A Study of Bleeding and Treatment in Participants With Von Willebrand Disease

Official title

Velora Discover: A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease

Overview

The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.(e.g. Velora Pioneer)

Detailed description

This is a prospective, screening study in participants with confirmed Type 1 VWD according to diagnostic guidelines. Participants with confirmed Type 1 VWD and associated bleeding symptoms will be enrolled. The study may also be opened to participants with Type 2 and Type 3 VWD with Sponsor approval. Up to a total of 200 participants may be enrolled in the study.

The study includes screening, a baseline evaluation, and an approximately 4 month observation period which will include every other week telemedicine check-ins (to monitor bleed diary entries and bleeding event treatments. There will be an optional extension to the observation period of up to a total of 12 months for participants wishing to continue.

Interventions

• Other Clinical outcomes of patients with VWD, Type 1
  Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1
• Other Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
  Accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 2A, Type 2M, Type 2N and Type 3.

Primary outcome measures

• Annualized bleeding event rate [Time frame: 4.5 to 12.5 months]
• Annualized treated bleeding rate [Time frame: 4.5 to 12.5 months]
• Annualized heavy menstrual bleed rate [Time frame: 4.5 to 12.5 months]
• Number of overnight admissions [Time frame: 4.5 to 12.5 months]

Secondary outcome measures (6)

• Prophylactic and on demand treatment [Time frame: 4.5 to 12.5 months]
• Iron status [Time frame: 4.5 to 12.5 months]
• Patient-Reported Outcomes Measurement Information System (PROMIS)-29 [Time frame: 4.5 to 12.5 months]
• Menstrual Bleeding Questionnaire (MBQ) [Time frame: 4.5 to 12.5 months]
• Epistaxis Severity Score (ESS) [Time frame: 4.5 to 12.5 months]
• Oral Bleeding Experience [Time frame: 4.5 to 12.5 months]

Eligibility criteria

Inclusion criteria

• Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.
• Has an understanding, ability, and willingness to comply with Study procedures and restrictions.
• Is 16 years and < 70 years at the time of screening.
• Weight 50 to 120 kg (±10%) at Screening and body mass index (BMI) <38.5 kg/m\*2.
• Has Von Willebrand Disease: Type 1 VWD (including Type 1C VWD) or Type 2A VWD. All participants must have: Documented lab results confirming their diagnosis consistent with ISTH/ASH diagnostic guidelines; VWF Activity ≤30 IU/dL and FVIII activity ≤70 IU/dL during Screening.
• Has symptomatic disease as defined by a history of bruising or bleeding events, with an expected minimum of 3 bleeding episodes (including heavy menstrual bleeding) per year that require treatment to control bleeding symptoms, and/or has recurrent and ongoing episodes of heavy menstrual bleeding at the time of enrollment.

Exclusion criteria

• Has a history of clinically significant hypersensitivity associated with monoclonal antibody therapies.
• Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.
• Has a high-risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/prothrombin gene mutation, antithrombin <50%, congenital protein C and protein S deficiency with levels <50%.
• Requires ongoing hemostatic (bleed-prophylaxis) treatment to prevent bleeding
• Has other known severe bleeding disorder(s) other than VWD.
• Planned major surgery during the study period.
• Has other conditions that substantially increase the risk of thrombosis either individually or in combination, at the discretion of the Investigator, including but not limited to: significant family history; BMI >30 and ≤38.5 kg/m² (moderately obese, adjusted for ethnicity and increased central adiposity); reduced mobility; active malignancy; major surgery within 6 weeks preceding Screening; or postpartum within 12 weeks preceding Screening.
• Is pregnant or plans to become pregnant within the next 6 months following informed consent sign off.
• Has clinically significant cardiovascular disease including, but not limited to: NYHA Class III or IV heart failure, coronary artery disease, uncontrolled arrythmia, moderate to severe valvular heart disease, peripheral vascular disease, and ischemic stroke.
• Has other combinations of conditions that substantially increase the risk of cardiovascular events at the discretion of the Investigator including, but not limited to, smoking, uncontrolled hyperlipidemia, and uncontrolled hypertension.
• Has any concurrent disease, treatment, medication (including but not limited to ongoing anticoagulation, antiplatelet therapy, or non-steroidal anti-inflammatory drugs or other drugs that affect hemostasis), condition, medication, or abnormality in clinical laboratory tests which may impact on the participant's bleeding symptoms or affect their ability to complete the study, in the Investigator's opinion.
• Has received any investigational product within 30 days prior to Screening. If the participant was enrolled and dosed in Velora Pioneer (study HMB-002-102; NCT06754852), they must have completed their End of Study Visit.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

United States · 13 centers

• Phoenix Children's Hospital — Phoenix
• Arkansas Children's Hospital — Little Rock
• Children's Hospital of Los Angeles — Los Angeles
• University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center — Miami
• Emory Children's Center — Atlanta
• Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center — Indianapolis
• Tulane University School of Medicine — New Orleans
• University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders — Ann Arbor
• … and 5 more centers

Australia · 3 centers

• Fiona Stanley Hospital — Murdoch
• Royal Prince Alfred Hospital — Camperdown
• The Alfred Hospital — Melbourne

United Kingdom · 1 center

• Richmond Pharmacology — London

Identifiers

NCT: NCT06610201 · HMB-002-101_SCR

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