ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
1 other identifier
observational
3,000
1 country
71
Brief 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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Sep 2020
Longer than P75 for all trials
71 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 11, 2020
CompletedFirst Posted
Study publicly available on registry
May 21, 2020
CompletedStudy Start
First participant enrolled
September 30, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2035
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2035
January 12, 2026
January 1, 2026
14.7 years
May 11, 2020
January 9, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
To determine the safety of therapies used in the treatment of participants with congenital or acquired non-neoplastic, bleeding and clotting disorders and connective tissue disorders with bleeding tendency (blood disorders).
Safety will be measured by those events in the European Safety Surveillance (EUHASS)1: 1. Allergic or other acute events 2. Treatment-emergent side effects of therapy 3. Transfusion transmitted infections 4. Inhibitor development 5. Thrombosis 6. Cardiovascular events 7. Malignancies 8. Neurological events 9. Death In addition to the modified EUHASS endpoints, the following events will be collected as adverse events of special interest (AESI): 1. The occurrence of thrombotic microangiopathies, injection site reactions and cases of potential drug-induced liver injury 2. The development of anti-drug antibodies, to be measured and confirmed, if feasible 3. Severe, unanticipated bleeding 4. Hospitalizations 5. Glomerulonephritis 6. Any arm or module-specific AESI as stated in their corresponding Safety Assessment section Additional safety events of interest may be collected.
15 years
Secondary Outcomes (5)
To establish a platform to support study arms and modules for participants with blood disorders.
15 years
To describe medication dosing regimens in participants with blood disorders.
15 years
To describe real-world effectiveness of therapies used for participants with blood disorders.
15 years
To grow and evolve the ATHN Transcends Biorepository for current and future research through the collection of biospecimens from participants enrolled on this protocol
15 years
To describe bleeding events, changes in overall bleeding, and annualized bleeding rate (ABR) as measured by individual bleeding components.
15 years
Study Arms (7)
Hemophilia
This cohort includes three Arms and six Modules: Previously Untreated Patients (PUPs) Arm Efanestoctocog alfa (ALTUVIIIO®) Module INHIBIT Module Hemophilia Natural History Arm Emicizumab (Hemlibra®) Module Nonacog beta pegol (Rebinyn®)Module Distress Module Hemophilia Gene Therapy Outcomes Arm Etranacogene dezaparvovec (HEMGENIX®) Module
Congenital Platelet Disorders
This cohort includes one Arm and Module: Congenital Platelet Disorders (CPD) Natural History Arm Glanzmann Thrombasthenia (GT) Module
Von Willebrand Disease
No arms or modules
Rare Disorders
No arms or modules
Bleeding NOS
No arms or modules
Thrombosis/Thrombophilia
No arms or modules
Non-Neoplastic Hematologic Conditions
No arms or modules
Eligibility Criteria
This is a real-world study in which participants with congenital or acquired blood disorders will be enrolled.
You may not qualify if:
- Any age
- Having a congenital or acquired blood disorder; or
- Having a bleeding phenotype as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score with an unknown diagnosis; or
- Connective tissue disorder with bleeding tendency as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score.
- Eligible for a currently active disease-specific arm.
- Concurrent enrollment in the ATHNdataset or current ATHNdataset participant.
- Cohort Participant Selection
- Each participant is to be enrolled in the cohort for which they qualify as defined below.
- Hemophilia Cohort
- Factor VIII or factor IX activity \<50%, without another explanation for low clotting factor other than congenital hemophilia or being a known carrier for congenital hemophilia; OR
- Carrier for congenital hemophilia with a factor VIII \>=50% or factor IX activity \>=50% with or without a bleeding phenotype as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years OR
- Known congenital hemophilia that have a factor level \>50% after receiving vector, OR 4. Acquired hemophilia.
