CompletedNot Applicable

Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment

United States, Belarus, Bulgaria56 participantsStarted 2019-06-25

Plain-language summary

The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months. The data obtained will be used as a basis for historical comparisons with the bleeding and treatment pattern obtained from a clinical study on the efficacy of prophylactic treatment with a VWF/FVIII concentrate.

Who can participate

Age range

66 Months

Sex

ALL

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: Patients who meet all of the following criteria are eligible for the study: * Male or female patients aged ≥5.5 years at the time of enrolment * VWD type 1 (baseline von Willebrand factor activity \[VWF:RCo\], \<30 IU/dL), 2A, 2B, 2M, or 3 according to medical history requiring substitution therapy with a VWF-containing product to control bleeding * Currently receiving frequent on-demand treatment with a VWF-containing product * In female patients of child-bearing potential using hormonal contraception, the medication class should remain unchanged for the duration of their study participation * Voluntarily given, fully informed written and signed consent obtained before collection of any patient data Exclusion Criteria: Patients who meet any of the following criteria are not eligible for the study: * Patients currently on prophylaxis for VWD (except for perioperative prophylaxis) as well as patients having received treatment once a month for menstrual bleeding, but not for any other bleeds * Patients whose VWD treatment is planned to be switched from on-demand to prophylactic treatment in the next 6 months * History, or current suspicion, of VWF or FVIII inhibitors * Medical history of a thromboembolic event within 6 months before enrolment * Severe liver or kidney diseases as described in the medical records * Female patients with an existing or suspected pregnancy or who are breast-feeding at the time of enrolment * Change in hormonal contraception …

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Total Annualized Bleeding Rate (TABR)

Timeframe: Screening through study completion (6 months)

Trial details

NCT IDNCT04053699

SponsorOctapharma

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2021-01-31

Results submitted2021-11-24

View on ClinicalTrials.gov More Von Willebrand Diseases trials