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

Conditions

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

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Study Details

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 Dates

Start date

Sep 30, 2020

Status verified

Jan 2026

Primary completion

Jun 30, 2035

Completion

Dec 31, 2035

Study Design

Enrollment

3,000 participants (estimated)

Arms

• Arm: Hemophilia
  This cohort includes three Arms and six Modules: Previously Untreated Patients (PUPs) Arm Efanestoctocog alfa (ALTUVIIIO®) Module INHIBIT Module Hemophilia Natural History Arm Emicizumab (Hemlibra®) Module Nonacog beta pegol (Rebinyn®)Module Distress Module Hemophilia Gene Therapy Outcomes Arm Etranacogene dezaparvovec (HEMGENIX®) Module
• Arm: Congenital Platelet Disorders
  This cohort includes one Arm and Module: Congenital Platelet Disorders (CPD) Natural History Arm Glanzmann Thrombasthenia (GT) Module
• Arm: Von Willebrand Disease
  No arms or modules
• Arm: Rare Disorders
  No arms or modules
• Arm: Bleeding NOS
  No arms or modules
• Arm: Thrombosis/Thrombophilia
  No arms or modules
• Arm: Non-Neoplastic Hematologic Conditions
  No arms or modules

Primary Outcome Measure

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). [ Time Frame: 15 years ]

Central Contacts

• Carol Fedor, ND, RN, CCRC
  800-360-2846
  [email protected]
• Nana Ama Afari-Dwamena, MPH
  800-360-2846
  [email protected]

Locations (71)

Find similar trials in Phoenix, AZ

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