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

Recruiting now

Conditions studied: Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease

In brief

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, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.8

Key facts

Study ID

NCT04398628

Run by

American Thrombosis and Hemostasis Network

People needed

3000

Starts

2020-09-30

Expected to finish

2035-12-01

Last updated by the study team

2026-07-13

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Any age
• Having a congenital or acquired blood disorder; or
• Having a bleeding phenotype as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score with an unknown diagnosis; or
• Connective tissue disorder with bleeding tendency as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score.
• Eligible for a currently active disease-specific arm.
• Concurrent enrollment in the ATHNdataset or current ATHNdataset participant.

You may not qualify if…

• 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:
• 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
• 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
• 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:
• 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:
• Abnormalities of platelet function a. Glanzmann thrombasthenia (GPIIb or GPIIIa) b. Bernard-Soulier syndrome (GPIbalpha, GPIbbeta, or GPIX)
• Abnormalities of platelet granules
• Abnormalities of platelet signal transduction
• Abnormalities of platelet secretion
• Collagen Receptor Defect

Where it is running

• Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital — Phoenix, Arizona, United States (enrolling)
• Orthopaedic Institute for Children HTC — Los Angeles, California, United States (enrolling)
• Arkansas Center for Bleeding Disorders — Little Rock, Arkansas, United States (enrolling)
• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States (enrolling)
• University of California at Davis Hemophilia Treatment Center — Sacramento, California, United States (enrolling)
• Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program — San Bernardino, California, United States (enrolling)
• Hemophilia & Thrombosis Treatment Center at UC San Diego Health — San Diego, California, United States (enrolling)
• Childrens Hospital Los Angeles — Los Angeles, California, United States (enrolling)
• University of California, San Francisco Hemophilia & Thrombosis Center — San Francisco, California, United States (enrolling)
• Connecticut Children's Medical Center — Hartford, Connecticut, United States (enrolling)
• Yale Hemophilia Treatment Center — New Haven, Connecticut, United States (enrolling)
• Delaware Hemophilia Treatment Center — Wilmington, Delaware, United States (enrolling)
• Georgetown University — Washington D.C., District of Columbia, United States (enrolling)
• Children's National Hemophilia Center — Washington D.C., District of Columbia, United States (enrolling)
• University of Florida Hemophilia Treatment Center — Gainesville, Florida, United States (enrolling)
• University of Miami Comprehensive Hemophilia Treatment Center — Miami, Florida, United States (enrolling)
• Rady Children's Hospital San Diego — San Diego, California, United States (enrolling)
• Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States (enrolling)
• St. Joseph's Hospital Center for Bleeding & Clotting Disorders — Tampa, Florida, United States (enrolling)
• Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta — Atlanta, Georgia, United States (enrolling)
• Emory/Children's Health Care of Atlanta — Atlanta, Georgia, United States (enrolling)
• Memorial Health University Medical Center — Savannah, Georgia, United States (enrolling)
• Rush University Medical Center — Chicago, Illinois, United States (enrolling)
• Bleeding and Clotting Disorders Institute — Peoria, Illinois, United States (enrolling)
• Indiana Hemophilia and Thrombosis Center — Indianapolis, Indiana, United States (enrolling)

Full record on ClinicalTrials.gov

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