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

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

Conditions

• 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

Sponsors & Collaborators

• Pfizer

  collaborator INDUSTRY

• Hemophilia of Georgia, Inc.

  collaborator OTHER

• Genentech, Inc.

  collaborator INDUSTRY

• CSL Behring

  collaborator INDUSTRY

• Sanofi

  collaborator INDUSTRY

• Novo Nordisk A/S

  collaborator INDUSTRY

• Hemab ApS

  collaborator INDUSTRY

• American Thrombosis and Hemostasis Network

  lead NETWORK

Principal Investigators

• Michael Recht, MD, PhD, MBA · Yale University School of Medicine & National Bleeding Disorders Foundation

• Tammuella Chrisentery-Singleton, MD · ATHN, Ochsner Clinic Foundation

Eligibility

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2020-09-30

Primary Completion

2035-06-30

Completion

2035-12-31

Countries

• United States

Study Locations