Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.
Participants who meet the following inclusion criteria and none of the exclusion criteria
are eligible for enrollment in the base study:
Inclusion Criteria:
1. Any age
2. Having a congenital or acquired non-neoplastic hematologic disorder; or
3. Having a bleeding phenotype as indicated by an age adjusted abnormal ISTH Bleeding
Assessment Tool score with an unknown diagnosis; or
4. Connective tissue disorder with bleeding tendency as indicated by an age adjusted
abnormal ISTH Bleeding Assessment Tool score.
Exclusion Criteria:
1. Does not qualify for inclusion in a cohort
2. Unable to give informed consent or assent
3. Unwilling to perform study procedures
Cohort Participant Selection
Each participant is to be enrolled in the cohort for which they qualify as defined below.
Hemophilia Cohort
Inclusion Criteria:
Participants who meet any of the following inclusion criteria are eligible for enrollment
into this cohort:
1. Factor VIII or factor IX activity < 50%, without another explanation for low clotting
factor other than congenital hemophilia or being a known carrier for congenital
hemophilia; OR
2. Being a known carrier for congenital hemophilia with a factor VIII or factor IX
activity greater than or equal to 50% with or without a bleeding phenotype as
indicated by an age-adjusted abnormal ISTH Bleeding Assessment Tool score; OR
3. Known congenital hemophilia that have a factor level >50% after receiving vector; OR
4. Acquired hemophilia
Exclusion Criteria:
None
Von Willebrand Disease Cohort
Inclusion Criteria:
Participants who meet the following inclusion criteria are eligible for enrollment into
this cohort:
1. Meeting the definition of VWD or low VWF per most recent international guidelines
Exclusion Criteria:
None
Congenital Platelet DisorderS Cohort
Inclusion Criteria:
Participants who meet the following inclusion criteria are eligible for enrollment into
this cohort:
1. Abnormalities of platelet function
1. Glanzmann thrombasthenia (GPIIb or GPIIIa)
2. Bernard-Soulier syndrome (GPIbalpha, GPIbbeta, or GPIX)
2. Abnormalities of platelet granules
3. Abnormalities of platelet signal transduction
4. Abnormalities of platelet secretion
5. Collagen Receptor Defect
6. ADP Receptor Defect
7. Thromboxane Receptor Defect
8. Giant Platelet Disorder
9. Abnormalities in platelet aggregation testing due to another or unknown cause (not
drug related)
Exclusion Criteria:
Platelet disorders secondary to medications or other substances
Rare Disorders Cohort
Inclusion Criteria:
Participants who meet the following inclusion criteria are eligible for enrollment into
this cohort:
1. Have an established Rare Coagulation Disorder (RCD) diagnosis of one of the following:
1. PAI-1 deficiency
2. Factor I, II, V, VII, X, XI, XIII deficiencies
3. Combined FV and FVIII deficiency
4. Plasminogen deficiency
5. Decreased tissue plasminogen activator
6. Afibrinogenemia/hypofibrinogenemia/dysfibrinogenemia
Exclusion Criteria:
None
Bleeding NOS Cohort
Inclusion Criteria:
Participants who meet the following inclusion criteria are eligible for enrollment into
this cohort:
1. Have a bleeding phenotype as indicated by an age-adjusted abnormal ISTH Bleeding
Assessment Tool score with an unknown diagnosis; OR
2. Connective tissue disorder with bleeding tendency as indicated by an age-adjusted
abnormal ISTH Bleeding Assessment Tool score
Exclusion Criteria:
None
Thrombosis/Thrombophilia Cohort
Inclusion Criteria
Participants who meet the following inclusion criteria are eligible for enrollment into
this cohort:
1. Have a prior history of arterial or venous thrombosis
2. Patients with a known congenital or acquired thrombophilia with or without a
thrombosis a. Common congenital thrombophilias:: i. Protein C deficiency ii. Protein S
deficiency iii. Antithrombin deficiency iv. Factor V Leiden v. Prothrombin gene
mutation b. Rare genetic factors i. Hyperhomocysteinemia c. Indeterminate genetic
factors i. Elevated factor VIII ii. Elevated factor IX iii. Elevated factor XI iv.
Elevated lipoprotein (a) d. Acquired thrombophilias i. Lupus anticoagulant ii.
Anti-cardiolipin antibodies/Beta2 glycoprotein antibodies iii. Antiphospholipid
syndrome
Exclusion Criteria
1. Acquired thrombophilia secondary to medications (birth control pills or hormone
replacement therapy, overweight or obesity, smoking, cancer, pregnancy, surgery, injury,
prolonged inactivity/bedrest, heart failure, inflammatory bowel disease, or kidney disease
Non-Neoplastic Hematologic Conditions Cohort
Inclusion Criteria
Participants who meet the following inclusion criteria are eligible for enrollment into
this cohort:
1. Having any congenital or acquired non-neoplastic hematologic disorder not included in
any other cohort
Exclusion Criteria
None
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Eligibility last updated 5/2/23. Questions regarding updates should be directed to the study team contact.