Natural History Study for Blood Disorders
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to gather important long-term safety and effectiveness data on treatments for individuals with non-cancerous blood disorders and connective tissue disorders that cause bleeding. The study will evaluate the efficacy and safety of these treatments over time. It involves several groups, each focusing on specific conditions such as hemophilia, Von Willebrand Disease, and platelet disorders. Individuals with these conditions who experience frequent bleeding might be suitable candidates for this study. As a Phase 4 trial, this research involves treatments already FDA-approved and proven effective, helping to understand their benefits for more patients.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor to get a clear answer based on your specific situation.
Why are researchers excited about this trial?
Researchers are excited about the ATHN Transcends trial because it seeks to uncover valuable insights into non-cancerous blood disorders, which could lead to improved patient care. Unlike traditional treatments that primarily focus on managing symptoms, this study aims to gather comprehensive data on the natural progression of these conditions, potentially revealing new therapeutic targets. By understanding the detailed history and progression of these disorders, the trial could pave the way for personalized treatment approaches and innovative therapies tailored to individual patient needs.
Who Is on the Research Team?
Michael Recht, MD, PhD, MBA
Principal Investigator
Yale University School of Medicine & National Bleeding Disorders Foundation
Tammuella Chrisentery-Singleton, MD
Principal Investigator
ATHN, Ochsner Clinic Foundation
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Enrollment and Baseline Data Collection
Harmonized data elements are collected at the time of enrollment
Longitudinal Data Collection
Participants are followed for a minimum of 15 years with data collection quarterly, annually, and ad hoc
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ATHN Transcends
Find a Clinic Near You
Who Is Running the Clinical Trial?
American Thrombosis and Hemostasis Network
Lead Sponsor
Genentech, Inc.
Industry Sponsor
Ashley Magargee
Genentech, Inc.
Chief Executive Officer since 2024
MBA from Harvard University, BA from Princeton University
Levi Garraway
Genentech, Inc.
Chief Medical Officer since 2021
MD, PhD
Sanofi
Industry Sponsor
Paul Hudson
Sanofi
Chief Executive Officer since 2019
Degree in Economics from Manchester Metropolitan University
Christopher Corsico
Sanofi
Chief Medical Officer
MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University
Novo Nordisk A/S
Industry Sponsor
Lars Fruergaard Jørgensen
Novo Nordisk A/S
Chief Executive Officer since 2017
MSc in Finance and Business Administration, Aarhus School of Business, Aarhus University, Denmark
Martin Holst Lange
Novo Nordisk A/S
Chief Medical Officer since 2021
MD from University of Copenhagen
Hemab Therapeutics
Collaborator
CSL Behring
Industry Sponsor
Dr. Paul McKenzie
CSL Behring
Chief Executive Officer since 2023
PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania
Dr. Bill Mezzanotte
CSL Behring
Chief Medical Officer since 2021
MD from Duke University
Hemophilia of Georgia, Inc.
Collaborator
Pfizer
Industry Sponsor
Albert Bourla
Pfizer
Chief Executive Officer since 2019
PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki
Patrizia Cavazzoni
Pfizer
Chief Medical Officer
MD from McGill University