ATHN 8: Previously Untreated Patients (PUPs) Matter Study
US Cohort Study of Previously Untreated Patients (PUPs) With Congenital Hemophilia
1 other identifier
observational
237
1 country
27
Brief Summary
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs). Participants will be followed as they receive their first 50 exposure days (ED) to clotting factor replacement product, both prospectively and retrospectively. The data collected on evolving treatment practices will define the incidence and risk factors for inhibitor development during the high risk period of first 50 ED and improve the outcomes of this vulnerable population.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Oct 2018
Longer than P75 for all trials
27 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 3, 2018
CompletedFirst Submitted
Initial submission to the registry
January 23, 2019
CompletedFirst Posted
Study publicly available on registry
January 28, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 26, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
December 31, 2022
CompletedFebruary 1, 2023
January 1, 2023
3.3 years
January 23, 2019
January 30, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Determine percentage of patients with confirmed inhibitors
Participants will be followed to assess inhibitor development (per CDC laboratory criteria; \>0.5 Nijmegen Bethesda Units for hemophilia A and \>0.3 Nijmegen Bethesda Units for hemophilia B) within 50 exposure days. Blood specimens will be submitted to the CDC for inhibitor testing at various time points outlined in the protocol.
6 years
Secondary Outcomes (9)
Determine risk factors including genetic variants associated with inhibitor development in PUPs
6 years
Determine percentage of eligible participants enrolled at each site
6 years
Determine mean age of diagnosis and first exposure to factor treatment product
6 years
Determine the number of exposure days prior to inhibitor development
6 years
Report bleeding complications that occur within the first 50 ED
6 years
- +4 more secondary outcomes
Eligibility Criteria
The study will enroll a cohort of 250 participants born with hemophilia on or after January 1, 2010 who meet the eligibility criteria and are receiving care from one of the participating HTCs.
You may qualify if:
- Congenital hemophilia A; FVIII \</=5% or congenital hemophilia B; FIX \</=5%;
- Birth date on or after January 1, 2010;
- Care established at one of the participating HTCs;
- Co-enrollment in the ATHNdataset; and
- Parent or authorized guardian can provide informed consent
You may not qualify if:
- Patients who are referred to the HTC with no record of bleed and factor utilization data
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- American Thrombosis and Hemostasis Networklead
- CSL Behringcollaborator
- Octapharmacollaborator
- Takedacollaborator
Study Sites (27)
Arizona Hemophilia and Thrombosis Center at Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Valley Children's Hospital
Madera, California, 93636, United States
Rady Children's Hospital San Diego
San Diego, California, 92123, United States
UCSF Pediatric Hemophilia Treatment Center at Mission Bay
San Francisco, California, 94158, United States
University of Colorado Denver Hemophilia and Thrombosis Center
Aurora, Colorado, 80045, United States
Connecticut Bleeding and Clotting Disorders Center
Farmington, Connecticut, 06030, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701, United States
Children's Healthcare of Atlanta/Emory
Atlanta, Georgia, 30322, United States
Augusta University Hemophilia Treatment Center
Augusta, Georgia, 30912, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Indiana Hemophilia and Thrombosis Center (IHTC)
Indianapolis, Indiana, 46260, United States
Kansas City Regional Hemophilia Center
Kansas City, Kansas, 64108, United States
Maine Hemophilia and Thrombosis Center
Scarborough, Maine, 04074, United States
Boston Hemophilia Center at Children's Hospital of Boston
Boston, Massachusetts, 02115, United States
University of Michigan Hemophilia and Coagulation Disorders
Ann Arbor, Michigan, 48109, United States
Mayo Comprehensive Hemophilia Center
Rochester, Minnesota, 55905, United States
Cincinnati Children's Hospital Medical Center, Hemophilia & Thrombosis Center
Cincinnati, Ohio, 45229, United States
UHHS Cleveland
Cleveland, Ohio, 44106, United States
Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital
Toledo, Ohio, 43606, United States
Oklahoma Center for Bleeding and Clotting Disorders
Oklahoma City, Oklahoma, 73104, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
St Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
Utah Center for Bleeding & Clotting Disorders at Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
Hemophilia Outreach Center
Green Bay, Wisconsin, 54311, United States
Blood Center of Wisconsin
Milwaukee, Wisconsin, 53201, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Shannon Carpenter, MD, MS
Children's Mercy Hospital Kansas City
- PRINCIPAL INVESTIGATOR
Courtney Thornburg, MD, MS
University of California San Diego, Rady Children's Hospital San Diego
- PRINCIPAL INVESTIGATOR
Marijke van den Berg, MD, PhD
Versiti Blood Health
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- OTHER
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 23, 2019
First Posted
January 28, 2019
Study Start
October 3, 2018
Primary Completion
January 26, 2022
Study Completion
December 31, 2022
Last Updated
February 1, 2023
Record last verified: 2023-01