ATHN 7: Hemophilia Natural History Study



Status:Recruiting
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any
Updated:3/20/2019
Start Date:October 24, 2018
End Date:December 2023
Contact:Angela Riedel
Email:ariedel@athn.org
Phone:800-360-2846

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A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Hemophilia

This is a real-world study of the safety of the treatments used for people with hemophilia.
The study will follow people with hemophilia A or B from across the country for about 4 years
as they receive treatment. The hemophilia treatment center (HTC) physician and participant
will decide on the FDA-approved treatment to be used which may include non-factor products,
bypassing agents, or clotting factor replacement products. The goal of this research is to
study the use of hemophilia treatment products and their outcomes.

This non-interventional, minimal risk cohort study will enroll and follow patients with
hemophilia A or B as they receive hemophilia treatment for 4 years. This is a pragmatic study
of real-world practices across a wide range of patients which will be ongoing as new
treatment products receive FDA approval and will be advantageous to the entire hemophilia
community. The total study duration is planned for 6 years.

The patients are seen at baseline, annually, and at study exit. Patients will also receive
routine quarterly follow-up phone calls from HTC staff to review medical history, bleed
events, and product treatment history. Other visits for unplanned events or for the change of
treatment product will be scheduled as necessary. All required study visits will be planned
to coincide with routine clinical care whenever possible. Co-enrollment in the ATHNdataset by
patients is required to participate in the study.

Please note - the treatment regimen will be at the discretion of the patients' 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 patients by the
Center for Disease Control and Prevention (CDC).

The primary objective is to determine the safety of non-factor products, bypassing agents or
clotting factor replacement products when used for people with hemophilia with or without
inhibitors. Safety will be measured by those events listed in the European Haemophilia Safety
Surveillance (EUHASS).

Data collected will include eligibility, demographics, medical history, hemophilia history
(genotype and family history), inhibitor history and immune tolerance induction (ITI)
treatment regimen (if applicable), co-morbidities at baseline (i.e., HIV, Hepatitis C),
detailed treatment product(s) usage, bleeding events, surgical procedures, and EUHASS adverse
events and other adverse events of special interest. Data collection will also include
patient-reported outcome questionnaires regarding health-related quality of life, treatment
use and patient perceptions of treatment products.

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 sub-studies (Product
Specific Modules) is optional and sub-study visits will be planned to coincide with HTC
visits. The modules 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:

1. Congenital hemophilia A or B of any severity with or without inhibitors receiving a
current therapy, a non-factor product, or for whom use of a non-factor product is a
possibility;

2. Able to give informed consent (by patient or parent/authorized guardian); and

3. Co-enrollment in the ATHNdataset.

Exclusion Criteria:

1. Presence of any known bleeding disorder other than congenital hemophilia A or B;

2. Presence of concurrent hemophilia and a second hemostatic defect (low Von Willebrand
Factor (VWF) without Von Willebrand disease (VWD) diagnosis is not excluded); and

3. Unable or unwilling to comply with the study protocol.
We found this trial at
38
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San Francisco, California 94158
Principal Investigator: Tiffany Lin, MD
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262 Danny Thomas Pl
Memphis, Tennessee 38105
(901) 495-3300
Principal Investigator: Ulrike Reiss, MD
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Michael Recht, MD, PhD
Phone: 503-418-4495
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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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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Albuquerque, New Mexico 87121
Principal Investigator: Shirley Abraham, MD
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Ann Arbor, Michigan 48109
Principal Investigator: Steven Pipe, MD
Phone: 734-936-5905
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Atlanta, Georgia 30322
Principal Investigator: Bobby Tran, MD
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Aurora, Colorado 80045
Principal Investigator: Tyler Buckner, MD, MSc
Phone: 303-724-5700
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300 Longwood Avenue
Boston, Massachusetts 02115
Principal Investigator: Stacy Croteau, MD
Phone: 617-919-6407
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Charlotte, North Carolina 28204
Principal Investigator: Paulette Bryant, MD
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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 45267
Principal Investigator: Joseph Palascak, MD
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Detroit, Michigan 48201
Principal Investigator: Meera Chitlur, MD
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East Lansing, Michigan 48824
Principal Investigator: Roshni Kulkarni, MD
Phone: 517-353-9385
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Fort Worth, Texas 76104
Principal Investigator: Timothy McCavit, MD
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Gainesville, Florida 32610
Principal Investigator: Tung Wynn, MD
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Houston, Texas 77030
Principal Investigator: Miguel Escobar, MD
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Hyde Park, New York 11040
Principal Investigator: Suchitra Acharya, MD
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8326 Naab Road
Indianapolis, Indiana 46260
Principal Investigator: Amy Shapiro, MD
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Kansas City, Kansas 64108
Principal Investigator: Shannon Carpenter, MD, MSc
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Las Vegas, Nevada 89178
Principal Investigator: Amber Federizo, NP
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Los Angeles, California 90007
Principal Investigator: Doris Quon, MD, PhD
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Memphis, Tennessee 38119
Principal Investigator: Sandeep Rajan, MD
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Milwaukee, Wisconsin 53233
Principal Investigator: Lynn Malec, MD
Phone: 414-257-2424
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New Orleans, Louisiana 70112
Principal Investigator: Tamuella Singleton, MD
Phone: 504-988-3596
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New York, New York 10065
Principal Investigator: Catherine McGuinn, MD
Phone: 212-746-3403
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201 Lyons Ave
Newark, New Jersey 07112
(973) 926-7000
Principal Investigator: Alice Cohen, MD
Newark Beth Israel Medical Center Newark Beth Israel Medical Center, a regional care, teaching hospital...
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Philadelphia, Pennsylvania 19104
Principal Investigator: Leslie Raffini, MD
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Philadelphia, Pennsylvania 19104
Principal Investigator: Adam Cuker, MD
Phone: 215-614-0506
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Phoenix, Arizona 85016
Principal Investigator: Christine Knoll, MD
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Pittsburgh, Pennsylvania 15213
Principal Investigator: Margaret Ragni, MD
Phone: 412-209-7288
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Sacramento, California 95817
Principal Investigator: Jonathan Ducore, MD, MPH
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Saint Louis, Missouri 63104
Principal Investigator: John Puetz, MD
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Scarborough, Maine 04074
Principal Investigator: Eric Larsen, MD
Phone: 207-396-7312
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921 Terry Avenue
Seattle, Washington 98104
Principal Investigator: Rebecca Kruse-Jarres, MD
Phone: 206-689-6539
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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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Winston-Salem, North Carolina 27157
Principal Investigator: Natalia Dixon, MD
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