Status:
RECRUITING
A Study Assessing HMB-002 in Participants With Von Willebrand Disease
Lead Sponsor:
Hemab ApS
Conditions:
Von Willebrand Disease (VWD)
Von Willebrand Disease (VWD), Type 1
Eligibility:
All Genders
18-64 years
Phase:
PHASE1
PHASE2
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...
Eligibility Criteria
Inclusion Criteria:
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Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.
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Has an understanding, ability, and willingness to comply with study procedures and restrictions.
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≥18 and <65 years.
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Weight 50 to 110 kg, inclusive.
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Congenital Type 1 VWD, Type 1C and Type 2A VWD diagnosis as documented by laboratory results for VWF antigen and activity.
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Vital signs are within the following ranges at Screening:
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Resting pulse rate ≤105 bpm
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Blood pressure (BP):
- Systolic blood pressure: 90 - 140 mmHg
- Diastolic blood pressure: 40 - 90 mmHg
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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.
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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.
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Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening:
- Renal: Estimated glomerular filtration rate (eGFR) of ≥45 ml/min/1.73m^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.
- Hematology (Hgb): Hemoglobin >85 g/L and platelet count >120 x 10^9/L.
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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.
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Part B only: Participants may be enrolled if they have completed Part A follow-up.
Exclusion Criteria:
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies.
- Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis.
- 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%.
- Requires ongoing hemostatic treatment to prevent bleeding, except prior to procedures/surgery.
- 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.
- 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.
- Planned major surgery during the course of the study.
- Body mass index (BMI) >35 kg/m^2 (obese, adjusted for ethnicity).
- Other conditions that substantially increase risk of thrombosis either individually or in combination by the discretion of the Investigator.
- Participants who are pregnant or breastfeeding.
- Clinically significant cardiovascular disease.
- Participants who are currently smoking and unable to refrain from cigarette/cigar/tobacco/vape smoking throughout the study duration.
- Other conditions that substantially increase the risk of cardiovascular events by the discretion of the Investigator.
- Congenital or acquired bleeding disorders other than Type 1, Type 1C, or Type 2A VWD.
- 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.
- Hypersensitivity to study drug or any of the excipients.
- Received investigational medication in another clinical study within 5 half-lives before administration of HMB-002.
- 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.
Key Trial Info
Start Date :
February 6 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 1 2027
Estimated Enrollment :
108 Patients enrolled
Trial Details
Trial ID
NCT06754852
Start Date
February 6 2025
End Date
July 1 2027
Last Update
December 9 2025
Active Locations (4)
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1
Fiona Stanley Hospital
Murdoch, Perth, Australia, WA 6150
2
Royal Prince Alfred Hospital
Camperdown, Sydney, Australia, NSW 2050
3
The Alfred Hospital
Melbourne, Victoria, Australia, VIC 3004
4
Richmond Pharmacology
London, United Kingdom, SE1 1YR