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New treatment option for Von Willebrand Disease (VWD)
Official title A Study Assessing HMB-002 in Participants With Von Willebrand Disease
ClinicalTrials.gov ID: NCT06754852
What this study is testing
What is HMB-002 (Part A)?
HMB-002 (Part A) is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for von willebrand disease (vwd).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 to 69
You may be able to join if
- Weight 50 to 120 kg, inclusive.
- Documented diagnosis of Congenital VWD, confirmed by laboratory testing consistent with ISTH/ASH) diagnostic guidelines).
- Vital signs are within normal ranges at Screening.
- Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening:
- Renal: Estimated glomerular filtration rate (eGFR) of ≥45 mL/min/1.73m\^2.
You likely can't join if
- Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis.
- High risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/Prothrombin gene mutation, Antithrombin deficiency with activity...
- Body mass index (BMI) \>35 kg/m\^2 (obese, adjusted for ethnicity).
- Presence of other conditions that substantially increase risk of thrombosis either individually (for participants \>65 years of age) or in...
- Clinically significant cardiovascular disease.
- Other known severe bleeding disorder(s) other than VWD.
See the full eligibility criteria
- Weight 50 to 120 kg, inclusive.
- Documented diagnosis of Congenital VWD, confirmed by laboratory testing consistent with ISTH/ASH) diagnostic guidelines).
- Vital signs are within normal ranges at Screening.
- Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening:
- Renal: Estimated glomerular filtration rate (eGFR) of ≥45 mL/min/1.73m\^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 ×...
- Hematology \>85 g/L and platelet count \>120 x 10\^9/L. Part A Only:
- Age: ≥18 and \<70 years of age at the time of informed consent.
- 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
- Residual VWF activity of ≤ 50 IU/dL and FVIII activity ≤ 70 IU/dL during screening. Part B Only:
- Age: ≥16 and \<70 years of age at the time of informed consent.
- VWD Subtype Eligibility: Participants with Type 1 VWD (including Type 1C) and Type 2A.
- Residual VWF activity of ≤50 IU/dL and FVIII activity ≤70 IU/dL during screening.
- Symptomatic Disease: Participants must be symptomatic, typically reporting bleeding events on a monthly basis.
- Bleeding History (must meet one of the following):
- 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
- 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:
- Age: ≥18 and \<70 years of age at the time of informed consent.
- 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).
- Receives regular VWF concentrate (at least 1/week) as part of their routine care (usual dose ≤50 IU/kg). Key
- Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis.
- 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...
- Body mass index (BMI) \>35 kg/m\^2 (obese, adjusted for ethnicity).
- 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...
- Clinically significant cardiovascular disease.
- Other known severe bleeding disorder(s) other than VWD.
- 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...
- Requirement for ongoing hemostatic treatment to prevent bleeding (bleed prophylaxis). Prophylaxis administered intermittently for procedures or surgery to reduce bleeding risk is permitted.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Little Rock, Arkansas, United States
- Los Angeles, California, United States
- Miami, Florida, United States
- Atlanta, Georgia, United States
- Indianapolis, Indiana, United States
- New Orleans, Louisiana, United States
- Ann Arbor, Michigan, United States
- Rochester, Minnesota, United States
- Portland, Oregon, United States
- Pittsburgh, Pennsylvania, United States
- Dallas, Texas, United States
- Seattle, Washington, United States
- Murdoch, Perth, Australia
- Camperdown, Sydney, Australia
- Melbourne, Victoria, Australia
- Basingstoke, Hampshire, United Kingdom
- Tooting, London, United Kingdom
- Whitechapel, London, United Kingdom
- Birmingham, United Kingdom
+ 4 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years to 69 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Little Rock, Arkansas, United States; Los Angeles, California, United States; Miami, Florida, United States; Atlanta, Georgia, United States; Indianapolis, Indiana, United States and 18 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.