Not recruitingRare disease

Study on the Effects of BT200 (Rondaptivon Pegol) for Patients with Type 2B von Willebrand Disease

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a treatment for Type 2B von Willebrand Disease, a condition that affects blood clotting and can lead to excessive bleeding. The treatment being tested is called Rondaptivon pegol, also known by its code name BT200. This medication is designed to help manage the symptoms of the disease by affecting certain blood components involved in clotting.

The purpose of the study is to evaluate how BT200 impacts platelet count, which are the cells in the blood that help with clotting, as well as other factors related to bleeding. Participants in the study will receive the treatment through an injection under the skin. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects. The study will last for a period of four weeks, during which the effects of the treatment will be closely monitored.

Throughout the study, researchers will observe changes in the number of bleeding events and measure various blood components to understand how BT200 works in the body. This includes looking at how long the medication stays in the body and its effects on specific proteins involved in blood clotting. The study aims to provide valuable information on the safety and effectiveness of BT200 for people with Type 2B von Willebrand Disease.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, you will be informed about the trial's purpose, procedures, and your role. You will be required to provide informed consent, confirming your understanding and willingness to participate.
Step 2
Initial assessment
An initial assessment will be conducted to evaluate your health status and confirm your eligibility. This may include a review of your medical history, physical examination, and laboratory tests.
Step 3
Treatment phase
During the treatment phase, you will receive the study medication, rondaptivon pegol, administered as a solution for injection/infusion. The medication will be given through subcutaneous use, which means it is injected under the skin.
The dosage, frequency, and duration of administration will be determined by the study protocol and communicated to you by the study team.
Step 4
Monitoring and follow-up
Throughout the trial, regular monitoring will be conducted to assess your response to the treatment and any side effects. This may involve blood tests, questionnaires, and other assessments.
You will be required to report any changes in your health or any bleeding events to the study team.
Step 5
End of study procedures
At the end of the study, a final assessment will be conducted to evaluate your overall health and the effects of the treatment. This may include a physical examination and laboratory tests.
You will be informed about the study's findings and any next steps regarding your health care.

Who can join the trial?

5 criteria

You must have **Type 2B Von Willebrand's Disease (VWD)**, which is a specific type of bleeding disorder.
You must have **thrombocytopenia**, which means having a low platelet count in your blood, and a recent history of bleeding.
You must be **18 years of age or older**.
You must be able to **understand the study and give informed consent**, which means you agree to participate after being fully informed about the study.
You must be able to **cooperate with the study team**, follow the study requirements, and complete all the necessary procedures.

Who cannot join the trial?

4 criteria

Patients with **Von Willebrand's Disease Type 2B** cannot participate.
Patients who are not within the specified age range cannot participate.
Patients who are not part of the specified clinical trial group cannot participate.
Patients who are part of a vulnerable population cannot participate.

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Verified sites

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Verified
Medical University Of Vienna
Vienna, Austria

Trial locations

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Investigated drugs

BT200 (rondaptivon pegol) is a medication being studied for its potential to help people with a specific type of bleeding disorder called Type 2B von Willebrand disease. This condition affects how blood clots, leading to excessive bleeding. BT200 is designed to improve the function of certain proteins in the blood that are important for clotting. By doing so, it may help increase the number of platelets, which are small blood cells that help stop bleeding, and improve other blood clotting factors. The goal of using BT200 in this trial is to see if it can reduce bleeding episodes and improve overall blood clotting in patients with this condition.

Rondaptivon Pegol

What is already known about the treatment

BT200 (rondaptivon pegol) – BT200 is administered through an injection, which means it is given directly into the body using a needle. Currently, this medication is being studied in clinical trials to determine its effectiveness and safety for treating patients with Type 2B von Willebrand's Disease, a rare bleeding disorder. The main goal of BT200 is to help manage bleeding by affecting certain blood components like platelets and specific proteins involved in clotting. It works at the molecular level by interacting with these blood components to improve their function and reduce bleeding episodes. BT200 is classified as a therapeutic agent specifically designed for blood disorders.

Investigated diseases

Von Willebrand's Disease Type 2B – This is a genetic bleeding disorder caused by a defect in the von Willebrand factor (VWF), a protein that helps blood to clot. In Type 2B, the VWF has an increased tendency to bind to platelets, which can lead to a reduction in platelet count. This abnormal interaction causes the platelets to clump together in the bloodstream, which can result in bleeding episodes. The disease is characterized by spontaneous bleeding, such as nosebleeds, gum bleeding, or heavy menstrual periods. The severity of bleeding can vary among individuals, and it may be triggered by surgery or injury. The condition is typically inherited in an autosomal dominant pattern, meaning one copy of the altered gene in each cell is sufficient to cause the disorder.