HMB-001 for Thrombasthenia

Not currently recruiting at 20 trial locations

Overseen By Andrew Law

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Hemab ApS

Must not be taking: Estrogen medications

Disqualifiers: Severe infection, Thrombosis, Cardiovascular disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called HMB-001 for individuals with Glanzmann Thrombasthenia, a rare bleeding disorder that impairs blood clotting. The researchers aim to determine the safety and effectiveness of HMB-001 and identify the optimal dosing to reduce the frequency and severity of bleeding episodes. The trial includes different parts that test single and multiple doses. It seeks participants who regularly experience bleeding symptoms, such as two or more bleeding events per week and at least one severe bleed in the past year. As a Phase 1, Phase 2 trial, this research focuses on understanding how HMB-001 functions in people and measuring its effectiveness in an initial, smaller group.

Do I need to stop taking my current medications to join the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, women using estrogen-containing medications must stop 8 weeks before and after the study drug. It's best to discuss your specific medications with the trial team.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, women using estrogen-containing medications must stop 8 weeks before and after the study drug. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that HMB-001 is likely to be safe for humans?

Research has shown that HMB-001 has been tested for safety and is generally well-tolerated. In earlier studies, individuals with Glanzmann Thrombasthenia experienced a significant reduction in bleeding episodes, with more than a 50% decrease at all dose levels tested. This suggests the treatment might help reduce bleeding, which is promising.

So far, studies have not identified major safety issues, but it is important to remember that this treatment is still under investigation. While early results appear promising, more information is needed to confirm its safety and any possible side effects. Participants in these studies did not report serious side effects, which is encouraging for those considering joining the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for thrombasthenia focus on managing symptoms with platelet transfusions or drugs like antifibrinolytics to prevent bleeding. But HMB-001 is different. Researchers are excited because it introduces a new mechanism of action that targets the underlying platelet dysfunction directly, potentially improving clotting without the need for frequent transfusions. This could mean fewer side effects and a more straightforward treatment regimen, making life easier for those affected by thrombasthenia.

What evidence suggests that HMB-001 might be an effective treatment for Glanzmann Thrombasthenia?

Research has shown that HMB-001 yields promising results in reducing bleeding for individuals with Glanzmann Thrombasthenia. Studies found that treatment with HMB-001 led to more than a 50% reduction in treated bleeding episodes at all tested doses. This trial will test single or multiple ascending doses of HMB-001 to further evaluate its effectiveness and safety. HMB-001 is a unique antibody that targets two different areas to help reduce bleeding. Early results are encouraging, but more research is needed to fully understand its benefits and safety. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for individuals with Glanzmann Thrombasthenia, a rare bleeding disorder. Participants should be willing to undergo various tests and follow-ups. Specific details on who can or cannot participate are not provided here, but typically include factors like age, health status, and the severity of their condition.

Inclusion Criteria

I have Glanzmann thrombasthenia with confirmed abnormal platelet function and a specific receptor deficiency.

Vital signs within specified ranges at Screening

My organ functions meet the required health standards.

Exclusion Criteria

History of clinically significant hypersensitivity associated with monoclonal antibody therapies

Positive test for certain infections or conditions

Received investigational medication in another clinical study within 5 half-lives before administration of study drug

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Run-in Observation

Optional observation period before treatment to monitor baseline conditions

2-4 weeks

Treatment Part A

Participants receive differing singular doses of HMB-001 to assess safety and tolerability

6 months

Follow-up Part A

Participants are monitored for safety and effectiveness after treatment

8 weeks

Treatment Part B

Participants receive multiple doses of HMB-001 over a 3-month period to assess safety and efficacy

3 months

Safety Follow-up Part B

Participants are monitored for safety following the last dose of HMB-001

Treatment Part C

Participants from Part B receive HMB-001 over a 9-month period to further assess safety and efficacy

9 months

Safety Follow-up Part C

Participants are monitored for safety following the last dose of HMB-001

What Are the Treatments Tested in This Trial?

