ATHN 7: Hemophilia Natural History Study

NCT03619863 | Completed

• 1 other identifier: ATHN 7
• Study Type: observational
• Target: 395
• Locations: 1 country
• Sites: 29

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.

Conditions

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

Keywords

Hemophilia; Non-Factor Products; Bypassing Agents; Clotting Factor Replacement Products

Outcome Measures

Primary Outcomes (1)

• Safety of treatment products used for hemophilia care will be assessed based on the number of reportable European Haemophilia Safety Surveillance (EUHASS) events documented on the ATHN Adverse Event Module Form.

  All treatment-related reportable adverse events will be documented on the ATHN Adverse Event Module Form based on EUHASS reportable events which include: death, factor inhibitor development, venous thrombosis, allergic reactions, treatment-emergent side effects, new malignancies, cardiovascular events, and blood-borne infections. Other treatment-related events to be documented on the ATHN Adverse Event Module Form including thrombotic microangiopathies, injection site reactions, drug-induced liver injury and anti-drug antibodies.

  6 years

Secondary Outcomes (5)

• Effectiveness of non-factor products, bypassing agents and clotting factor replacement products will be evaluated based on the participant's number of bleeding events reported as the annualized bleeding rate (ABR).

  6 years

• Dosing regimens for hemophilia treatment products and total amount utilized by the study participant for prophylaxis and treatment of bleeds will be assessed.

  6 years

• Target joint monitoring

  6 years

• Efficacy of treatment is rated by health-related outcomes tools: EQ-5D-5L, Patient-Reported Outcomes Measurement Information System (PROMIS), Global Adherence Rating (GAR), and Treatment Satisfaction with Medicines Questionnaire (SATMED-Q).

  6 years

• Real world effectiveness of treatment products assessed by the healthcare providers as measure by the number and types of medical visits and/or hospitalizations per year.

  6 years

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study will enroll approximately 280 patients with hemophilia who meet the eligibility criteria and receiving care from one of the ATHN-affiliated Hemophilia Treatment Centers (HTC).

You may qualify if:

• 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;
• Able to give informed consent (by patient or parent/authorized guardian); and
• Co-enrollment in the ATHNdataset.

You may not qualify if:

• Presence of any known bleeding disorder other than congenital hemophilia A or B;
• Presence of concurrent hemophilia and a second hemostatic defect (low Von Willebrand Factor (VWF) without Von Willebrand disease (VWD) diagnosis is not excluded); and
• Unable or unwilling to comply with the study protocol.

Sponsors & Collaborators

• American Thrombosis and Hemostasis Network: lead
• Genentech, Inc.: collaborator

Study Sites (29)

Arizona Hemophilia and Thrombosis Center at Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Orthopaedic Institute for Children HTC

Los Angeles, California, 90007, United States

Location

Orthopedic Institute for Children Hemophilia Program

Los Angeles, California, 90007, United States

Location

Rady Children's Hospital San Diego

San Diego, California, 92123, United States

Location

Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

University of Florida Hemophilia Treatment Center

Gainesville, Florida, 32610, United States

Location

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

Atlanta, Georgia, 30308, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Indiana Hemophilia and Thrombosis Center (IHTC)

Indianapolis, Indiana, 46260, United States

Location

Louisiana Center for Bleeding and Clotting Disorders, Tulane University

New Orleans, Louisiana, 70112, United States

Location

Louisiana Center for Bleeding and Clotting Disorders

New Orleans, Louisiana, 70112, United States

Location

Maine Hemophilia and Thrombosis Center

Scarborough, Maine, 04074, United States

Location

Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center

Boston, Massachusetts, 02114, United States

Location

Michigan State University Center for Bleeding and Clotting Disorders

East Lansing, Michigan, 48823, United States

Location

Children's Mercy Hospital - Kansas City

Kansas City, Missouri, 64108, United States

Location

The John Bouhasin Center for Children with Bleeding Disorders

St Louis, Missouri, 63104, United States

Location

Hemostasis and Thrombosis Center of Nevada

Las Vegas, Nevada, 89178, United States

Location

Hemostasis and Thrombosis Center of Nevada

Reno, Nevada, 89509, United States

Location

Newark Beth Israel Medical Center

Newark, New Jersey, 07122, United States

Location

Weill Cornell Medicine

New York, New York, 10065, United States

Location

Wake Forest University Health Science

Winston-Salem, North Carolina, 27157, United States

Location

Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital

Toledo, Ohio, 43606, United States

Location

The Hemophilia Center at Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States

Location

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

Philadelphia, Pennsylvania, 19104, United States

Location

St Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

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

Dallas, Texas, 75235, United States

Location

Gulf States Hemophilia and Thrombosis Center

Houston, Texas, 77030, United States

Location

Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (7)

• Ragni MV. Targeting Antithrombin to Treat Hemophilia. N Engl J Med. 2015 Jul 23;373(4):389-91. doi: 10.1056/NEJMcibr1505657. No abstract available.

  PMID: 26200986 BACKGROUND

• 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.

  PMID: 23553768 BACKGROUND

• Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016 May 26;374(21):2044-53. doi: 10.1056/NEJMoa1511769.

  PMID: 27223146 BACKGROUND

• Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. doi: 10.1056/NEJMoa1703068. Epub 2017 Jul 10.

  PMID: 28691557 BACKGROUND

• 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.

  PMID: 26366108 BACKGROUND

• 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.

  PMID: 25849132 BACKGROUND

• Buckner TW, Carpenter SL, Daoud N, Kempton CL, Lee L, Malec L, McLean TW, Morton P, O'Neill C, Staber JM, Wang M, Croteau SE, Recht M. Safety and Effectiveness of Emicizumab in People With Haemophilia A Enrolled in the ATHN 7 Haemophilia Natural History Study. Haemophilia. 2025 Nov 11. doi: 10.1111/hae.70151. Online ahead of print.

  PMID: 41220279 DERIVED

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Tyler Buckner, MD, MSc

  Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical Campus

  PRINCIPAL INVESTIGATOR

• Michael Recht, MD, PhD

  The Hemophilia Center at Oregon Health & Science University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 4 Years
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 25, 2018
• First Posted: August 8, 2018
• Study Start: October 24, 2018
• Primary Completion: May 31, 2023
• Study Completion: March 31, 2024
• Last Updated: February 12, 2025

Record last verified: 2025-02

Locations

US (29)