A Study Assessing HMB-002 in Participants With Von Willebrand Disease

NCT06754852 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: HMB-002-102
• Study Type: interventional
• Target: 108
• Locations: 2 countries
• Sites: 4

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.

Conditions

Von Willebrand Disease (VWD); Von Willebrand Disease (VWD), Type 1; Von Willebrand Disease (VWD), Type 2

Keywords

Von Willebrand Disease (VMD); Type 1 VWD; Type 2 VWD; Von Willebrand Factor (VWF)

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment emergent adverse events (TEAE)

  up to Day 113

Secondary Outcomes (8)

• Pharmacokinetic Parameter: Maximum observed plasma concentration (Cmax)

  Day 1 to Day 113

• Pharmacokinetic Parameter: Area under the curve from time zero to last quantifiable concentration (AUClast)

  Day 1 to Day 113

• Pharmacokinetic Parameter: Area under the curve from time zero to extrapolated infinite time (AUCinf)

  Day 1 to Day 113

• Pharmacokinetic Parameter: Time to reach maximum observed plasma concentration (Tmax)

  Day 1 to Day 113

• Pharmacodynamics Parameters: Assessment of VWF antigen (VWF:Ag)

  Day 1 to Day 113

Study Arms (2)

Part A Single Ascending Dose Design

EXPERIMENTAL

A multicenter study to evaluate the safety, tolerability, PK, and PD effect of single dose HMB-002 in participants with Type 1 VWD.

Drug: HMB-002 (Part A)

Part B Multiple Dose Assessment

EXPERIMENTAL

A multicenter study to evaluate the safety, tolerability, PK, and PD effect of 3 repeat doses of HMB-002, as well as the preliminary prophylactic effects on bleeding events.

Drug: HMB-002 (Part B)

Interventions

HMB-002 (Part A) DRUG

HMB-002 will be administered subcutaneously. Part A will utilize sentinel dosing. The planned duration of study participants in Part A is approximately 12 weeks.

Part A Single Ascending Dose Design

HMB-002 (Part B) DRUG

HMB-002 will be administered subcutaneously. Part B dosing intervals will be determined following evaluation of Part A results. The planned duration of study participants in Part B will be approximately 21 weeks.

Part B Multiple Dose Assessment

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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):
• Systolic blood pressure: 90 - 140 mmHg
• Diastolic blood pressure: 40 - 90 mmHg
• 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.
• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Hemab ApS: lead

Study Sites (4)

Fiona Stanley Hospital

Murdoch, Perth, WA 6150, Australia

NOT YET RECRUITING

Royal Prince Alfred Hospital

Camperdown, Sydney, NSW 2050, Australia

RECRUITING

The Alfred Hospital

Melbourne, Victoria, VIC 3004, Australia

RECRUITING

Richmond Pharmacology

London, SE1 1YR, United Kingdom

RECRUITING

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Clinical Trials

CONTACT

080 8304 6409; clinicaltrials@hemab.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 19, 2024
• First Posted: January 1, 2025
• Study Start: February 6, 2025
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: December 9, 2025

Record last verified: 2025-12

Locations

AU (3); GB (1)