ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders (NCT04398628) | Clinical Trial Compass
RecruitingNot Applicable
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
United States3,000 participantsStarted 2020-09-30
Plain-language 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
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Exclusion criteria
. 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.
. 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a natural history study with no assigned phase, it seems focused on observing and collecting data rather than testing a new treatment — can you help me understand what that would actually mean for my day-to-day care if I enrolled?
2This study covers a wide range of conditions, including my specific disorder — how would participating affect my current treatment plan, and would I still receive the same standard therapies I'd otherwise get?
3The study is measuring the safety of therapies already used to treat blood and clotting disorders — does that mean my treatment decisions would be guided by the research team, or would you as my doctor remain in full control of what I receive?
4Given that this is a long-term observational registry, what kind of ongoing time commitment would I be making — for example, how often would I need to come in for visits or provide data?
5Before I consider joining this study, are there standard treatment options or other clinical trials more directly aimed at treating my specific condition that might be worth exploring first?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To determine the safety of therapies used in the treatment of participants with congenital or acquired non-neoplastic, bleeding and clotting disorders and connective tissue disorders with bleeding tendency (blood disorders).