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Study on the Safety and Effects of HMB-001 for Patients with Glanzmann Thrombasthenia

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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 a rare bleeding disorder called Glanzmann Thrombasthenia. This condition affects the blood's ability to clot properly, leading to frequent and sometimes severe bleeding episodes. The trial will investigate a new treatment called HMB-001, which is administered as a solution for injection or infusion under the skin. The purpose of the study is to assess the safety and tolerability of HMB-001 and to explore its potential to reduce the frequency and severity of bleeding episodes in individuals with Glanzmann Thrombasthenia.

The study is divided into different parts. Initially, participants will receive single and multiple doses of HMB-001 to determine the safest and most effective dose levels. Following this, the study will continue with repeated doses to confirm the safety and tolerability of the treatment. Throughout the trial, researchers will monitor participants for any side effects and changes in their health, as well as measure the treatment's impact on bleeding frequency and severity.

Participants will undergo regular health assessments, including physical exams and laboratory tests, to ensure their well-being during the study. The trial aims to provide valuable information on the potential benefits of HMB-001 for managing Glanzmann Thrombasthenia, with the hope of improving the quality of life for those affected by this condition.

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
Initial treatment phase
The study begins with the administration of HMB-001, a solution for injection or infusion, given subcutaneously. This phase is designed to determine the safety and tolerability of the medication.
Participants will receive single and multiple ascending doses of HMB-001 to establish the appropriate dose levels and intervals for further study.
Step 2
Monitoring and assessment
Throughout the trial, participants will undergo regular monitoring to assess safety. This includes checking for any adverse effects, changes in physical health, vital signs, and laboratory test results.
Electrocardiogram (ECG) parameters will also be evaluated to ensure heart health is maintained.
Step 3
Repeat dosing phase
In this phase, participants will receive repeated doses of HMB-001 to confirm its safety and tolerability.
The study will also estimate the preliminary effect of HMB-001 on reducing the frequency and severity of bleeding events.
Step 4
Evaluation of treatment effects
The study will measure the concentration of HMB-001 in the blood to understand how the body processes the medication.
Additional assessments will include the formation of anti-drug antibodies and changes in bleeding-related parameters.
Step 5
Quality of life assessment
Participants will complete questionnaires to evaluate changes in quality of life, including aspects such as daily activities and work productivity.
These assessments will help determine the overall impact of HMB-001 on participants' well-being.

Who can join the trial?

10 criteria

Participants must be between 18 and 65 years old.
Participants must have a condition called Glanzmann Thrombasthenia, which can be confirmed by specific tests or genetic diagnosis.
Participants must be able to understand and agree to the study procedures and provide informed consent.
Participants must have stable vital signs, including a resting heart rate of 105 beats per minute or less and blood pressure within a specific range.
Women who can have children must have a negative pregnancy test before starting the study and agree to use effective birth control methods during the study and for six months after.
Men who can father children must agree to use effective birth control methods and avoid donating sperm during the study and for six months after.
Participants must have certain organ functions within acceptable limits, as shown by specific blood tests.
Participants must have a hemoglobin level greater than 85 g/L and a platelet count greater than 120 x 10⁹/L.
Participants in Part B must have experienced bleeding symptoms related to Glanzmann Thrombasthenia, with an average of two bleeding events per week and at least one significant bleeding event in the past year.
Participants who complete Part B may be eligible for Part C if they meet the necessary requirements and are considered suitable by the study investigator.

Who cannot join the trial?

10 criteria

Patients who have any other serious health condition that might interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients who are pregnant or breastfeeding.
Patients who have a history of allergic reactions to similar medications.
Patients who are unable to follow the study procedures or attend the required visits.
Patients who have a history of substance abuse or alcohol dependency.
Patients who have an infection that requires treatment with antibiotics.
Patients who have a condition that affects their immune system, making them more prone to infections.
Patients who have a bleeding disorder other than **Glanzmann Thrombasthenia**.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Assistance Publique Hopitaux De Marseille
Marseille, France
Responsive
Katholieke Universiteit te Leuven
Leuven, Belgium
Responsive
Universitair Medisch Centrum Utrecht
Utrecht, The Netherlands

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Investigated drugs

HMB-001 is an investigational medication being studied for its potential to help people with Glanzmann Thrombasthenia, a rare bleeding disorder. The trial aims to assess how safe and tolerable this medication is for patients. Researchers are also looking to find the best dose and how often it should be given. In later parts of the study, they will check if HMB-001 can reduce how often and how severely bleeding occurs in patients.

Hmb-001

What is already known about the treatment

HMB-001 – This medication is administered intravenously, meaning it is given directly into a vein. It is currently in the early stages of clinical trials, specifically Phase 1/2, to assess its safety, tolerability, and effectiveness. The main therapeutic indication for HMB-001 is Glanzmann Thrombasthenia, a rare bleeding disorder. At the molecular level, HMB-001 is designed to reduce bleeding frequency and severity by targeting specific pathways involved in platelet function. It falls under the pharmacological classification of investigational drugs, as it is still being studied for its potential benefits and risks.

Investigated diseases

Glanzmann Thrombasthenia – This is a rare genetic disorder that affects the blood's ability to clot properly. It is caused by a deficiency or dysfunction of a protein on the surface of platelets, which are cells that help stop bleeding by forming clots. Individuals with this condition often experience frequent nosebleeds, easy bruising, and prolonged bleeding from minor injuries. Women may have heavy menstrual bleeding, and there can be excessive bleeding after surgery or dental work. The severity of symptoms can vary widely among those affected. The condition is usually diagnosed in childhood, but symptoms can appear at any age.