Patient case (clinical description): A 23-year-old female has prolonged oral bleeding immediately after a tooth extraction.  Despite several interventions, the bleeding persists for hours and stops only after desmopressin (DDAVP) administration.  The patient has heavy menstrual cycles each month.  She has no other medical problems and takes no medications.  Her mother and grandmother have also had excessive bleeding during menstrual period.  Review of systems is positive for mild bruising on his legs. Laboratory findings reveal a normal platelet count and an abnormal ristocetin cofactor assay, as well as CB <= 0.30 IU/mL and FVIII:C <= 0.40 IU/mL.

Patient's own account: I'm a 23 woman and I went to the ER because of non-stop bleeding after my tooth extraction. I tried to make the bleeding stop but it just kept going for hours and it finally stopped when I took some medication called DDAVP. I usually have heavy periods, just like my mom and grandma had. I don't have any other medical condition and I don't take any other medication. The doctor noticed the bruises on my legs. I had to do some lab tests. I had a normal platelet count but an abnormal ristocetin cofactor assay. I had low CB and FVIII:C levels.

Clinical trial: ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders

Eligibility criteria:
Inclusion Criteria:

          -  Male or female;

          -  Age 18-75 years;

          -  vWD-2b - confirmed diagnosis, or;

          -  TTP Remission - prior episode(s) of primary acute TTP, or;

          -  Acute TTP - any episode, first or relapse, with presence of all of the following:

               1. Microangiopathic hemolytic anemia (schistocytosis present, Coombs test negative);

               2. Severe thrombocytopenia;

               3. Clinical diagnosis of either a primary or secondary form of TTP:(1) Primary TTP:
                  e.g., familial TTP (Upshaw-Schulman syndrome), or acquired idiopathic TTP, or
                  "atypical HUS"; (2) Secondary TTP: e.g., TTP occurring post-bone marrow
                  transplant, drug-induced TTP, lupus-related TTP, etc.;

          -  Negative qualitative urine drug test at screening, and no history of alcohol or drug
             abuse;

          -  Not considering or scheduled to undergo any surgical procedure during the duration of
             the study;

          -  Has not donated or lost more than a unit of blood within 30 days prior to screening
             visit;

          -  Has not received an experimental drug within 30 days prior to screening;

          -  Female patients must be non-pregnant [for TTP Remission and vWD-2b Cohorts, a serum
             pregnancy test at screening and a urine pregnancy test at Day 1 pre-dose must be
             negative; for the Acute TTP Cohort, a serum pregnancy test at Day 1 pre-dose must be
             negative], and willing to use effective, redundant methods of contraception (i.e., for
             both self and male partner) throughout the study and for at least 30 days after
             participation. If possible, the treatment will be initiated within 5 days of the
             cessation of the preceding menstrual period;

          -  Male patients must agree to use a medically acceptable contraceptive (abstinence or
             use of a condom with spermicide) throughout the study and for at least 30 days after
             participation;

          -  Patients must be capable of understanding and complying with the protocol and must
             have signed the informed consent document prior to performance of any study-related
             procedures.

        Exclusion Criteria:

          -  History of recent surgery or trauma;

          -  Any major, active health problem, e.g., cancer or heart disease, which could render
             the patient medically unstable during the period of participation in the study.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.
Subject must be at most 75 Years

Eligibility (NLI label): Entailment