Interventions

• HMB-001

Trial Overview The study is testing HMB-001's safety, how well it's tolerated by patients' bodies (tolerability), how it moves through and affects the body (pharmacokinetics/pharmacodynamics), and its effectiveness in preventing bleeds in Glanzmann Thrombasthenia patients. It has three parts: Part A tests single doses; Part B multiple doses over 3 months; Part C continues treatment from Part B for about 9 months.

How Is the Trial Designed?

Group I: Single or Multiple ascending dose of HMB-001 Experimental Treatment 1 Intervention

Open-label, single or multiple ascending dose of HMB-001

Who Is Running the Clinical Trial?

Hemab ApS

Lead Sponsor

Trials

6

Recruited

1,100+

Published Research Related to This Trial

The study developed a new ultrasound contrast agent using perfluorobutane-filled microbubbles conjugated with thrombin-sensitive peptides, which can effectively detect acute deep vein thrombosis (DVT).

When tested, the contrast agent retained 91.7% of its signal in the presence of thrombin, indicating its effectiveness in identifying acute thrombosis, while only 16.7% of the signal was retained when a thrombin inhibitor was present, demonstrating the specificity of the agent.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Thrombin-Activatable Microbubbles as Potential Ultrasound Contrast Agents for the Detection of Acute Thrombosis. Lux, J., Vezeridis, AM., Hoyt, K., et al.[2022]

In a study involving 5142 patients, rivaroxaban demonstrated a lower incidence of major bleeding (0.8%) compared to standard anticoagulation therapy (2.1%), suggesting it may be a safer option for treating deep-vein thrombosis.

Rivaroxaban also showed comparable effectiveness in preventing recurrent venous thromboembolism (1.4% vs. 2.3% for standard therapy), indicating it is an effective alternative in routine clinical practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and effectiveness of oral rivaroxaban versus standard anticoagulation for the treatment of symptomatic deep-vein thrombosis (XALIA): an international, prospective, non-interventional study. Ageno, W., Mantovani, LG., Haas, S., et al.[2022]

New oral anticoagulants (NOAs) like dabigatran, rivaroxaban, apixaban, and edoxaban are as effective as vitamin K antagonists (VKAs) in preventing recurrent venous thromboembolism (VTE), with a risk ratio of 0.89 based on a review of 10 randomized controlled trials involving nearly 38,000 patients.

NOAs have a significantly lower incidence of major bleeding compared to VKAs (1.08% vs. 1.73%), leading to a net clinical benefit favoring NOAs, with a risk ratio of 0.79, indicating they are a safer option for VTE management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Editor's Choice - efficacy and safety of the new oral anticoagulants dabigatran, rivaroxaban, apixaban, and edoxaban in the treatment and secondary prevention of venous thromboembolism: a systematic review and meta-analysis of phase III trials. Kakkos, SK., Kirkilesis, GI., Tsolakis, IA.[2018]

Dabigatran etexilate shows similar relationships between plasma concentrations and coagulation parameters (aPTT, dTT, ECT) in both children and adults, indicating consistent efficacy across age groups.

In children under 6 months, baseline aPTT and ECT values were slightly higher than in older children, but overall, developmental changes in the hemostatic system do not significantly affect the response to dabigatran.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anticoagulant Effects of Dabigatran on Coagulation Laboratory Parameters in Pediatric Patients: Combined Data from Five Pediatric Clinical Trials. Mitchell, LG., Röshammar, D., Huang, F., et al.[2022]

Endovascular treatment using self-expanding nitinol stents for patients with iliofemoral venous obstruction showed a primary patency rate of 79% at 12 months, indicating effective long-term vessel openness, especially in non-thrombotic iliac vein lesions (NIVL).

