Endothelial colony-forming cell behavior in people with von Willebrand disease and acquired von Willebrand syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

Researchers will collect blood from patients with congenital or acquired von Willebrand disease and from matched healthy volunteers at a single center in Milan to isolate ECFCs and plasma. ECFCs will be cultured and characterized ex vivo while plasma samples are used for VWF measurements and genetic testing when needed. Patient-derived cellular and molecular findings will be compared with the healthy reference population and correlated with clinical and biochemical data. The protocol also includes a dedicated sub-study for one patient with type 2A VWD involving allele-specific siRNA silencing of the mutant allele.

Why it matters

Who can join: the full rules

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.

Where it's running

• Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, A.B.Bonomi Hemophilia and Thrombosis Center
  Milan, Italy Recruiting

Study contacts

• Study coordinator: Flora Peyvandi, MD, PhD
• Email: flora.peyvandi@policlinico.mi.it
• Phone: 02-55035414

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.