RecruitingNot Applicable

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

United States200 participantsStarted 2024-08-30

Plain-language 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)

Who can participate

Age range16 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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%.

What they're measuring

Annualized bleeding event rate

Timeframe: 4.5 to 12.5 months

Annualized treated bleeding rate

Timeframe: 4.5 to 12.5 months

Annualized heavy menstrual bleed rate

Timeframe: 4.5 to 12.5 months

Number of overnight admissions

Timeframe: 4.5 to 12.5 months

Trial details

NCT IDNCT06610201

SponsorHemab ApS

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2026-12

Contact for this trial

Clinical Trials (USA; UK)

+1 888 493 8148; 080 8304 6409 Clinicaltrials@hemab.com

View on ClinicalTrials.gov More Von Willebrand Disease (VWD) trials

Plain-language summary

Who can participate

Age range16 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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%.