A significant majority of patients (67%) experienced clinical improvement, as measured by the Villalta score, suggesting that this treatment not only maintains venous patency but also enhances patient quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Endovascular treatment of post-thrombotic and non-thrombotic iliofemoral venous outflow obstructions with self-expanding nitinol stents. Stuck, AK., Reich, T., Engelberger, RP., et al.[2018]

In a study of 499 patients with acute symptomatic venous thromboembolism (VTE) treated with rivaroxaban, the major and clinically relevant bleeding rate was lower (5.4%) compared to those treated with vitamin K antagonists (VKAs) and heparin/fondaparinux, indicating rivaroxaban's safety profile is favorable.

Rivaroxaban also demonstrated a significantly lower rate of recurrent VTE (1.4%) compared to VKAs (3.1%) and heparin/fondaparinux (11.6%), suggesting it is an effective treatment option for preventing VTE recurrence.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rivaroxaban versus standard anticoagulation for symptomatic venous thromboembolism (REMOTEV observational study): Analysis of 6-month outcomes. Gaertner, S., Cordeanu, EM., Nouri, S., et al.[2017]

In patients undergoing intrauterine hypertonic saline infusion, levels of Fibrinopeptide A (FPA) rose quickly after treatment, indicating immediate thrombin activity that cleaves FPA from fibrinogen, while TIFPB levels peaked at 4 hours, suggesting ongoing fibrin degradation processes.

The study reveals a complex sequence of proteolysis where plasmin acts on fibrin I to regulate the formation of fibrin II, highlighting the dynamic interactions between coagulation and fibrinolysis during and after the procedure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sequence of fibrinogen proteolysis and platelet release after intrauterine infusion of hypertonic saline. Nossel, HL., Wasser, J., Kaplan, KL., et al.[2018]

In a review of 16 studies involving over 22,000 patients, standard-intensity vitamin K antagonists (VKAs) and direct oral anticoagulants (DOACs) were found to be more effective than aspirin in preventing recurrent venous thromboembolism (VTE) beyond 3 months.

While VKAs showed a higher risk of major bleeding, they were also associated with a lower risk of all-cause mortality compared to placebo, suggesting that despite safety concerns, VKAs may offer significant benefits in long-term VTE management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Extended treatment of venous thromboembolism: a systematic review and network meta-analysis. Wang, KL., van Es, N., Cameron, C., et al.[2019]

The review covers various drug therapies aimed at treating venous thromboembolism, which is a condition where blood clots form in veins, highlighting the importance of effective anticoagulant treatments.

It also discusses strategies for managing hypertension and promoting plaque regression, as well as methods to prevent stent thrombosis, which is the formation of clots in stents used to open blocked arteries.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

European society of cardiology congress 2013. Alexander, W.[2021]

The study found that using new oral anticoagulants (NOACs) like rivaroxaban, apixaban, and edoxaban resulted in lower rates of major bleeding and recurrent venous thromboembolism (VTE) compared to standard therapy, indicating improved safety and efficacy.

Overall medical costs were significantly reduced when using NOACs instead of standard therapy, with apixaban showing the greatest cost reduction, suggesting that these medications not only improve patient outcomes but also lower healthcare expenses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of medical costs associated with use of new oral anticoagulants compared with standard therapy among venous thromboembolism patients. Amin, A., Jing, Y., Trocio, J., et al.[2022]

Pulmonary endarterectomy has significantly improved outcomes for patients with chronic thromboembolic disease by reducing morbidity and mortality through the removal of organized thrombus.

Despite advancements in treatment, the underlying causes and mechanisms of chronic thromboembolic disease are still not well understood, highlighting a gap between clinical progress and basic research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Chronic thromboembolic pulmonary hypertension: time for research in pathophysiology to catch up with developments in treatment. Toshner, M., Pepke-Zaba, J.[2021]

A new thrombus-specific ultrasound contrast agent, MRX-408, was developed using a targeted lipid blend that effectively binds to blood clots, enhancing the detection of deep venous thrombosis.

