ATHN 7: Hemophilia Natural History Study

NCT ID: NCT03619863

Last Updated: 2025-02-12

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 395 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2018-10-24
• Study Completion Date: 2024-03-31

Brief Summary

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.

Detailed Description

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.

Conditions

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

Study Design

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

Eligibility Criteria

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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genentech, Inc.

INDUSTRY

Sponsor Role: collaborator

American Thrombosis and Hemostasis Network

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Tyler Buckner, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical Campus

Michael Recht, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

The Hemophilia Center at Oregon Health & Science University

Locations

Arizona Hemophilia and Thrombosis Center at Phoenix Children's Hospital

Phoenix, Arizona, United States

Orthopaedic Institute for Children HTC

Los Angeles, California, United States

Orthopedic Institute for Children Hemophilia Program

Los Angeles, California, United States

Rady Children's Hospital San Diego

San Diego, California, United States

Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical Campus

Aurora, Colorado, United States

University of Florida Hemophilia Treatment Center

Gainesville, Florida, United States

Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

Indiana Hemophilia and Thrombosis Center (IHTC)

Indianapolis, Indiana, United States

Louisiana Center for Bleeding and Clotting Disorders, Tulane University

New Orleans, Louisiana, United States

Louisiana Center for Bleeding and Clotting Disorders

New Orleans, Louisiana, United States

Maine Hemophilia and Thrombosis Center

Scarborough, Maine, United States

Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center

Boston, Massachusetts, United States

Michigan State University Center for Bleeding and Clotting Disorders

East Lansing, Michigan, United States

Children's Mercy Hospital - Kansas City

Kansas City, Missouri, United States

The John Bouhasin Center for Children with Bleeding Disorders

St Louis, Missouri, United States

Hemostasis and Thrombosis Center of Nevada

Las Vegas, Nevada, United States

Hemostasis and Thrombosis Center of Nevada

Reno, Nevada, United States

Newark Beth Israel Medical Center

Newark, New Jersey, United States

Weill Cornell Medicine

New York, New York, United States

Wake Forest University Health Science

Winston-Salem, North Carolina, United States

Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital

Toledo, Ohio, United States

The Hemophilia Center at Oregon Health & Science University

Portland, Oregon, United States

Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, United States

Penn Comprehensive Hemophilia and Thrombophilia Program/Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

St Jude Children's Research Hospital

Memphis, Tennessee, United States

The Center for Cancer and Blood Disorders, Children's Medical Center of Dallas

Dallas, Texas, United States

Gulf States Hemophilia and Thrombosis Center

Houston, Texas, United States

Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin, United States

Countries

United States

References

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Reference Type: BACKGROUND

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Gouw SC, van den Berg HM, Fischer K, Auerswald G, Carcao M, Chalmers E, Chambost H, Kurnik K, Liesner R, Petrini P, Platokouki H, Altisent C, Oldenburg J, Nolan B, Garrido RP, Mancuso ME, Rafowicz A, Williams M, Clausen N, Middelburg RA, Ljung R, van der Bom JG; PedNet and Research of Determinants of INhibitor development (RODIN) Study Group. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013 May 16;121(20):4046-55. doi: 10.1182/blood-2012-09-457036. Epub 2013 Apr 3.

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Carr ME, Tortella BJ. Emerging and future therapies for hemophilia. J Blood Med. 2015 Sep 3;6:245-55. doi: 10.2147/JBM.S42669. eCollection 2015.

Reference Type: BACKGROUND

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Sehgal A, Barros S, Ivanciu L, Cooley B, Qin J, Racie T, Hettinger J, Carioto M, Jiang Y, Brodsky J, Prabhala H, Zhang X, Attarwala H, Hutabarat R, Foster D, Milstein S, Charisse K, Kuchimanchi S, Maier MA, Nechev L, Kandasamy P, Kel'in AV, Nair JK, Rajeev KG, Manoharan M, Meyers R, Sorensen B, Simon AR, Dargaud Y, Negrier C, Camire RM, Akinc A. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat Med. 2015 May;21(5):492-7. doi: 10.1038/nm.3847. Epub 2015 Apr 13.

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Other Identifiers

ATHN 7

Identifier Type: -

Identifier Source: org_study_id