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

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

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

This is a longitudinal, natural history observational cohort study being conducted at approximately 150 ATHN-affiliated sites with a target accrual of 3,000 participants. Participants will be followed for a minimum of 15 years on an assigned arm within a cohort; however, arm or module participation may last longer, and participants will continue participation in the arm or module for its duration. Harmonized data elements will be collected at the time of enrollment, semi-annually (every 6 months), annually, ad hoc, and as defined by the terms of individual arms and modules. Data will be collected for participants enrolled in cohort-specific arms and modules. Each participant will be assigned to a single cohort: Hemophilia, von Willebrand Disease, Congenital Platelet Disorders, Rare Disorders, Bleeding Not Otherwise Specified (NOS), Thrombosis/Thrombophilia, or Non-Neoplastic Hematologic Conditions. Study arms and study modules are developed to advance the exploration of blood disorders disease specific insights by ATHN and its partners. Arms may branch off into product-specific data collection via Modules to be collected during the study, in conjunction with planned study assessments. ATHN Transcends Co- Principal Investigators: Tammuella Chrisentery-Singleton, MD Ochsner Clinic Foundation American Thrombosis and Hemostasis Network Michael Recht, MD, PhD, MBA Yale University School of Medicine National Bleeding Disorders Foundation PUPs Arm Principal Investigator: Shannon Carpenter, MD, MS University of Missouri Kansas City School of Medicine Children's Mercy Hospital ALTUVIIO Module Principal Investigator: Shannon Carpenter, MD, MS University of Missouri Kansas City School of Medicine Children's Mercy Hospital INHIBIT Module Principal Investigator: Nicoletta Machin DO, MS Hemophilia Center of Western Pennsylvania University of Pittsburgh Medical Center Hemophilia Natural History Arm Principal Investigator: Fernando Corrales-Medina, MD, FAAP University of Miami-Comprehensive Hemophilia Treatment Center University of Miami-Miller School of Medicine Rebinyn Module Co-Principal Investigators: Lauren Amos, MD University of Missouri Kansas City School of Medicine Children's Mercy Hospital Guy Young, MD University of Southern California Children's Hospital Los Angeles Distress Module Principal Investigator: Tammuella Chrisentery-Singleton, MD Ochsner Clinic Foundation American Thrombosis and Hemostasis Network Hemlibra Module Principal Investigator: Fernando Corrales-Medina, MD, FAAP University of Miami-Comprehensive Hemophilia Treatment Center University of Miami-Miller School of Medicine Hemophilia Gene Therapy Outcomes Arm: Co-Principal Investigators: Janice M. Staber, MD Iowa Hemophilia and Thrombosis Center University of Iowa Stead Family Children's Hospital Ulrike M. Reiss, MD Hemophilia Treatment Center St. Jude's Children's Research Hospital HEMGENIX Module Co-Principal Investigators: Janice M. Staber, MD Iowa Hemophilia and Thrombosis Center University of Iowa Stead Family Children's Hospital Ulrike M. Reiss, MD Hemophilia Treatment Center, St. Jude's Children's Research Hospital Severe VWD Natural History Arm: Co-Principal Investigators: Robert F. Sidonio, Jr., MD, MSc Aflac Cancer and Blood Disorders Center, Hemophilia of Georgia Center for Bleeding and Clotting Disorders Angela C. Weyand, MD C.S. Mott Children's Hospital, University of Michigan Medical School, Ann Arbor Congenital Platelet Disorders Natural History Arm: Principal Investigator Sanjay Ahuja, MD Innovative Hematology, Indiana Hemophilia & Thrombosis Center Glanzmann Thrombasthenia Module: Co-Principal Investigators: Divya Citla-Sridhar, MD University of Arkansas for Medical Sciences Arkansas Children's Hospital Meera Chitlur, MD Central Michigan University, Children's Hospital of Michigan Hemophilia Cohort This cohort includes three Arms and six Modules: Previously Untreated Patients (PUPs) Arm This is a pediatric focused Arm of PUPs with hemophilia A or B of any severity. Efanestoctocog alfa (ALTUVIIIO®) Module The purpose is to investigate the safety, tolerability, and effectiveness of efanesoctocog alfa (ALTUVIIIO®) in PUPs with severe hemophilia A. INHIBIT Module This is an observational study assessing the rate of inhibitor formation in young children with severe hemophilia A in the current treatment era. Hemophilia Natural History Arm This Arm is investigating the safety, effectiveness, and practice of treatment for people with hemophilia. Emicizumab (Hemlibra®) Module All participants treated with Hemlibra® are eligible to participate. Distress Module Participants with Congenital Hemophilia A or B, 18 years of age or older, will be followed longitudinally for 2 years or from time of enrollment for a total planned study duration of 3 years Nonacog beta pegol (Rebinyn®) Module The Rebinyn® Module is a prospective study in hemophilia B participants without inhibitors. Hemophilia Gene Therapy Outcomes Arm This Arm is investigating the safety and effectiveness of gene therapy in people with hemophilia. Etranacogene dezaparvovec (HEMGENIX®) Module This is an observational study to characterize the effectiveness and safety of HEMGENIX® in participants with hemophilia B. Congenital Platelet Disorders (CPD) Natural History Arm: The CPD Arm is investigating the natural history of the safety and efficacy of hemostatic therapies (such as platelet transfusions, desmopressin, antifibrinolytics, recombinant factor VIIa) in the prevention or treatment of bleeding events (on demand, surgery, prophylaxis) in adult and pediatric participants with inherited congenital platelet disorders.. Glanzmann Thrombasthenia (GT) Module: This Module is a study of bleeding symptoms, treatments, and treatment outcomes in patients with Glanzmann thrombasthenia. Von Willebrand Disease Cohort No arms or modules open at this time. Rare Disorders Cohort No arms or modules open at this time. Bleeding NOS No arms or modules open at this time. Thrombosis/Thrombophilia No arms or modules open at this time. Non-Neoplastic Hematologic Conditions No arms or modules open at this time.

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United States