Actively Recruiting
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)
Led by Hemab ApS · Updated on 2025-12-09
108
Participants Needed
4
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in adults aged 18 to 64 with Von Willebrand Disease (VWD), including Type 1, Type 1C, and Type 2A. This first-in-human, open-label Phase 1/2 study aims to understand how this investigational drug behaves in the body and its preliminary effects on bleeding episodes in affected participants. The study has two parts: Part A involves a single ascending dose of HMB-002 given subcutaneously to assess initial safety, tolerability, and drug behavior in the body over about 12 weeks. Part B involves multiple repeat doses, with dosing intervals determined after Part A results, lasting approximately 21 weeks. This phase evaluates safety and tolerability with repeated dosing and explores the drug's potential to reduce bleeding events. Participants will undergo regular monitoring including vital signs and laboratory tests to assess blood factors related to VWD and overall health. The study will track adverse events up to Day 113, measure drug levels and activity, and record bleeding rates. Participants must meet specific health criteria and agree to contraceptive measures if applicable. The total participation time varies by study part, with follow-ups and safety checks throughout the study period.
CONDITIONS
Brief Title
A Study Assessing HMB-002 in Participants With Von Willebrand Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ability to provide informed consent
- Willingness and ability to follow study procedures
- Age 18 to less than 65 years
- Weight between 50 and 110 kg
- Diagnosed with congenital Type 1, Type 1C, or Type 2A Von Willebrand Disease confirmed by lab tests
- Resting pulse rate 105 bpm or less at screening
- Systolic blood pressure between 90 and 140 mmHg at screening
- Diastolic blood pressure between 40 and 90 mmHg at screening
- Negative pregnancy test within 72 hours before first dose for females of child-bearing potential
- Women of childbearing potential must agree to use two forms of contraception during the study; men with partners of childbearing potential must use condoms plus one additional contraception method
- Adequate organ function based on lab tests: eGFR 45 ml/min/1.73m2 or higher; liver enzymes and bilirubin within limits; hemoglobin above 85 g/L; platelet count above 120 x 10^9/L
- For Part B only: symptomatic participants with at least 3 treated bleeding events in prior observation or medical records
- For Part B only: participants may join after completing Part A follow-up
You will not qualify if you...
- History of serious allergic reactions to monoclonal antibody therapies
- Personal history of venous or arterial thrombosis, except catheter-associated superficial venous thrombosis
- High-risk thrombophilia conditions like homozygous Factor V Leiden or significant protein deficiencies
- Need for ongoing hemostatic treatment except before surgery or procedures
- Positive test for Hepatitis B surface antigen, Hepatitis C antibody, or HIV antibody with detectable RNA
- Received live vaccine within 28 days before consent or planning live vaccine during study
- Planned major surgery during the study
- Body mass index over 35 kg/m2 adjusted for ethnicity
- Other conditions increasing thrombosis or cardiovascular risks as judged by investigator
- Pregnant or breastfeeding
- Clinically significant cardiovascular disease
- Current smokers unable to stop during the study
- Other bleeding disorders beyond specified VWD types
- Concurrent diseases, treatments, or medications posing additional risk
- Allergies to study drug or its ingredients
- Received investigational drugs within 5 half-lives before study drug
- Need for drugs affecting blood clotting that cannot be stopped 14 days before and during study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 12 weeks
Participants receive a single subcutaneous dose of HMB-002 with sentinel dosing to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics.
Multiple visits during 12 weeks for assessments
Duration - Approximately 21 weeks
Participants receive multiple subcutaneous doses of HMB-002 to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary prophylactic effects on bleeding events.
Multiple visits during 21 weeks for assessments
Trial Site Locations
Total: 4 locations
1
Fiona Stanley Hospital
Murdoch, Perth, Australia, WA 6150
Not Yet Recruiting
2
Royal Prince Alfred Hospital
Camperdown, Sydney, Australia, NSW 2050
Actively Recruiting
3
The Alfred Hospital
Melbourne, Victoria, Australia, VIC 3004
Actively Recruiting
4
Richmond Pharmacology
London, United Kingdom, SE1 1YR
Actively Recruiting
Research Team
C
Clinical Trials
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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