- None
- Von Willebrand Disease Cohort
- \. Meeting the definition of VWD or low VWF per most recent international guidelines
- +154 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- American Thrombosis and Hemostasis Networklead
- Pfizercollaborator
- Hemophilia of Georgia, Inc.collaborator
- Genentech, Inc.collaborator
- CSL Behringcollaborator
- Sanoficollaborator
- Novo Nordisk A/Scollaborator
- Hemab ApScollaborator
Study Sites (71)
Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Arkansas Center for Bleeding Disorders
Little Rock, Arkansas, 72202, United States
Orthopaedic Institute for Children HTC
Los Angeles, California, 90007, United States
Childrens Hospital Los Angeles
Los Angeles, California, 90027-6016, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94610, United States
University of California at Davis Hemophilia Treatment Center
Sacramento, California, 95817, United States
Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program
San Bernardino, California, 92408, United States
Hemophilia & Thrombosis Treatment Center at UC San Diego Health
San Diego, California, 92121, United States
Rady Children's Hospital San Diego
San Diego, California, 92123, United States
University of California, San Francisco Hemophilia & Thrombosis Center
San Francisco, California, 94143, United States
Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
Yale Hemophilia Treatment Center
New Haven, Connecticut, 06520, United States
Delaware Hemophilia Treatment Center
Wilmington, Delaware, 19801, United States
Georgetown University
Washington D.C., District of Columbia, 20007, United States
Children's National Hemophilia Center
Washington D.C., District of Columbia, 20010, United States
University of Florida Hemophilia Treatment Center
Gainesville, Florida, 32610, United States
University of Miami Comprehensive Hemophilia Treatment Center
Miami, Florida, 33136, United States
Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders
Orlando, Florida, 32806, United States
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701, United States
St. Joseph's Hospital Center for Bleeding & Clotting Disorders
Tampa, Florida, 33607, United States
Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta
Atlanta, Georgia, 30308, United States
Emory/Children's Health Care of Atlanta
Atlanta, Georgia, 30322, United States
Memorial Health University Medical Center
Savannah, Georgia, 31403, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61664, United States
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260, United States
Iowa Hemophilia and Thrombosis Center
Iowa City, Iowa, 52242, United States
Louisiana Center for Bleeding and Clotting Disorders, Tulane University
New Orleans, Louisiana, 70112, United States
Maine Hemophilia and Thrombosis Center
Scarborough, Maine, 04074, United States
Johns Hopkins University Hemophilia Treatment Center
Baltimore, Maryland, 21287, United States
Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center
Boston, Massachusetts, 02114, United States
Central Michigan Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
Henry Ford Health System Bleeding and Thrombosis Treatment Center
Detroit, Michigan, 48202, United States
Mayo Comprehensive Hemophilia Center
Rochester, Minnesota, 55905, United States
Children's Mercy Hospital - Kansas City
Kansas City, Missouri, 64108, United States
The John Bouhasin Center for Children with Bleeding Disorders
St Louis, Missouri, 63104, United States
Cure 4 The Kids Foundation
Las Vegas, Nevada, 89135, United States
Hemostasis and Thrombosis Center of Nevada
Reno, Nevada, 89509, United States
Newark Beth Israel Medical Center - Hemophilia Center
Newark, New Jersey, 07122, United States
University of New Mexico Ted R. Montoya Hemophilia & Thrombosis Program
Albuquerque, New Mexico, 87131, United States
Western New York BloodCare
Buffalo, New York, 14202, United States
Northwell Health Hemostasis and Thrombosis Center at Long Island Jewish and Cohen Children's Medical Center
Hyde Park, New York, 11040, United States
Weill Cornell Medical College - New York Presbyterian Hospital
New York, New York, 10065, United States
American Thrombosis and Hemostasis Network
Rochester, New York, 14626, United States
Montefiore Medical Center
The Bronx, New York, 10461, United States
Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27517, United States
St. Jude Affiliate Clinic at Novant Health Hemby Children's Hospital
Charlotte, North Carolina, 28204, United States
East Carolina University Hemophilia Treatment Center
Greenville, North Carolina, 27834, United States
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
Akron Children's Hospital - Showers Center for Cancer & Blood Disorders
Akron, Ohio, 44308, United States
Cincinnati Children's Hospital Medical Center, Hemophilia & Thrombosis Center
Cincinnati, Ohio, 45229, United States
University of Cincinnati Medical Center Hemophilia Treatment Center
Cincinnati, Ohio, 45267, United States
University Hospitals Health System Cleveland
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital Columbus
Columbus, Ohio, 43205, United States
Dayton Children's Hemostasis and Thrombosis Center
Dayton, Ohio, 45404, United States
Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital
Toledo, Ohio, 43606, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Penn Comprehensive Hemophilia and Thrombophilia Program/Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213, United States
Rhode Island Hospital Hemostasis and Thrombosis Center
Providence, Rhode Island, 02903, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
Children's Blood and Cancer Center of Central Texas
Austin, Texas, 78723, United States
North Texas Hemophilia and Thrombosis Program - Pediatric Program / Center for Cancer & Blood Disorders
Dallas, Texas, 75235, United States
North Texas Comprehensive Hemophilia Treatment Center
Dallas, Texas, 75390, United States
Gulf States Hemophilia and Thrombophilia Center
Houston, Texas, 77030, United States
Texas Children's Hemophilia & Thrombosis Center/Baylor College of Medicine
Houston, Texas, 77030, United States
South Texas Comprehensive Hemophilia and Thrombophilia Treatment Center
San Antonio, Texas, 78229, United States
Washington Center for Bleeding Disorders
Seattle, Washington, 98101, United States
Hemophilia Outreach Center Green Bay
Green Bay, Wisconsin, 54311, United States
Comprehensive Center for Bleeding Disorders
Milwaukee, Wisconsin, 53226, United States
Related Publications (8)
Weijer C, Freedman B, Fuks A, Robbins J, Shapiro S, Skrutkowska M. What difference does it make to be treated in a clinical trial? A pilot study. Clin Invest Med. 1996 Jun;19(3):179-83.
