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ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

Recruitingn/aAges any - anyAllAmerican Thrombosis and Hemostasis Network
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In plain words

What the trial is, and whether it could suit you

In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.2,3,4,5 In 2019 alone, the FDA has issued approvals for 24 new therapies for congenital and acquired hematologic conditions.6 In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.7 With this increase in potential new therapies possible, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, s

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📋 Eligibility rules (as published)
Participants who meet the following inclusion criteria and none of the exclusion criteria are eligible for enrollment in one of the open disease-specific arms. Inclusion Criteria: 1. Any age 2. Having a congenital or acquired blood 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. 5. Eligible for a currently active disease-specific arm. 6. Concurrent enrollment in the ATHNdataset or current ATHNdataset participant. Exclusion Criteria: 1\. Does not qualify for inclusion in a currently activedisease-specific arm; participants may be eligible to enroll as future cohorts and arms are activated; 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. Carrier for congenital hemophilia with a factor VIII \>=50% or factor IX activity \>=50% with or without a bleeding phenotype as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years 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 a. Glanzmann thrombasthenia (GPIIb or GPIIIa) b. 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: 1\. 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 7. Thrombotic Thrombocytopenia Purpura or Congenital Hemolytic Uremic Syndrome 8. Wiskott-Aldrich 9. Methylenetetrahydrofolate Reductase Deficiency 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 ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years with an unknown diagnosis, OR 2. Connective tissue disorder with bleeding tendency as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years. 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. Participants with a known congenital or acquired thrombophilia with or without 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 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 Arm/Module Participant Selection Previously Untreated Patients Arm Inclusion Criteria: 1. Diagnosis of congenital hemophilia A (FVIII \<40%) or hemophilia B (FIX \<40% or below lower limit for age) 2. Age \<18 years at time of enrollment 3. Parent or authorized guardian or legally authorized representative (LAR) can provide informed consent 4. Care established at one of the ATHN Transcends participating HTCs 5. Clotting Factor Concentrate (CFC) exposure, fresh frozen plasma (FFP), cryoprecipitate, and single donor platelets \<3 exposure days (ED) Exclusion Criteria 1. Concomitant diagnosis with another bleedin
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What it tests

  • Not listed
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Where

76 sites
Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital
Phoenix, United States
Arkansas Center for Bleeding Disorders
Little Rock, United States
Orthopaedic Institute for Children HTC
Los Angeles, United States
Childrens Hospital Los Angeles
Los Angeles, United States
UCSF Benioff Children's Hospital Oakland
Oakland, United States
University of California at Davis Hemophilia Treatment Center
Sacramento, United States
Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program
San Bernardino, United States
Hemophilia & Thrombosis Treatment Center at UC San Diego Health
San Diego, United States

…and 68 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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