American Thrombosis and Hemostasis Network
In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have yet to demonstrate 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.(1,2,3,4) In 2019 alone, the United States Food and Drug Administration (FDA) has issued approvals for twenty-four new therapies for congenital and acquired hematologic conditions.(5) In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.(6) With this increase in potential new therapies on the horizon, 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. 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. The study consists of 7 cohorts with additional study "arms" and "modules" branching off from the cohorts. The overarching objective of this longitudinal, observational study is to characterize the safety, effectiveness and practice of treatments for all people with congenital and acquired hematologic disorders in the US. 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.(7)
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
This is a longitudinal, natural history observational cohort study being conducted at approximately 150 ATHN-affiliated sites. Participants will be followed for a minimum of 15 years. Harmonized data elements will be collected at the time of enrollment, quarterly, annually, and ad hoc. Base data will be collected for all participants. Specific 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 may be developed to provision disease and/or disease specific insights related to stakeholders, including but not limited to pharmaceutical companies, ATHN, and Hemophilia Treatment Centers (HTCs). 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 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: Co-Principal Investigators Shannon Carpenter, MD, MS University of Missouri Kansas City School of Medicine Children's Mercy Hospital Co-Principal Investigator Julie Jaffray, MD University of California San Diego Rady Children's Hospital San Diego INHIBIT Module: Co-Principal Investigators: Nicoletta Machin DO, MS Assistant Professor, Division of Hematology/Oncology Hemophilia Center of Western Pennsylvania University of Pittsburgh Medical Center Hemophilia Natural History Arm: Co-Principal Investigators: Tyler Buckner, MD, MSc Hemophilia and Thrombosis Center University of Colorado Anschutz Medical Campus Michael Recht, MD, PhD, MBA Yale University School of Medicine National Bleeding Disorders Foundation Rebinyn Module Co-Principal Investigators: Lauren Amos, MD Children's Mercy Hospital, Kansas City Guy Young, MD University of Southern California Children's Hospital Los Angeles 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 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 Rainbow Babies \& Children's Hospital, Case Western Reserve University Glanzmann Thrombasthenia Module: Co-Principal Investigators: Divya Citla-Sridhar, MD University of Arkansas for Medical Sciences Arkansas Children's Hospital Meera Chitlur, MD Children's Hospital of Michigan Hemophilia Cohort This cohort includes three Arms and five Modules: Previously Untreated Patients (PUPs) Arm This is a pediatric focused Arm of PUPs with hemophilia A or B. ALTUVIIIO® Module The purpose is to investigate the safety and effectiveness of ALTUVIIIO® in PUPs with hemophilia A. INHIBIT Module This is an observational study assessing inhibitor formation in children with severe hemophilia A. Hemophilia Natural History Arm This Arm is investigating the safety, effectiveness, and practice of treatment for people with hemophilia. Hemlibra® Module All participants treated with Hemlibra® are eligible to participate. 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. 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 safety and efficacy of hemostatic therapies in the prevention or treatment of bleeding events 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.
| Study Type : | OBSERVATIONAL |
| Estimated Enrollment : | 3000 participants |
| Official Title : | ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People With Non-Neoplastic Hematologic Disorders |
| Actual Study Start Date : | 2020-09-30 |
| Estimated Primary Completion Date : | 2035-06 |
| Estimated Study Completion Date : | 2035-12 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | |
| Sexes Eligible for Study: | ALL |
| Accepts Healthy Volunteers: |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
RECRUITING
Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
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Arkansas Center for Bleeding Disorders
Little Rock, Arkansa, United States, 72202
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Orthopaedic Institute for Children HTC
Los Angeles, California, United States, 90007
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Childrens Hospital Los Angeles
Los Angeles, California, United States, 90027-6016
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UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94610
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University of California at Davis Hemophilia Treatment Center
Sacramento, California, United States, 95817
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Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program
San Bernardino, California, United States, 92408
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Hemophilia & Thrombosis Treatment Center at UC San Diego Health
San Diego, California, United States, 92121
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Rady Children's Hospital San Diego
San Diego, California, United States, 92123
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University of California, San Francisco Hemophilia & Thrombosis Center
San Francisco, California, United States, 94143
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University of Colorado Denver Hemophilia and Thrombosis Center
Aurora, Colorado, United States, 80045
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Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
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Yale Hemophilia Treatment Center
New Haven, Connecticut, United States, 06520
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Delaware Hemophilia Treatment Center
Wilmington, Delaware, United States, 19801
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Georgetown University
Washington D.C., District of Columbia, United States, 20007
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Children's National Hemophilia Center
Washington D.C., District of Columbia, United States, 20010
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University of Florida Hemophilia Treatment Center
Gainesville, Florida, United States, 32610
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University of Miami Comprehensive Hemophilia Treatment Center
Miami, Florida, United States, 33136
NOT YET RECRUITING
Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders
Orlando, Florida, United States, 32806
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Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States, 33701
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St. Joseph's Hospital Center for Bleeding & Clotting Disorders
Tampa, Florida, United States, 33607