PMID: 8724821BACKGROUNDBraunholtz DA, Edwards SJ, Lilford RJ. Are randomized clinical trials good for us (in the short term)? Evidence for a "trial effect". J Clin Epidemiol. 2001 Mar;54(3):217-24. doi: 10.1016/s0895-4356(00)00305-x.
PMID: 11223318BACKGROUNDWest J, Wright J, Tuffnell D, Jankowicz D, West R. Do clinical trials improve quality of care? A comparison of clinical processes and outcomes in patients in a clinical trial and similar patients outside a trial where both groups are managed according to a strict protocol. Qual Saf Health Care. 2005 Jun;14(3):175-8. doi: 10.1136/qshc.2004.011478.
PMID: 15933313BACKGROUNDUnger JM, Barlow WE, Martin DP, Ramsey SD, Leblanc M, Etzioni R, Hershman DL. Comparison of survival outcomes among cancer patients treated in and out of clinical trials. J Natl Cancer Inst. 2014 Mar;106(3):dju002. doi: 10.1093/jnci/dju002. Epub 2014 Mar 13.
PMID: 24627276BACKGROUNDhttps://www.fda.gov/drugs/resources-information-approved-drugs/hematologyoncology-cancer-approvals-safety-notifications. Accessed 04 Jul 2019
BACKGROUNDhttps://www.clinicaltrials.gov/ct2/results?cond=Hematologic+Diseases&term=&cntry=&state=&city=&dist=. Accessed 04 Jul 2019
BACKGROUNDKonkle BA, Recht M; members of Working Group 2, the NHLBI State of the Science Workshop on factor VIII inhibitors: Generating a national blueprint for future research. The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors. Haemophilia. 2019 Jul;25(4):590-594. doi: 10.1111/hae.13772.
PMID: 31329362BACKGROUNDIorio A, Keepanasseril A, Foster G, Navarro-Ruan T, McEneny-King A, Edginton AN, Thabane L; WAPPS-Hemo co-investigator network. Development of a Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo): Study Protocol. JMIR Res Protoc. 2016 Dec 15;5(4):e239. doi: 10.2196/resprot.6558.
PMID: 27977390BACKGROUND
Biospecimen
All participants have the option to provide consent to have specimens stored in the ATHN Transcends Biorepository. Inhibitor titer testing will be performed locally (unless otherwise specified) as per standard of care which is generally expected to coincide with the following: * Baseline Visit * Annual Visit * Product switch * Suspected inhibitor development * Confirmatory test for previously elevated result * Study Exit * Additional timepoints per any modules Genetic testing (performed once) will be optional and provided by central labs, as funding allows, across all cohorts. Anti-drug antibodies testing - For participants receiving non-factor products (e.g., emicizumab) and if testing is clinically appropriate (suspicion of anti-drug antibodies forming), this specimen will be analyzed, as funding allows. Factor activity testing will be performed locally at the following timepoints: * Baseline visit * 6 and 18-month follow-up visits * Annual visit * Ad hoc * Study arm exit
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Michael Recht, MD, PhD, MBA
Yale University School of Medicine & National Bleeding Disorders Foundation
- PRINCIPAL INVESTIGATOR
Tammuella Chrisentery-Singleton, MD
ATHN, Ochsner Clinic Foundation
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 11, 2020
First Posted
May 21, 2020
Study Start
September 30, 2020
Primary Completion (Estimated)
June 1, 2035
Study Completion (Estimated)
December 1, 2035
Last Updated
January 12, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will not share