At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- HMB-002 (Part A) (drug), HMB-002 (Part B) (drug), HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US) (drug)
- How long the study runs
- Study runs about 29 months (dates as stated)
- About the drug or intervention
- HMB-002 (Part A) — drug: HMB-002 will be administered subcutaneously. · HMB-002 (Part B) — drug: HMB-002 will be administered subcutaneously. · HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US) — drug: HMB-002 will be administered as a single dose with a concomitant single dose of factor concentrate.
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a medicine called HMB-002 in people with von Willebrand disease (VWD), a condition that affects blood clotting. It looks at different types of VWD, including Type 1, Type 2A and Type 3, across different parts of the study (Parts A, B and C). The sponsor is Hemab ApS.
Who can take part
- Weight between 50 and 120 kg
- A confirmed diagnosis of von Willebrand disease (VWD) from birth, backed by laboratory tests following standard guidelines
- Normal vital signs (like heart rate and blood pressure) at screening
- Healthy enough organs, shown by blood tests at screening (kidneys, liver, blood counts)
- Age 18 to under 70 (Part A and Part C) or 16 to under 70 (Part B)
- Part A: Type 1 VWD (some groups) or Type 1 and Type 2A VWD (other groups), with low von Willebrand factor (VWF, a clotting protein) and factor VIII (FVIII, another clotting protein) levels
- Part B: Type 1 or Type 2A VWD, with symptoms such as monthly bleeding, plus a record of at least 3 treated bleeds in the past 12 months or having taken part in the observational study VELORA Discover
- Part C: Type 3 VWD, or Type 1 VWD with very low VWF and FVIII levels, and receiving regular VWF treatment at least once a week
Who may not be able to
- A history of blood clots in veins or arteries (with one exception: catheter-related clots near the surface of the skin)
- Certain inherited conditions that greatly raise the risk of blood clots
- Body mass index (BMI, a measure of body size) over 35
- Other conditions that raise the risk of blood clots, as judged by the study doctor or medical monitor
- Serious heart disease
- Other known severe bleeding disorders besides VWD
- Needing medicines that affect blood clotting (such as blood thinners, antiplatelet medicines, or some anti-inflammatory painkillers) that cannot be stopped 14 days before the first dose and for the rest of the study
- Parts A and B only: needing ongoing treatment to prevent bleeds (prevention given just for surgery or procedures is allowed)
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 16 Years to 69 Years
- Who
- All
- Number of participants
- 108
- Started
- 2025-02-06
- Last checked
- 2026-06
Plain English Summary
What is this study?
- • Testing a new treatment for von willebrand disease (vwd)
- • Phase1/Phase2 - 108 participants
- • This is a first-in-human (FIH), Phase 1/2, 3-part open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study evaluating HMB-002 in participants with VWD
Who can take part?
- • Ages 16 Years to 69 Years
- • Diagnosed with von willebrand disease (vwd)
Where?
- • Basingstoke - Basingstoke and North Hampshire Hospital
- • Tooting - St George's Hospital
- • Whitechapel - Royal London Hospital
- • Birmingham - University Hospitals Birmingham NHS Foundation Trust
- • +5 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a first-in-human (FIH), Phase 1/2, 3-part open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study evaluating HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) regimen 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. Part C will evaluate the safety, PK, and PD of a single concomitant dose of HMB-002 and factor concentrate with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII who use factor concentrate as prophylaxis.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 16 Years - 69 Years
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Weight 50 to 120 kg, inclusive.
- ✓Documented diagnosis of Congenital VWD, confirmed by laboratory testing consistent with ISTH/ASH) diagnostic...
- ✓Vital signs are within normal ranges at Screening.
- ✓Participants must meet the following baseline organ health, indicated by laboratory criteria as Screening:
- ✓kidney: Estimated glomerular filtration rate (eGFR) of ≥45 mL/min/1.73m\^2.
Who cannot take part
- ✗Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated,...
- ✗Body mass index (BMI) \>35 kg/m\^2 (obese, adjusted for ethnicity).
- ✗Clinically significant cardiovascular disease.
- ✗Other known severe bleeding disorder(s) other than VWD.
- ✗Exclusion Criteria for Part A and Part B Only
See the full criteria
Where Is This Study? (9 UK sites)
Basingstoke and North Hampshire Hospital
Basingstoke RG24 9NA, United Kingdom
St George's Hospital
Tooting SW17 0QT, United Kingdom
Royal London Hospital
Whitechapel E1 1FR, United Kingdom
University Hospitals Birmingham NHS Foundation Trust
Birmingham B15 2TH, United Kingdom
University Hospital of Wales
Cardiff CF14 4XW, United Kingdom
St James's University Hospital, Leeds Haemophilia Centre
Leeds LS9 7TF, United Kingdom
Royal Liverpool and Broadgreen University Hospitals NHS TRUST, The Roald Dahl Haemostasis and Thrombosis Centre
Liverpool L7 8XP, United Kingdom
Richmond Pharmacology
London SE1 1YR, United Kingdom
St Thomas' Hospital
London SE1 7EH, United Kingdom
How to Get in Touch
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