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

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 Years - 69 Years

Healthy Volunteers

Not accepted

Interventions

• HMB-002 (Part A) — DRUG
  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) — DRUG
  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) — DRUG
  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.

Study Details

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.

Key Dates

Start date

Feb 6, 2025

Status verified

Jun 2026

Primary completion

Jul 31, 2027

Completion

Jul 31, 2027

Study Design

Enrollment

108 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

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.

Primary Outcome Measure

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

Central Contacts

• Clinical Trials
  080 8304 6409
  [email protected]

Locations (13)

Find similar trials in Phoenix, AZ

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