Von Willebrand Disease, Type 2A, Von Willebrand Disease, Type 2M, Von Willebrand Disease, Type 2N, Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
Conditions
Keywords
Von Willebrand Disease (VWD), Prospective Study, Type 1 VWD, Type 2 VWD, Type 3 VWD, Prophylaxis, Von Willebrand Factor (VWF)
Brief summary
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
OTHERClinical 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
OTHERClinical 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.
Sponsors
Hemab ApS
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations. 2. Has an understanding, ability, and willingness to comply with Study procedures and restrictions. 3. Is 16 years and \< 70 years at the time of screening. 4. Weight 50 to 120 kg (±10%) at Screening and body mass index (BMI) \<38.5 kg/m\*2. 5. 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. 6. 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
1. Has a history of clinically significant hypersensitivity associated with monoclonal antibody therapies. 2. Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events. 3. 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%. 4. Requires ongoing hemostatic (bleed-prophylaxis) treatment to prevent bleeding 5. Has other known severe bleeding disorder(s) other than VWD. 6. Planned major surgery during the study period. 7. 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. 8. Is pregnant or plans to become pregnant within the next 6 months following informed consent sign off. 9. 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. 10. 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. 11. 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. 12. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized bleeding event rate | 4.5 to 12.5 months | — |
| Annualized treated bleeding rate | 4.5 to 12.5 months | — |
| Annualized heavy menstrual bleed rate | 4.5 to 12.5 months | — |
| Number of overnight admissions | 4.5 to 12.5 months | Hospitalization monitoring of bleeding events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Prophylactic and on demand treatment | 4.5 to 12.5 months | Details of treatment used for bleeding events |
| Iron status | 4.5 to 12.5 months | Blood samples will be collected for the determination of ferritin and hemoglobin levels. |
| Patient-Reported Outcomes Measurement Information System (PROMIS)-29 | 4.5 to 12.5 months | An assessment which evaluates four items from each of the seven PROMIS domains. Each question is rated on a scale of 1 to 5. In addition, the PROMIS-29 includes one pain intensity question which is rated on a scale of 1 to 10. Higher scores on the PROMIS-29 indicate worse symptoms. |
| Menstrual Bleeding Questionnaire (MBQ) | 4.5 to 12.5 months | Measure the effect of heavy menstrual bleeding on a self-assessment of menstrual blood loss, limitations in social and leisure activities, physical activities, and work activities. |
| Epistaxis Severity Score (ESS) | 4.5 to 12.5 months | Assessment of nose bleed severity |
| Oral Bleeding Experience | 4.5 to 12.5 months | Questionnaire to evaluate bleeding symptoms |
Countries
Australia, United Kingdom, United States
Contacts
CONTACTClinical Trials (USA; UK)
CONTACTClinical Trials (Australia)
STUDY_DIRECTORVP of Clinical Research
Hemab ApS
Outcome results
None listed