A Study Assessing HMB-002 in Participants With Von Willebrand Disease (NCT06754852) | Clinical Trial Compass
RecruitingPhase 1/2
A Study Assessing HMB-002 in Participants With Von Willebrand Disease
Australia, United Kingdom108 participantsStarted 2025-02-06
Plain-language 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.
Who can participate
Age range18 Years – 64 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.
✓. Has an understanding, ability, and willingness to comply with study procedures and restrictions.
✓. ≥18 and \<65 years.
✓. Weight 50 to 110 kg, inclusive.
✓. Congenital Type 1 VWD, Type 1C and Type 2A VWD diagnosis as documented by laboratory results for VWF antigen and activity.
✓. Vital signs are within the following ranges at Screening:
✓. Resting pulse rate ≤105 bpm
✓. Blood pressure (BP):
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.
What they're measuring
1
Incidence of Treatment emergent adverse events (TEAE)
. 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).