In vitro studies demonstrated that MRX-408 microbubbles are stable and significantly improve ultrasound imaging of blood clots compared to nontargeted microbubbles, suggesting potential for better differentiation between new and old thrombosis in clinical settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

In vitro studies of a new thrombus-specific ultrasound contrast agent. Unger, EC., McCreery, TP., Sweitzer, RH., et al.[2019]

MRX-408, a thrombus-specific ultrasound contrast agent, effectively binds to blood clots rich in activated platelets, enhancing their visibility during ultrasound imaging.

In preliminary studies, MRX-408 improved clot lysis efficiency to 34% when combined with ultrasound treatment, demonstrating its potential to facilitate sonothrombolysis compared to saline, which showed no lysis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Binding and lysing of blood clots using MRX-408. Wu, Y., Unger, EC., McCreery, TP., et al.[2019]

The newly developed nanomedicine, FTIAN, effectively targets and images obstructed blood vessels while also inhibiting thrombus formation, showcasing its dual role as a diagnostic and therapeutic agent.

In mouse models, FTIAN demonstrated significant antithrombotic effects by suppressing thrombus formation when combined with the antiplatelet drug tirofiban, indicating its potential for treating obstructive thrombosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fibrin-Targeted and H2O2-Responsive Nanoparticles as a Theranostics for Thrombosed Vessels. Kang, C., Gwon, S., Song, C., et al.[2019]

New oral anticoagulants like rivaroxaban, apixaban, and dabigatran have been developed to address the limitations of traditional therapies, showing effectiveness in preventing thromboembolic diseases with predictable pharmacokinetics and safety profiles.

These agents have undergone extensive phase 3 testing and are approved for various uses, such as stroke prevention in atrial fibrillation and VTE prevention after major orthopedic surgery, indicating their growing role in modern anticoagulation therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Moving toward a more ideal anticoagulant: the oral direct thrombin and factor Xa inhibitors. King, CS., Holley, AB., Moores, LK.[2017]

Citations

1. hemab.com hemab.com/news-items/hemab-therapeutics-presents-interim-data-from-ongoing-phase-2-study-of-hmb-001-as-first-ever-prophylactic-treatment-in-glanzmann-thrombasthenia-and-preclinical-data-from-hmb-002-in-von-willebrand-disease-at-the-2025-eahad-annual-congress

Hemab Therapeutics Presents Interim Data from Ongoing ... Interim results demonstrate treatment with HMB-001 resulted in >50% reduction in treated bleeds at all tested dose levels.

2. prnewswire.com prnewswire.com/news-releases/hemab-therapeutics-presents-interim-data-from-ongoing-phase-2-study-of-hmb-001-as-first-ever-prophylactic-treatment-in-glanzmann-thrombasthenia-and-preclinical-data-from-hmb-002-in-von-willebrand-disease-at-the-2025-eahad-annual-c-302370216.html

Hemab Therapeutics Presents Interim Data from Ongoing ... Interim results demonstrate treatment with HMB-001 resulted in >50% reduction in treated bleeds at all tested dose levels.

3. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/3953.1/528987/A-Phase-1-2-Study-to-Investigate-the-Safety

A Phase 1/2 Study to Investigate the Safety, Tolerability ... ... Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia. ... Recent natural history data reveals a higher disease burden in GT ...

4. bleeding.org bleeding.org/news/investigational-data-on-vwd-and-glanzmann-thrombasthenia-therapies-presented-at-isth

Hemab Provides Trial Updates for Investigational ... The data presented at ISTH is based on the Velora Pioneer trial program which is designed to evaluate safety, tolerability, pharmacokinetics, ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124082636

A Phase 1/2 Study to Investigate the Safety, Tolerability ... HMB-001, an investigational bispecific antibody, is being trialed for subcutaneously administered prophylaxis in GT.

6. clinicaltrials.ucsd.edu clinicaltrials.ucsd.edu/trial/NCT06211634

Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann ... A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia.