ATHN 8: PUPs Matter Study



Status:Recruiting
Conditions:Anemia, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any - 13
Updated:3/20/2019
Start Date:October 3, 2018
End Date:December 2023
Contact:Angela Riedel
Email:ariedel@athn.org
Phone:800-360-2846

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US Cohort Study of Previously Untreated Patients (PUPs) With Congenital Hemophilia

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.

This multi-center, longitudinal, observational, prospective and retrospective study of
previously untreated patients (PUPs) with moderate to severe hemophilia A or B during the
initial 50 exposure days (ED) to clotting factor replacement product or until the development
of a confirmed inhibitor. The Primary Investigators have designed the study to utilize the
American Thrombosis and Hemostasis Network (ATHN) electronic infrastructure to leverage
existing data and enable the collection of more in-depth clinical and laboratory data on
PUPs. The study aligns with the National Hemophilia Foundation Medical and Scientific
Advisory Council (MASAC) recently issued Recommendation #243 which includes: "Regardless of
which option is chosen, all PUPs should be enrolled in the ATHN data collection system or a
clinical trial to assess outcomes." Co-enrollment in the ATHNdataset by participants is
required. The total study duration is planned for 6 years.

The primary objective is to determine the percentage of patients with confirmed inhibitors
within the first 50 ED. Confirmed inhibitors are defined as two consecutive positive
inhibitor titers (per CDC laboratory criteria; >0.5 Nijmegen Bethesda Units for hemophilia A
and >0.3 Nijmegen Bethesda Units for hemophilia B) on different blood samples which result in
change in treatment recommendations.

Please note - the treatment regimen will be at the discretion of the participants' hemophilia
caregivers. No treatment products are being provided by the study nor will the participants
be paid. However, inhibitor titer testing will be provided at no cost to participants by the
Centers for Disease Control and Prevention (CDC).

All study procedures and follow-up will be timed to coincide with scheduled hemophilia care
whenever possible. Ad hoc, quarterly follow-up, annual and final visits are for participants
who have not met study endpoints (50 ED or inhibitor development) prior to enrollment.

Data collected will include eligibility, demographics, medical history (co-morbidities,
surgery/procedures, immunizations and allergies), hemophilia history (severity, genotype and
family history), birth history, inhibitor testing results, detailed treatment product(s)
usage, bleeding events, bleeding disorder related medical visits during the study, and EUHASS
adverse events.

Sub-studies

A number of sub-studies are planned with pharmaceutical sponsors to collect information from
patients about their specific product use. Participation in these product specific
sub-studies is optional and sub-study visits will be planned to coincide with HTC visits. The
sub-study will collect information from patients about their perception and use of treatment
products, physical activity levels and other general health questions. This data will be
collected via questionnaire.

Data Collection System

All data collected will be entered into electronic case report forms (eCRFs) within the
secure ATHN System by HTC site personnel. All participating study sites will have in place a
current, executed Data Use and Business Associate Agreement (DUBAA) with ATHN.

Inclusion Criteria:

- Congenital hemophilia A; FVIII
- 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

Exclusion Criteria:

- Patients who are referred to the HTC with no record of bleed and factor utilization
data
We found this trial at
27
sites
Madera, California 93638
Principal Investigator: Vinod Balasa, MD
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262 Danny Thomas Pl
Memphis, Tennessee 38105
(901) 495-3300
Principal Investigator: Ulrike Reiss, MD
Phone: 901-595-6411
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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Memphis, TN
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Principal Investigator: Allison Wheeler, MD
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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Nashville, TN
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Michael Recht, MD
Phone: 503-418-4495
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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Portland, OR
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3020 Childrens way
San Diego, California 92123
(858) 576-1700
Principal Investigator: Courtney Thornburg, MD
Rady Children's Hospital - San Diego Rady Children's Hospital-San Diego is the region’s pediatric medical...
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Ann Arbor, Michigan 48109
Principal Investigator: Angela Weyand, MD
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Atlanta, Georgia 30322
Principal Investigator: Robert Sidonio, MD
Phone: 404-727-9698
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Augusta, Georgia 30912
Principal Investigator: Afshin Ameri, MD
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Aurora, Colorado 80045
Principal Investigator: Michael Wang, MD
Phone: 303-724-0363
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300 Longwood Avenue
Boston, Massachusetts 02115
Principal Investigator: Stacy Croteau, MD
Phone: 617-919-6407
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1653 W. Congress Parkway
Chicago, Illinois 60612
(312) 942-5000
Principal Investigator: Mindy Simpson, MD
Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
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Cincinnati, Ohio 45229
Principal Investigator: Cristina Tarango, MD
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Cleveland, Ohio 44106
Principal Investigator: Sanjay Ahuja, MD
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Farmington, Connecticut 06030
Principal Investigator: Jonathan Bernstein, MD
Phone: 860-679-2100
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Green Bay, Wisconsin 54311
Principal Investigator: Kenneth Friedman, MD
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8326 Naab Road
Indianapolis, Indiana 46260
Principal Investigator: Amy Shapiro, MD
Phone: 317-871-0011
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Kansas City, Kansas 64108
Principal Investigator: Shannon Carpenter, MD, MSc
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Milwaukee, Wisconsin 53233
Principal Investigator: Lynn Malec, MD
Phone: 414-257-2424
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Oklahoma City, Oklahoma 73104
Principal Investigator: Osman Khan, MD
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Phoenix, Arizona 85016
Principal Investigator: Christine Knoll, MD
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Rochester, Minnesota 55905
Principal Investigator: Rajiv Pruthi, MD
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Saint Petersburg, Florida 33701
Principal Investigator: Irmel Ayala, MD
Phone: 727-767-4178
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Salt Lake City, Utah 84113
Principal Investigator: Hassan Yaish, MD
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San Francisco, California 94158
Principal Investigator: Tiffany Lin, MD
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Scarborough, Maine 04074
Principal Investigator: Eric Larsen, MD
Phone: 207-396-7312
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Toledo, Ohio 43606
Principal Investigator: Dagmar Stein, MD
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111 Michigan Ave NW
Washington, District of Columbia
(202) 476-5000
Principal Investigator: Michael Guerrera, MD
Phone: 202-476-3622
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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