Ongoing Clinical Trials for Glanzmann’s Disease
There is currently 1 ongoing clinical trial investigating new treatments for Glanzmann’s disease (also known as Glanzmann Thrombasthenia), a rare genetic bleeding disorder. This trial is testing an investigational medication called HMB-001 and is being conducted across multiple European countries.
Clinical trial locations
- Belgium
- France
- Italy
- Netherlands
Study on the Safety and Effects of HMB-001 for Patients with Glanzmann Thrombasthenia
This trial is investigating HMB-001, an investigational medication administered as an injection or infusion under the skin. The study is designed to evaluate both the safety and potential benefits of this new treatment for people living with Glanzmann Thrombasthenia.
Main focus and goals: The trial aims to determine whether HMB-001 is safe and well-tolerated by patients. Researchers want to find the most appropriate dose and dosing schedule for the medication. Beyond safety, the study will explore whether HMB-001 can reduce how often bleeding episodes occur and how severe they are. The trial is structured in multiple phases, starting with single doses and progressing to repeated doses to confirm safety and gather information about effectiveness.
Who can participate: The trial is looking for adults between 18 and 65 years old who have been diagnosed with Glanzmann Thrombasthenia, confirmed either through specific blood tests or genetic testing. Participants need to have stable vital signs, including a resting heart rate of 105 beats per minute or less and blood pressure within an acceptable range. Their organ function must be within normal limits based on blood tests, and they must have a hemoglobin level greater than 85 g/L and a platelet count above 120 x 109/L.
For certain parts of the study, participants must have experienced bleeding symptoms, with an average of two bleeding events per week and at least one significant bleeding event in the past year. Women of childbearing potential must have a negative pregnancy test and agree to use effective birth control during the study and for six months afterward. Men who can father children must also use birth control and avoid donating sperm during this period.
Who cannot participate: The trial excludes people with other serious health conditions that could interfere with the study results or those currently participating in another clinical trial. Patients who have recently had major surgery or are planning surgery during the study period cannot join. Pregnant or breastfeeding women are not eligible. Anyone with a history of allergic reactions to similar medications, substance abuse, alcohol dependency, or infections requiring antibiotic treatment cannot participate. The trial also excludes people with conditions affecting their immune system or those with bleeding disorders other than Glanzmann Thrombasthenia.
What happens during the trial: Participants will be closely monitored throughout the study. This includes regular physical examinations, vital sign checks, laboratory tests, and electrocardiogram assessments to ensure heart health. The study begins with administering single and multiple doses of HMB-001 to establish safe dose levels. This is followed by a repeat dosing phase where participants receive the medication multiple times to confirm safety and assess its effects on bleeding frequency and severity.
Researchers will measure the concentration of HMB-001 in the blood to understand how the body processes the medication. They will also check for the formation of antibodies against the drug and monitor changes in bleeding-related parameters. Throughout the trial, participants will complete questionnaires about their quality of life, including questions about daily activities and work productivity, to understand the overall impact of the treatment on their well-being.
Summary
Currently, there is one active clinical trial available for patients with Glanzmann’s disease across Europe. This trial is being conducted in four countries: Belgium, France, Italy, and the Netherlands, providing multiple geographic options for potential participants. The study focuses exclusively on HMB-001, a new investigational medication designed to target the underlying mechanisms of bleeding in this rare disorder.
This Phase 1/2 trial represents an important step in developing new treatment options for people with Glanzmann Thrombasthenia, a condition that significantly affects quality of life due to frequent and sometimes severe bleeding episodes. The study’s multi-phase approach allows researchers to carefully establish safety before examining effectiveness, which is standard practice for investigational medications.
Patients interested in participating should discuss the trial with their healthcare provider to determine if they meet the eligibility criteria and to understand what participation would involve. As this is currently the only ongoing trial for this condition, it may represent an important opportunity for eligible patients to access a potential new treatment while contributing to medical research.
