ATHN 8: Previously Untreated Patients (PUPs) Matter Study

NCT ID: NCT03818529

Last Updated: 2023-02-01

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 237 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2018-10-03
• Study Completion Date: 2022-12-31

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.

Detailed Description

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.

Conditions

Hemophilia; Hemophilia A; Hemophilia B; Hemophilia A With Inhibitor; Hemophilia B With Inhibitor; Haemophilia B Without Inhibitor; Haemophilia A Without Inhibitor; Haemophilia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: OTHER

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• Patients who are referred to the HTC with no record of bleed and factor utilization data

• Minimum Eligible Age: 0 Years
• Maximum Eligible Age: 13 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CSL Behring

INDUSTRY

Sponsor Role: collaborator

Octapharma

INDUSTRY

Sponsor Role: collaborator

Takeda

INDUSTRY

Sponsor Role: collaborator

American Thrombosis and Hemostasis Network

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Shannon Carpenter, MD, MS

Role: PRINCIPAL_INVESTIGATOR

Children's Mercy Hospital Kansas City

Courtney Thornburg, MD, MS

Role: PRINCIPAL_INVESTIGATOR

University of California San Diego, Rady Children's Hospital San Diego

Marijke van den Berg, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Versiti

Locations

Arizona Hemophilia and Thrombosis Center at Phoenix Children's Hospital

Phoenix, Arizona, United States

Valley Children's Hospital

Madera, California, United States

Rady Children's Hospital San Diego

San Diego, California, United States

UCSF Pediatric Hemophilia Treatment Center at Mission Bay

San Francisco, California, United States

University of Colorado Denver Hemophilia and Thrombosis Center

Aurora, Colorado, United States

Connecticut Bleeding and Clotting Disorders Center

Farmington, Connecticut, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Children's Healthcare of Atlanta/Emory

Atlanta, Georgia, United States

Augusta University Hemophilia Treatment Center

Augusta, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

Indiana Hemophilia and Thrombosis Center (IHTC)

Indianapolis, Indiana, United States

Kansas City Regional Hemophilia Center

Kansas City, Kansas, United States

Maine Hemophilia and Thrombosis Center

Scarborough, Maine, United States

Boston Hemophilia Center at Children's Hospital of Boston

Boston, Massachusetts, United States

University of Michigan Hemophilia and Coagulation Disorders

Ann Arbor, Michigan, United States

Mayo Comprehensive Hemophilia Center

Rochester, Minnesota, United States

Cincinnati Children's Hospital Medical Center, Hemophilia & Thrombosis Center

Cincinnati, Ohio, United States

UHHS Cleveland

Cleveland, Ohio, United States

Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital

Toledo, Ohio, United States

Oklahoma Center for Bleeding and Clotting Disorders

Oklahoma City, Oklahoma, United States

Oregon Health and Science University

Portland, Oregon, United States

St Jude Children's Research Hospital

Memphis, Tennessee, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Utah Center for Bleeding & Clotting Disorders at Primary Children's Hospital

Salt Lake City, Utah, United States

Hemophilia Outreach Center

Green Bay, Wisconsin, United States

Blood Center of Wisconsin

Milwaukee, Wisconsin, United States

Countries

United States

Other Identifiers

ATHN 8

Identifier Type: -

Identifier Source: org_study_id