safety and how well children tolerate this treatment when used regularly to prevent bleeding or to treat bleeding episodes when they occur.

Investigated diseases:

Factor VIII deficiency

Severe Hemophilia A – This is a genetic disorder where the blood does not clot properly due to a deficiency of clotting factor VIII. It is characterized by spontaneous bleeding episodes, which can occur internally or externally, often without any apparent cause. The condition is typically diagnosed in early childhood, especially when a child experiences prolonged bleeding from minor injuries or surgeries. Over time, repeated bleeding into joints and muscles can lead to chronic pain and joint damage. Individuals with severe hemophilia A have less than 1% of the normal level of factor VIII activity in their blood. This deficiency makes them prone to frequent and severe bleeding episodes.

Trial ID:

2023-504388-18-00

Protocol code:

BAY 94-9027/21824

NCT ID:

NCT05147662

Trial Phase:

Therapeutic confirmatory (Phase III)

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Safety Study of Damoctocog Alfa Pegol for Children Aged 7 to 11 with Severe Hemophilia A

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?