Study on the Effectiveness and Safety of Vonicog Alfa with or without Octocog Alfa for Children with Severe von Willebrand Disease Experiencing Bleeding or Undergoing Surgery

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What is this study about?

This clinical trial is focused on studying a condition called Severe von Willebrand Disease in children. This is a hereditary bleeding disorder where the blood does not clot properly, leading to excessive bleeding. The study is testing the effectiveness, safety, and tolerability of a treatment called Recombinant Von Willebrand Factor (rVWF), which may be used with or without another medication called Advate. These treatments are given as injections to help control bleeding episodes and to assist during surgeries, whether they are planned or emergencies.

The purpose of the study is to evaluate how well rVWF works in managing bleeding events in children under 18 years old who have severe von Willebrand Disease. The study will also look at how safe the treatment is and how well children tolerate it. Participants in the study will receive the treatment over a period of time, and their response to the treatment will be monitored. Some children may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment.

Throughout the study, researchers will collect information on how effective the treatment is in stopping bleeding, how many doses are needed, and any side effects that may occur. They will also study how the body processes the treatment, which is known as pharmacokinetics. This includes understanding how long the treatment stays in the body and how it is broken down. The study aims to provide valuable information that could improve the management of severe von Willebrand Disease in children.

1 joining the study

Upon joining the study, the patient is diagnosed with severe von Willebrand Disease, a condition affecting blood clotting.

The patient is under 18 years of age and has provided necessary consent to participate.

2 treatment initiation

The patient begins treatment with recombinant Von Willebrand Factor (rVWF), which may be administered with or without ADVATE.

The treatment is designed to manage and control bleeding episodes.

3 medication administration

The medications are provided as a solution for injection and are administered through intravenous use.

The specific products used include ADVATE 1000 IU, VEYVONDI 1300 IU, VEYVONDI 650 IU, and ADVATE 500 IU.

4 monitoring and evaluation

The effectiveness of the treatment is evaluated based on the control of bleeding episodes, using a 4-point scale to measure success.

The safety of the treatment is monitored by assessing any adverse events or reactions.

5 surgery considerations

For patients undergoing surgery, the treatment’s effectiveness is assessed 24 hours after the last infusion.

The evaluation is conducted by a hematologist using a 4-point scale.

6 pharmacokinetics study

The study includes an analysis of how the medication is processed in the body, measuring factors like concentration over time and clearance rates.

This analysis helps understand the medication’s behavior in different age groups.

Who Can Join the Study?

The child must have a diagnosis of severe von Willebrand Disease (VWD). This means their blood has very low levels of a protein called von Willebrand factor (VWF), which helps with blood clotting.
The child must be younger than 18 years old at the time of joining the study.
The child, if old enough, must agree to participate, and their parent or guardian must give permission for them to join the study.
If the child is a girl who can become pregnant, she must have a negative pregnancy test before joining the study.
If the child is a girl who can become pregnant, she must agree to use birth control during the study.
The child and their parent or guardian must be willing and able to follow the study rules and requirements.
If the child has been treated before, they must have had at least one bleeding event in the past year that needed treatment with a VWF product, and they must have used VWF treatment on at least three different days in the past.
If the child has not been treated before, they must not have received any VWF treatment in the past.

Who Cannot Join the Study?

Children who do not have a diagnosis of severe hereditary von Willebrand Disease cannot participate. This is a genetic condition that affects blood clotting.
Children who are 18 years or older are not eligible to join the study.
Children who have any other medical conditions that might interfere with the study’s treatment or results may be excluded.
Children who are unable to follow the study procedures or attend required visits may not be able to participate.
Children who have had a recent major surgery or are planning to have surgery soon might not be eligible.
Children who are currently participating in another clinical trial may not be allowed to join this study.
Children who have allergies or reactions to the study medications or similar treatments may be excluded.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Medical University Of Vienna	Vienna	Austria
Centre Hospitalier Regional Et Universitaire De Brest	Brest	France
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Azienda Ospedaliera Santobono Pausilipon	Naples	Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Hopital Beaujon	Clichy	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Centre Hospitalier Universitaire De Caen Normandie	Caen	France

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	28.10.2016
Belgium	Not recruiting	28.10.2016
France	Not recruiting	28.10.2016
Italy	Not recruiting	28.10.2016
Spain	Not recruiting	28.10.2016

Trial locations

Investigated drugs:

rVWF is a medication used in this clinical trial to help manage bleeding episodes in children with severe von Willebrand Disease. It works by replacing the von Willebrand factor, a protein that is missing or not functioning properly in these patients, which is essential for blood clotting.

ADVATE is another medication used in combination with rVWF in this trial. It is a form of factor VIII, a protein that helps blood to clot. When used together with rVWF, it aims to improve the control of bleeding episodes in patients with von Willebrand Disease.

Hereditary Severe von Willebrand Disease – This is a genetic disorder that affects the blood’s ability to clot properly. It is caused by a deficiency or dysfunction of von Willebrand factor, a protein that is crucial for blood clotting. Individuals with this condition experience frequent and prolonged bleeding episodes, which can occur spontaneously or after an injury. The disease is present from birth and can vary in severity, with some individuals experiencing mild symptoms and others having severe bleeding issues. In children, the condition can lead to challenges in managing bleeding during everyday activities and minor injuries. The progression of the disease involves ongoing bleeding risks, requiring careful management to prevent complications.

Trial ID:

2023-509769-18-00

Protocol code:

071102

NCT ID:

NCT02932618

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effectiveness and Safety of Vonicog Alfa with or without Octocog Alfa for Children with Severe von Willebrand Disease Experiencing Bleeding or Undergoing Surgery

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?