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

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What is this study about?

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.

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.

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.

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.

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.

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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Hopital Beaujon Clichy France
Ayeyngcgxq Pgkradas Hjcftgil Dr Mkixsthrz Marseille France
Cis Kvznmrc Bmpxdtj Le Kremlin-Bicetre France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
02.04.2024
France France
Recruiting
02.04.2024
Italy Italy
Recruiting
02.04.2024
The Netherlands The Netherlands
Recruiting
02.04.2024

Trial locations

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.

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.

Trial ID:
2023-505995-31-00
Protocol code:
HMB-001-CL101
NCT ID:
NCT06211634
Trial Phase:
Phase I and Phase II (Integrated) – First administration to humans

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