Atlanta, GANCT06754852Now EnrollingIRB Ready

Von Willebrand Disease (VWD) Clinical Trial in Atlanta, GA

Access cutting-edge von willebrand disease (vwd) treatment through this clinical trial at a research site in Atlanta. Study-provided care at no cost to qualified participants.

Sponsored by Hemab ApS

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Expert Care in Atlanta

Access von willebrand disease (vwd) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related von willebrand disease (vwd) treatment provided free

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Why Participate?

  • No-Cost Study Care

  • Local to Atlanta

    Convenient for GA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Atlanta site if eligible
  4. 4Begin participation

About This Von Willebrand Disease (VWD) Study in Atlanta

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.

Sponsor: Hemab ApS

Who Can Participate

Inclusion 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 × ULN.
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

Exclusion Criteria

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 \<50%.
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 Investigator or Medical Monitor.
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 prior to the first dose of study drug and throughout the study. Exclusion Criteria for Part A and Part B Only
Requirement for ongoing hemostatic treatment to prevent bleeding (bleed prophylaxis). Prophylaxis administered intermittently for procedures or surgery to reduce bleeding risk is permitted.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Atlanta?

Yes, this clinical trial (NCT06754852) has an active research site in Atlanta, GA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Von Willebrand Disease (VWD) Treatment Options in Atlanta, GA

If you're searching for von willebrand disease (vwd) treatment options in Atlanta, GA, this clinical trial (NCT06754852) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Atlanta research site is actively enrolling participants for this clinical trial. You'll receive care from experienced von willebrand disease (vwd) specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all von willebrand disease (vwd) clinical trials near you to find additional studies recruiting in your area.

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