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)

Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 64
Healthy Volunteers: f
View:

• 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.

‣ Hematology (Hgb): Hemoglobin \>85 g/L and platelet count \>120 x 10\^9/L.

⁃ 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.

⁃ Part B only: Participants may be enrolled if they have completed Part A follow-up.

Locations
Other Locations
Australia
Royal Prince Alfred Hospital
RECRUITING
Camperdown
The Alfred Hospital
RECRUITING
Melbourne
Fiona Stanley Hospital
NOT_YET_RECRUITING
Murdoch
United Kingdom
Richmond Pharmacology
RECRUITING
London
Contact Information
Primary
Clinical Trials
clinicaltrials@hemab.com
080 8304 6409
Time Frame
Start Date: 2025-02-06
Estimated Completion Date: 2027-07
Participants
Target number of participants: 108
Treatments
Experimental: 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.
Experimental: 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.
Sponsors
Leads: Hemab ApS

This content was sourced from clinicaltrials.gov

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