Recruiting
Phase 1
Phase 2

HMB-002

Sponsor:

Hemab ApS

Code:

NCT06754852

Conditions

Von Willebrand Disease (VWD)

Von Willebrand Disease (VWD), Type 1

Von Willebrand Disease (VWD), Type 2

Von Willebrand Disease (VWD), Type 3

Eligibility Criteria

Sex: All

Age: 16 - 69

Healthy Volunteers: Not accepted

Interventions

HMB-002 (Part A)

HMB-002 (Part B)

HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)

Study Details

Brief summary:

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.

Conditions

Von Willebrand Disease (VWD)

Von Willebrand Disease (VWD), Type 1

Von Willebrand Disease (VWD), Type 2

Von Willebrand Disease (VWD), Type 3

Study ID

NCT06754852

Start date

Feb 6, 2025

Status verified date

Jun, 2026

Completion date

Jul, 2027

Anticipated

Primary completion date

Jul, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16 - 69

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

108 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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 repeat doses of HMB-002, as well as the preliminary prophylactic effects on bleeding events.

experimental: Part C HMB-002 with Concomitant Factor Concentrate

A multicenter study to evaluate the safety and tolerability of a single dose of HMB-002, administered to patients concurrently receiving regular factor concentrate as standard of care.

Interventions

HMB-002 (Part A)

HMB-002 will be administered subcutaneously. Part A will utilize sentinel dosing. The planned duration of study participants in Part A is approximately 12 weeks.

HMB-002 (Part B)

HMB-002 will be administered subcutaneously. Part B dosing intervals will be determined following evaluation of Part A results. The planned duration of study participants in Part B will be approximately 21 weeks.

HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)

HMB-002 will be administered as a single dose with a concomitant single dose of factor concentrate. The planned duration of study participants in Part C will be approximately 17 weeks.

Primary outcome measure

  • Incidence of Treatment emergent adverse events (TEAE) [ Time Frame: up to Day 113 ]

Central Contacts and Locations

Central contacts

Locations

Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center

Recruiting

Indianapolis, Indiana, United States, 46260

More Information

Sponsor

Hemab ApS

Last update posted

Jun 30, 2026

Last verified

Jun, 2026

Keywords

  • Von Willebrand Disease (VMD)
  • Type 1 VWD
  • Type 1 with low residual VWF and FVIII who use factor concentrate as prophylaxis
  • Type 2 VWD
  • Type 3 VWD
  • Von Willebrand Factor (VWF)

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Hemab ApS on 2026-06-30.