NCT04398628 ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
| NCT ID | NCT04398628 |
| Status | Recruiting |
| Phase | — |
| Sponsor | American Thrombosis and Hemostasis Network |
| Condition | Hematologic Disorder |
| Study Type | OBSERVATIONAL |
| Enrollment | 3,000 participants |
| Start Date | 2020-09-30 |
| Primary Completion | 2035-06 |
Trial Parameters
Eligibility Fast-Check
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Brief Summary
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
Eligibility Criteria
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 wh