In short
Clinical trials are investigating Hmb-001 in people with Glanzmann Thrombasthenia, a rare bleeding disorder. The study is looking at safety, tolerability, dosing, and early signs of benefit on bleeding. It includes different trial parts to help choose doses and repeat-dose schedules.
Key points
- Clinical trials of Hmb-001 are being done in people with Glanzmann Thrombasthenia, a rare bleeding disorder. The main trial is a Phase 1/2 study that first checks safety and tolerability, then looks for early signs that bleeding may become less frequent or less severe. The study includes Part A to help choose dose levels and dosing intervals, and Parts B and C to test repeat dosing. Researchers are measuring side effects, physical exams, vital signs, lab tests, ECGs, and bleeding rates. The trial is authorised and plans to enroll 41 participants.
Trial overview
The clinical trial NCT06211634 is studying Hmb-001 in people with Glanzmann Thrombasthenia, a rare bleeding disorder. The study is authorised and plans to enroll 41 participants.
This trial is interventional, which means participants receive the study treatment so researchers can observe what happens. The treatment is given by subcutaneous administration, meaning it is given under the skin.
Study design and phase
This is a Phase 1/2 trial. Phase 1 studies mainly focus on safety and tolerability, while Phase 2 studies also look for early signs that a treatment may help.
The study has three parts: Part A, Part B, and Part C. Part A is used to determine safe dose levels and dosing intervals for later parts of the study.
Who can participate
The trial is designed for participants with Glanzmann Thrombasthenia. The source data does not list all eligibility rules, so the full entry criteria are not shown here.
In studies like this, eligibility usually depends on the study’s detailed rules, which are checked by the research team.
What is being measured
The main safety checks include the number of treatment-emergent adverse events, which are new or worsening problems that happen after treatment starts. Researchers also track changes in physical examinations, vital signs, clinical laboratory tests, and ECG results, which measure the heart’s electrical activity.
For Parts B and C, the study also looks at early signs of benefit on bleeding. These measures include the annualized treated bleed rate and the annualized bleed rate, which estimate how often bleeding happens over one year.
The brief study summary also says the trial will estimate the preliminary prophylactic effect of Hmb-001 on bleeding frequency and bleeding severity. “Prophylactic” means used to help prevent bleeding episodes before they happen.
Why the study has parts A, B, and C
Part A is the first step and is focused on finding the dose level or levels and dosing interval or intervals to test later. This helps researchers choose a study plan for the next parts of the trial.
Parts B and C then repeat dosing to confirm the safety and tolerability profile of Hmb-001. These parts also look for early signs that the treatment may lower bleeding frequency and bleeding severity.
What the trial may help researchers learn
This study is designed to answer practical questions about Hmb-001 in a rare bleeding disorder, not to give a final answer about long-term benefit. The main goals are to see whether the treatment can be given safely, how it behaves in the body, and whether it shows early signs of helping with bleeding.
Because the trial is still early, the results will mainly help guide future research rather than prove how well the treatment works on its own.