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Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30308
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Emory/Children's Health Care of Atlanta
Atlanta, Georgia, United States, 30322
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Memorial Health University Medical Center
Savannah, Georgia, United States, 31403
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Rush University Medical Center
Chicago, Illinois, United States, 60612
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Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States, 61664
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Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States, 46260
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Iowa Hemophilia and Thrombosis Center
Iowa City, Iowa, United States, 52242
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Louisiana Center for Bleeding and Clotting Disorders, Tulane University
New Orleans, Louisiana, United States, 70112
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Maine Hemophilia and Thrombosis Center
Scarborough, Maine, United States, 04074
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Johns Hopkins University Hemophilia Treatment Center
Baltimore, Maryland, United States, 21287
NOT YET RECRUITING
Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center
Boston, Massachusetts, United States, 02114
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Central Michigan Children's Hospital of Michigan
Detroit, road cancer, United States, 48201
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Henry Ford Health System Bleeding and Thrombosis Treatment Center
Detroit, road cancer, United States, 48202
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Mayo Comprehensive Hemophilia Center
Rochester, Minnesota, United States, 55905
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Children's Mercy Hospital - Kansas City
Kansas City, Missouri, United States, 64108
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The John Bouhasin Center for Children with Bleeding Disorders
St Louis, Missouri, United States, 63104
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Cure 4 The Kids Foundation
vegas, Nevada, United States, 89135
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Hemostasis and Thrombosis Center of Nevada
Reno, Nevada, United States, 89509
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Newark Beth Israel Medical Center - Hemophilia Center
Newark, New Jersey, United States, 07122
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University of New Mexico Ted R. Montoya Hemophilia & Thrombosis Program
Albuquerque, New Mexico, United States, 87131
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Western New York BloodCare
Buffalo, New York, United States, 14202
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Northwell Health Hemostasis and Thrombosis Center at Long Island Jewish and Cohen Children's Medical Center
Hyde Park, New York, United States, 11040
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Weill Cornell Medical College - New York Presbyterian Hospital
New York, New York, United States, 10065
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American Thrombosis and Hemostasis Network
Rochester, New York, United States, 14626
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Montefiore Medical Center
The Bronx, New York, United States, 10461
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Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27517
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St. Jude Affiliate Clinic at Novant Health Hemby Children's Hospital
Charlotte, North Carolina, United States, 28204
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East Carolina University Hemophilia Treatment Center
Greenville, North Carolina, United States, 27834
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Wake Forest University Health Sciences
Winston-Salem, North Carolina, United States, 27157
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Akron Children's Hospital - Showers Center for Cancer & Blood Disorders
Akron, Ohio, United States, 44308
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Cincinnati Children's Hospital Medical Center, Hemophilia & Thrombosis Center
Cincinnati, Ohio, United States, 45229
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University of Cincinnati Medical Center Hemophilia Treatment Center
Cincinnati, Ohio, United States, 45267
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University Hospitals Health System Cleveland
Cleveland, Ohio, United States, 44106
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Nationwide Children's Hospital Columbus
Columbus, Ohio, United States, 43205
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Dayton Children's Hemostasis and Thrombosis Center
Dayton, Ohio, United States, 45404
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Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital
Toledo, Ohio, United States, 43606
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
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Penn Comprehensive Hemophilia and Thrombophilia Program/Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
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Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213
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Rhode Island Hospital Hemostasis and Thrombosis Center
Providence, Rhode Island, United States, 02903
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St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
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Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37212
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Children's Blood and Cancer Center of Central Texas
Austin, Texas, United States, 78723
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North Texas Hemophilia and Thrombosis Program - Pediatric Program / Center for Cancer & Blood Disorders
dallas, Texas, United States, 75235
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North Texas Comprehensive Hemophilia Treatment Center
dallas, Texas, United States, 75390
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Fort Worth Bleeding Disorders Program
Fort Worth, Texas, United States, 76104
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Gulf States Hemophilia and Thrombophilia Center
Houston, Texas, United States, 77030
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Texas Children's Hemophilia & Thrombosis Center/Baylor College of Medicine
Houston, Texas, United States, 77030
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South Texas Comprehensive Hemophilia and Thrombophilia Treatment Center
Saint Anthony, Texas, United States, 78229
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Washington Center for Bleeding Disorders
Seattle, Washington, United States, 98101
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Hemophilia Outreach Center Green Bay
Green Bay, Wisconsin, United States, 54311
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Comprehensive Center for Bleeding Disorders
Milwaukee, Wisconsin, United States, 53226