Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease

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What is this study about?

This clinical trial is focused on studying the effects of a medication called Emicizumab in patients with a rare bleeding disorder known as Type 3 Von Willebrand Disease. This condition is characterized by a severe deficiency of a protein called von Willebrand factor, which is essential for blood clotting. The study aims to evaluate how well Emicizumab works in preventing bleeding episodes when used regularly, compared to the usual treatment that is given only when bleeding occurs.

Participants in the study will receive Emicizumab as a preventive treatment, which means it will be administered on a regular schedule to help prevent bleeding episodes. The study will monitor the number of bleeding events over time, as well as any side effects or reactions to the medication. The trial will also assess the overall safety of Emicizumab and its impact on the participants’ health, including any changes in their physical condition and laboratory test results.

The study will take place over several years, allowing researchers to gather comprehensive data on the long-term effects of Emicizumab in managing Type 3 Von Willebrand Disease. This research is important for understanding how this medication can improve the quality of life for individuals with this challenging condition by reducing the frequency and severity of bleeding episodes.

1 joining the study

Upon joining the study, you will be introduced to the trial procedures and objectives. You will receive detailed information about the study, including the purpose, duration, and what is expected from you as a participant.

2 initial assessment

An initial assessment will be conducted to evaluate your current health status. This may include a physical examination, blood tests, and a review of your medical history to confirm your eligibility for the study.

3 medication administration

You will receive the study medication, emicizumab, which is administered as a solution for injection. The medication is given subcutaneously, meaning it is injected under the skin. The dosage and frequency will be determined by the study protocol and your specific needs.

4 regular follow-up visits

You will attend regular follow-up visits to monitor your response to the medication and any side effects. These visits will include physical examinations, blood tests, and discussions about any symptoms or concerns you may have.

5 monitoring and reporting

Throughout the study, you will be asked to report any bleeding episodes, side effects, or other health changes. This information is crucial for assessing the safety and effectiveness of the medication.

6 end of study evaluation

At the end of the study, a final evaluation will be conducted. This will include a comprehensive assessment of your health and a review of your experience during the trial. The results will contribute to the overall findings of the study.

Who Can Join the Study?

Must be at least 2 years old at the time of signing the consent form.
Must have adequate hematological function, which means the blood is working well enough for the study.
Must have a history of using both on-demand (as needed) and prophylactic (preventive) standard care treatments.
If able to have children, must agree to follow the contraception requirements to prevent pregnancy during the study.
Must have a confirmed diagnosis of Type 3 Von Willebrand Disease (VWD), based on specific blood tests that measure VWF and FVIII levels.
Must have information about the status of VWF inhibitor, which is a substance that can affect how the treatment works.

Who Cannot Join the Study?

Patients with any other bleeding disorders besides Type 3 Von Willebrand Disease cannot participate.
Patients who have had a recent major surgery or are planning to have surgery during the study period are not eligible.
Patients who are currently participating in another clinical trial are excluded.
Patients with a known allergy or severe reaction to emicizumab or any of its ingredients cannot join the study. Emicizumab is a medication used to prevent bleeding episodes.
Patients with severe liver or kidney disease are not eligible to participate.
Pregnant or breastfeeding women cannot take part in the study.
Patients who are unable to comply with the study procedures or follow-up visits are excluded.
Patients with any condition that the study doctors believe would make participation unsafe or affect the study results are not eligible.

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
Hopital Necker Enfants Malades	Paris	France
Centre Hospitalier Universitaire De Lille	Lille	France
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Instytut Hematologii I Transfuzjologii	Warsaw	Poland
Virgen del Rocío University Hospital	Sevilla	Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Rheinische Friedrich-Wilhelms-Universitaet Bonn	Bonn	Germany
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Grkoliobieeljgalc Rmsphgibha Aaxaxzfaaspihmbbdta Dmk mdcy Hadcgsiht Rocn Fvsceeskwge fghq Tbljpcqqulwiyruzfgk Hasvqnbmxojxkwgp Diu mjgx Szwei Hbxexnj Fuebtavhhoa fpiz Tjyerkazisadvhlyhsj Habunecapzdmvvbk Dtq miol Gqtbgrgq Ksradip Foqwyubw fhjk Lpfaaugidpyrkytsjjnv Hpgielvkkxrolsbr	Duisburg	Germany
Efkasoy Uimujcxscfjk Msvlcye Cbrnesu Rwzuechzd (ieuubti Mye	Rotterdam	The Netherlands
Gqxdmv Ubyirtdpcl Fbbuuhuue	Frankfurt	Germany
Uzcrvxcgvb Diynj Szdss Dz Ruvj Li Swlwfzze	Rome	Italy
Fqkygixdz Para Lx Ilexqvxkhufcf Btjbviqup Djh Hjdhtkdq Ufimbwrjwyuxg Lh Pzf	Madrid	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	01.05.2025
France	Recruiting	01.05.2025
Germany	Recruiting	01.05.2025
Italy	Recruiting	01.05.2025
Poland	Recruiting	01.05.2025
Spain	Recruiting	01.05.2025
Sweden	Not yet recruiting	01.05.2025
The Netherlands	Recruiting	01.05.2025

Trial locations

Investigated drugs:

Emicizumab is a medication used in this clinical trial to help prevent bleeding in patients with Type 3 Von Willebrand Disease. It is given on a regular schedule to reduce the number of bleeding episodes. Emicizumab works by helping the blood to clot more effectively, which is particularly important for people who have a bleeding disorder like Von Willebrand Disease. This medication is being tested to see how well it works compared to the usual treatment that is given only when bleeding occurs.

Investigated diseases:

Von Willebrand's disease

Type 3 Von Willebrand Disease – This is the most severe form of Von Willebrand Disease, a genetic bleeding disorder caused by a deficiency or dysfunction of the von Willebrand factor, a protein crucial for blood clotting. Individuals with this condition have very low or undetectable levels of von Willebrand factor and often have low levels of factor VIII, another protein important for clotting. The disease is characterized by frequent and severe bleeding episodes, which can occur spontaneously or after minor injuries. Bleeding can affect various parts of the body, including the skin, mucous membranes, joints, and muscles. Women with this condition may experience heavy menstrual bleeding. The progression of the disease involves recurrent bleeding episodes that can lead to complications if not managed properly.

Trial ID:

2024-515622-80-00

Protocol code:

WP45338

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?