Welcome to ATHN Transcends
ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People with Non-Neoplastic Hematologic Disorders
ATHN Transcends is a cohort study to determine the safety, effectiveness, and practice of therapies used in the treatment of participants with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency. Participants are followed longitudinally for at least 15 years from the time of enrollment. The study consists of 7 cohorts with additional study “arms” and “modules” branching off from the cohorts.
Principal Investigators: Tammuella Chrisentery-Singleton, MD, American Thrombosis and Hemostasis Network; and Michael Recht, MD, PhD, MBA, Yale School of Medicine and National Bleeding Disorders Foundation
Get Involved with ATHN Transcends
ATHN Transcends is growing—with new study arms and modules in development and more investigators and treatment centers signing up to participate.
Contact support@athn.org today for more information about getting involved with ATHN Transcends.
ATHN Transcends Projects
The following cohorts, arms, and modules are in development or open and enrolling at participating sites.
HEMOPHILIA COHORT
ATHN Transcends: Hemophilia Natural History Arm
The Hemophilia Natural History Arm of ATHN Transcends is a longitudinal, observational study that investigates the natural history of the safety, effectiveness, and practice of treatment for participants with acquired or congenital hemophilia A or B.
Principal Investigators: Tyler Buckner, MD, MSc, Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus; and Michael Recht, MD, MBA, PhD, Yale School of Medicine and National Bleeding Disorders Foundation
Current Status: Open and Enrolling
Hemophilia Natural History Arm Modules:
The HEMLIBRA® Module of ATHN Transcends is an open-label, multi-center study that is part of the Hemophilia Natural History Arm of ATHN Transcends. Its primary objective is to describe the safety, effectiveness, and practice of treatment for participants with congenital hemophilia A, with and without inhibitors, taking HEMLIBRA® (emicizumab).
Principal Investigators: Tyler Buckner, MD, MSc, Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus; and Michael Recht, MD, MBA, PhD, Yale University School of Medicine and National Bleeding Disorders Foundation
Current Status: Open and Enrolling
The Rebinyn® Module is an open-label, multi-center study that is part of the Hemophilia Natural History Arm of ATHN Transcends. Its primary objective is to describe the safety and tolerability of nonacog beta pegol, N9-GP in hemophilia B patients who are naïve or minimally exposed (up to 3 exposure days) to nonacog beta pegol, N9-GP.
Principal Investigators: Guy Young, MD, Hemostasis and Thrombosis Program Children’s Hospital Los Angeles; and Lauren Amos, MD, Children’s Mercy Kansas City, University of Missouri-Kansas City
Current Status: Opening Soon
ATHN Transcends: Hemophilia Gene Therapy Outcomes Arm
The Hemophilia Gene Therapy Outcomes Arm is a pragmatic study designed to understand the outcomes of gene therapy with real-world practices across a wide range of products, including the safety of adeno-associated viral vector or lentiviral vector-mediated factor VIII and factor IX therapies when used for participants with hemophilia. This longitudinal, observational arm follows participants for 15 years after vector infusion. Its goal is to enroll all persons who get commercial vector and person who received vector as part of a clinical trial as the trials close.
Principal Investigators: Janice M. Staber, MD, Iowa Hemophilia and Thrombosis Center, University of Iowa Stead Family Children’s Hospital; and Ulrike M. Reiss, MD, Hemophilia Treatment Center, St. Jude Children’s Research Hospital
Current Status: Open and Enrolling
Hemophilia Gene Therapy Outcomes Arm Modules:
HEMGENIX® (etranacogene dezaparovovec) is the first FDA-approved gene therapy for hemophilia B. This module is part of the Hemophilia Gene Therapy Outcomes Arm and investigates the effectiveness and safety of etranacogene dezaparvovec by following adults with hemophilia B for a period of 15 years.
Principal Investigators: Janice M. Staber, MD, Iowa Hemophilia and Thrombosis Center, University of Iowa Stead Family Children’s Hospital; and Ulrike M. Reiss, MD, Hemophilia Treatment Center, St. Jude Children’s Research Hospital
Current Status: Open and Enrolling
ATHN Transcends: Hemophilia PUPs Arm
The Hemophilia PUPs Arm of ATHN Transcends aims to provide a platform for ongoing clinical studies of minimally treated and previously untreated patients (PUPs) with congenital hemophilia A or B. The study will assess safety, such as inhibitor development and associated risk factors, along with effectiveness outcomes.
Principal Investigators: Shannon Carpenter, MD, Children’s Mercy Hospital, University of Missouri-Kansas City; and Julie Jaffray, MD, Rady Children’s Hospital, San Diego and University of California San Diego
Current Status: In Development
Hemophilia PUPs Arm Modules:
The ALTUVIIIO® Module is an open-label, single arm, multicenter study evaluating the safety, tolerability, and effectiveness of ALTUVIIIO® (efanesoctocog alfa) in minimally treated and previously untreated patients (PUPs) with hemophilia A. The study will last for a total of up to 7 years and end when at least 50 participants have attained 50 exposure days (EDs).
Principal Investigators: Shannon Carpenter, MD, Children’s Mercy Hospital, University of Missouri-Kansas City; and Julie Jaffray, MD, Rady Children’s Hospital, San Diego and University of California San Diego
Current Status: Open and Enrolling
The INHIBIT Module is a longitudinal, observational study that aims to describe the rate of inhibitor formation as well as evaluate safety and identify biomarkers/correlates of inhibitor suppression in young children with severe hemophilia A. The minimum planned duration of study participation is approximately 48 weeks.
Principal Investigators: Nicoletta Machin, DO, MS, Hemophilia Center of Western Pennsylvania and University of Pittsburgh Medical Center
Current Status: Open and Enrolling
CONGENITAL PLATELET DISORDERS COHORT
ATHN Transcends: Congenital Platelet Disorders Natural History Arm
The Congenital Platelet Disorders Natural History Arm of ATHN Transcends aims to evaluate the effectiveness and safety of hemostatic therapies (platelet transfusions, desmopressin, antifibrinolytics, recombinant factor VIIa, etc.) in the prevention or treatment of bleeding events—on-demand, surgery, prophylaxis—in children and adults with congenital platelet disorders (CPDs).
Principal Investigator: Sanjay Ahuja, MD, MSc, MBA, Rainbow Babies & Children’s Hospital, Case Western Reserve University
Current Status: In Development
Congenital Platelet Disorders Natural History Arm Modules:
The Glanzmann Thrombasthenia (GT) Module is the first study to open under the Congenital Platelet Disorders Cohort of ATHN Transcends. It is a natural history study that aims to improve scientific understanding of GT by describing the bleeding phenotype in GT, the real-world effectiveness of GT therapies, and create a data set that will contribute to future research.
Principal Investigators: Divyaswathi Citla Sridhar, MD, University of Arkansas for Medical Sciences, Arkansas Children’s Hospital; and Meera Chitlur, MD, Children’s Hospital of Michigan
Current Status: Open and Enrolling
To learn more about ATHN Transcends, visit ClinicalTrials.gov or contact support@athn.org.
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