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Looking for participantsPhase1/Phase2

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

Sponsor: Hemab ApS

NCT ID: NCT06754852

View on ClinicalTrials.gov ↗

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.

Von Willebrand Disease (VWD)Von Willebrand Disease (VWD), Type 1Von Willebrand Disease (VWD), Type 2Von Willebrand Disease (VWD), Type 3

How this trial compares with your answers

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What we know so far

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Still need:

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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

eGFR

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
Key Inclusion Criteria: 1. Weight 50 to 120 kg, inclusive. 2. Documented diagnosis of Congenital VWD, confirmed by laboratory testing consistent with ISTH/ASH) diagnostic guidelines). 3. Vital signs are within normal ranges at Screening. 4. Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening: 1. Renal: Estimated glomerular filtration rate (eGFR) of ≥45 mL/min/1.73m\^2. 2. Hepatic: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and total bilirubin ≤1.5 upper limit of normal (ULN) at Screening. For participants with a history of Gilbert's Syndrome, total bilirubin ≤2 × ULN. 3. Hematology \>85 g/L and platelet count \>120 x 10\^9/L. Part A Only: 5. Age: ≥18 and \<70 years of age at the time of informed consent. 6. VWD Subtype Eligibility: * Cohorts A1 and A2: Participants with Type 1 VWD, only. * Cohorts A3 and A4: Participants with Type 1 VWD (including Type 1C) and Type 2A VWD 7. Residual VWF activity of ≤ 50 IU/dL and FVIII activity ≤ 70 IU/dL during screening. Part B Only: 8. Age: ≥16 and \<70 years of age at the time of informed consent. 9. VWD Subtype Eligibility: Participants with Type 1 VWD (including Type 1C) and Type 2A. 10. Residual VWF activity of ≤50 IU/dL and FVIII activity ≤70 IU/dL during screening. 11. Symptomatic Disease: Participants must be symptomatic, typically reporting bleeding events on a monthly basis. 12. Bleeding History (must meet one of the following): 1. Prior Observational Study Participation: The participant must have participated in the observational study HMB-002-101\_SCR (VELORA Discover), have a minimum annualized treated bleeding event (ATBR) of 3; OR 2. Medical Record-Documented Bleeding History: The Investigator confirms that ≥3 treated bleeding events have been documented in the participant's medical record within the preceding 12 months. Part C Only: 13. Age: ≥18 and \<70 years of age at the time of informed consent. 14. Participants with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII activity levels (VWF activity \<5 IU/dL and FVIII activity \<10 IU/dL). 15. Receives regular VWF concentrate (at least 1/week) as part of their routine care (usual dose ≤50 IU/kg). Key Exclusion Criteria: 1. Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis. 2. High risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/Prothrombin gene mutation, Antithrombin deficiency with activity \<50%. Congenital Protein C and Protein S deficiency with levels \<50%. 3. Body mass index (BMI) \>35 kg/m\^2 (obese, adjusted for ethnicity). 4. Presence of other conditions that substantially increase risk of thrombosis either individually (for participants \>65 years of age) or in combination (for participants ≤65 years of age), at the discretion of the Investigator or Medical Monitor. 5. Clinically significant cardiovascular disease. 6. Other known severe bleeding disorder(s) other than VWD. 7. Requirement for concomitant medications that affect hemostasis (including, but not limited to anticoagulation, antiplatelet agents, certain non-steroidal anti-inflammatory drugs) and cannot refrain from use for 14 days prior to the first dose of study drug and throughout the study. Exclusion Criteria for Part A and Part B Only 8. Requirement for ongoing hemostatic treatment to prevent bleeding (bleed prophylaxis). Prophylaxis administered intermittently for procedures or surgery to reduce bleeding risk is permitted.

Where Is This Study? (9 UK sites)

Basingstoke and North Hampshire Hospital

Basingstoke RG24 9NA, United Kingdom

Recruiting
Hospital R&D contact (matched)

Research Manager

research.team@hhft.nhs.uk01256 473202 Ext 4557

St George's Hospital

Tooting SW17 0QT, United Kingdom

NOT_YET_RECRUITING
Hospital R&D contact (matched)

Mr Subhir Bedi

researchgovernance@sgul.ac.uk020 8725 4986

Royal London Hospital

Whitechapel E1 1FR, United Kingdom

NOT_YET_RECRUITING
Hospital R&D contact (matched)

Natasha Ajraam

rf-tr.randd@nhs.net020 375 82150

University Hospitals Birmingham NHS Foundation Trust

Birmingham B15 2TH, United Kingdom

NOT_YET_RECRUITING
Hospital R&D contact (matched)

Sarah Pountain Head of Research Governance

R&D@uhb.nhs.uk0121 371 4185

University Hospital of Wales

Cardiff CF14 4XW, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elen de Lacy

PHW.Research@wales.nhs.uk02920 104468

St James's University Hospital, Leeds Haemophilia Centre

Leeds LS9 7TF, United Kingdom

NOT_YET_RECRUITING
Hospital R&D contact (matched)

R&I Team

leedsth-tr.researchfacilitation@nhs.net0113 2060469

Royal Liverpool and Broadgreen University Hospitals NHS TRUST, The Roald Dahl Haemostasis and Thrombosis Centre

Liverpool L7 8XP, United Kingdom

NOT_YET_RECRUITING

Richmond Pharmacology

London SE1 1YR, United Kingdom

Recruiting

St Thomas' Hospital

London SE1 7EH, United Kingdom

NOT_YET_RECRUITING
Hospital R&D contact (matched)

Main Email: gstt.research.rbhh@nhs.net

gstt.research.rbhh@nhs.netn/a

How to Get in Touch

Clinical Trials

Sponsor contact

CONTACT

080 8304 6409 clinicaltrials@hemab.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-06