NCT06754852 A Study Assessing HMB-002 in Participants With Von Willebrand Disease
| NCT ID | NCT06754852 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Hemab ApS |
| Condition | Von Willebrand Disease (VWD) |
| Study Type | INTERVENTIONAL |
| Enrollment | 108 participants |
| Start Date | 2025-02-06 |
| Primary Completion | 2027-07 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 108 participants in total. It began in 2025-02-06 with a primary completion date of 2027-07.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is a first-in-human (FIH), Phase 1/2, open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study of HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) design to establish safety, tolerability, PK, and PD effect. In Part B of the study, the safety and tolerability of repeat dosing will be established prior to cohort expansion to explore efficacy.
Eligibility Criteria
Inclusion Criteria: 1. Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations. 2. Has an understanding, ability, and willingness to comply with study procedures and restrictions. 3. ≥18 and \<65 years. 4. Weight 50 to 110 kg, inclusive. 5. Congenital Type 1 VWD, Type 1C and Type 2A VWD diagnosis as documented by laboratory results for VWF antigen and activity. 6. Vital signs are within the following ranges at Screening: 1. Resting pulse rate ≤105 bpm 2. Blood pressure (BP): * Systolic blood pressure: 90 - 140 mmHg * Diastolic blood pressure: 40 - 90 mmHg 7. Participants assigned female at birth and of child-bearing potential must have a negative serum pregnancy test within 72 hours prior to the first dose of HMB-002. 8. Women of childbearing potential (CBP) must agree to use two medically acceptable methods of contraception throughout the study. Men with sexual partners of CBP must agree to use a condom please one additional method of contraception (used by their female partner) throughout the study. 9. Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening: 1. Renal: Estimated glomerular filtration rate (eGFR) of ≥45 ml/min/1.73m\^2. 2. Hepatic: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and total bilirubin ≤1.5 upper limit of normal (ULN) range at Screening. For participants with a history of Gilbert's Syndrome, total bilirubin ≤2 × ULN. 3. Hematology (Hgb): Hemoglobin \>85 g/L and platelet count \>120 x 10\^9/L. 10. PART B ONLY- Participants must be symptomatic (typically reporting bleeding events every month) with a minimum of 3 treated bleeding events reported in either the observational study HMB-002-101\_SCR or in the participant's medical record. 11. Part B only: Participants may be enrolled if they have completed Part A follow-up. Exclusion Criteria: 1. History of clinically significant hypersensitivity associated with monoclonal antibody therapies. 2. Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis. 3. High risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/Prothrombin gene mutation, Antithrombin \<50%. Congenital Protein C and Protein S deficiency with levels \<50%. 4. Requires ongoing hemostatic treatment to prevent bleeding, except prior to procedures/surgery. 5. Has a positive test for Hepatitis B surface antigen (HbsAg), Hepatitis C antibody (HCV Ab), or human immunodeficiency virus antibody (HIV Ab) at Screening with RNA level above the lower limit of detection. 6. Has received any live vaccine within 28 days prior to signing of informed consent and/or is planning to have a live vaccine during the study period. 7. Planned major surgery during the course of the study. 8. Body mass index (BMI) \>35 kg/m\^2 (obese, adjusted for ethnicity). 9. Other conditions that substantially increase risk of thrombosis either individually or in combination by the discretion of the Investigator. 10. Participants who are pregnant or breastfeeding. 11. Clinically significant cardiovascular disease. 12. Participants who are currently smoking and unable to refrain from cigarette/cigar/tobacco/vape smoking throughout the study duration. 13. Other conditions that substantially increase the risk of cardiovascular events by the discretion of the Investigator. 14. Congenital or acquired bleeding disorders other than Type 1, Type 1C, or Type 2A VWD. 15. Concurrent disease, treatment, medication (including but not limited to drugs that would affect hemostasis), or abnormality in clinical laboratory tests may pose additional risk in the opinion of the investigator. 16. Hypersensitivity to study drug or any of the excipients. 17. Received investigational medication in another clinical study within 5 half-lives before administration of HMB-002. 18. Requires the use of drugs that would affect hemostasis (including, but not limited to anticoagulation, antiplatelet agents, certain non-steroidal anti-inflammatory drugs) and cannot refrain from use for 14 days prior to the first dose of study drug and throughout the study.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06754852 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 64 Years, studying Von Willebrand Disease (VWD). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06754852 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06754852 currently recruiting?
Yes, NCT06754852 is actively recruiting participants. Contact the research team at clinicaltrials@hemab.com for enrollment information.
Where is the NCT06754852 trial being conducted?
This trial is being conducted at Murdoch, Australia, Camperdown, Australia, Melbourne, Australia, London, United Kingdom.
Who is sponsoring the NCT06754852 clinical trial?
NCT06754852 is sponsored by Hemab ApS. The trial plans to enroll 108 participants.