Endothelial Colony-Forming Cells in Patients With VWD, AVWS and Healthy Subjects (NCT07358013) | Clinical Trial Compass
RecruitingNot Applicable
Endothelial Colony-Forming Cells in Patients With VWD, AVWS and Healthy Subjects
Italy48 participantsStarted 2023-11-11
Plain-language summary
The goal of this observational study is to learn how endothelial colony-forming cells (ECFCs) behave in people with von Willebrand disease (VWD), acquired von Willebrand syndrome (AVWS), and in healthy individuals.
Who can participate
Age range
16 Years
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 for patients:
Patients with von Willebrand disease (VWD) or acquired von Willebrand syndrome (AVWS)
Age ≥ 16 years.
Previous diagnosis of von Willebrand disease or acquired von Willebrand syndrome, defined as one of the following:
Group A - Type 1 VWD:
VWF levels ≤ 30 IU/dL, regardless of bleeding history, or
VWF levels ≤ 0.50 IU/mL in the presence of abnormal bleeding.
Group B - Congenital or acquired VWD (VWD or AVWS):
Diagnosis of congenital or acquired VWD, with or without gastrointestinal bleeding.
Group C - Subgroup study (Type 2A VWD):
One patient with type 2A VWD selected for a dedicated sub-study involving allele-specific siRNA silencing of the mutant allele.
Ability and willingness to provide written informed consent.
For patients without prior molecular characterization: willingness to undergo VWF gene sequencing and to sign the related informed consent.
Inclusion criteria for healthy volunteers
* No prior diagnosis of VWD, bleeding disorders, or thrombotic disorders.
* Willingness to donate blood for study procedures.
* Ability and willingness to provide written informed consent.
* Age ≥ 18 years.
Exclusion criteria for both patients and healthy volunteers:
* Pregnancy.
* Anemia, as determined at screening or based on medical history.
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
1
ECFCs isolation and characterization
Timeframe: 42 weeks from enrollment start
Trial details
NCT IDNCT07358013
SponsorFondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico