A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-002 in Participants With Von Willebrand Disease (Velora Pioneer)

Hemab ApS4 sites in 2 countries108 target enrollmentFebruary 6, 2025

InterventionsHMB-002 (Part A)HMB-002 (Part B)

DrugsHMB-002 (Part A)HMB-002 (Part B)

Overview

Phase: Phase 1
Intervention: HMB-002 (Part A)
Conditions: Not specified
Sponsor: Hemab ApS
Enrollment: 108
Locations: 4
Primary Endpoint: Incidence of Treatment emergent adverse events (TEAE)
Status: Recruiting
Last Updated: 4 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

February 6, 2025

End Date

July 1, 2027

Last Updated

4 months ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Hemab ApS

Responsible Party

Sponsor

Eligibility Criteria

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

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.

Arms & Interventions

Part A Single Ascending Dose Design

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

Intervention: HMB-002 (Part A)

Part B Multiple Dose Assessment

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.

Intervention: HMB-002 (Part B)

Outcomes

Primary Outcomes

Incidence of Treatment emergent adverse events (TEAE)

Time Frame: up to Day 113

Secondary Outcomes

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)
Pharmacodynamics Parameters: Assessment of VWF activity(Day 1 to Day 113)
Pharmacodynamics Parameters: Assessment of FVIII activity(Day 1 to Day 113)
Annualized Bleeding Rate Assessments(Day 1 to Day 113)