ATHN 2: Factor Switching Study

Completed

Conditions studied: Hemophilia

In brief

This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products. The study will follow each patient for up to 1 year. Patients will be recruited at Hemophilia Treatment Centers (HTC) which are ATHN-affiliates. The primary outcome being studied is the development of inhibitor (i.e., antibodies to factor) at 1 year or 50 exposure days, whichever comes first. The study will be conducted at approximately 30 HTCs, with a planned enrollment of 600 patients.The entire study duration is projected to be approximately 6 years. In addition, optional substudies will be included for some products, as "Product-Specific Modules". These will be questionnaires to collect data for subjects receiving selected Factor products. For example, subjects receiving Kovaltry will be approached to participate in the 'Kovaltry Product-Specific Module'; subjects receiving Adynovate will be approached to participate in the 'Adynovate Product-Specific Module'. Questions will be related to product use, perceptions of product use, and other post-marketing consumer data.

Key facts

Study ID

NCT02546622

Run by

American Thrombosis and Hemostasis Network

People needed

310

Starts

2015-09-01

Expected to finish

2020-06-30

Last updated by the study team

2021-12-09

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Moderate or Severe Congenital Hemophilia A or B (FVIII or FIX clotting activity less than or equal to 5% of normal).
• Able to give informed consent (by patient or parent/authorized guardian).
• Previously treated with plasma-derived or recombinant clotting factor replacement products with at least 50 exposure days (as assessed either from direct clinical records in children under age 5, or by clinical history of dosing in older patients). For Arm B being enrolled retrospectively, this previous treatment must be prior to product switch under study.
• Planning to switch, or recently switched within the previous 50 weeks, to a new brand or type of replacement factor VIII or IX, FDA approved after January 1, 2013.
• Arm B only: Negative inhibitor screen within the last 6 months prior to switching.
• Note: History of prior transient inhibitor or inhibitor eradicated by immune tolerance induction (ITI) are eligible.

You may not qualify if…

• Presence of any known bleeding disorder other than hemophilia A or B (i.e., patients with concurrent hemophilia and a second hemostatic defect are NOT eligible). Low Von Willebrand Factor (VWF) without VWF diagnosis are not excluded.
• Presence of an active inhibitor >0.6 BU for factor VIII, > 0.4 BU for factor IX at the time of eligibility assessment. Detection of such an inhibitor at the baseline visit prior to dosing with the new product (Arm A), or after dosing with new factor dosing (Arm B), would result in early termination without other study assessments.
• Currently undergoing ITI.
• Immunosuppressive therapy (cyclophosphamide, mycophenolate, IVIG) within 90 days and Rituximab within 6 months; topical steroid treatments and short course steroids for asthma or allergy allowed.
• Previous participation in Phase I, II or III interventional trials of the factor product being switched to.

Where it is running

• UTSW Medical Center at Dallas/Children's Medical Center — Dallas, Texas, United States
• Washington Center for Bleeding Disorders Bloodworks Northwest d/b/a Puget Sound Blood Center — Seattle, Washington, United States
• Blood Center of Wisconsin — Milwaukee, Wisconsin, United States
• University of California San Diego (UCSD) — San Diego, California, United States
• University of Colorado Denver Hemophilia and Thrombosis Center — Aurora, Colorado, United States
• Yale Hemophilia Treatment Center — New Haven, 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
• St. Joseph's Children's Hospital — Tampa, Florida, United States
• Children's Healthcare of Atlanta/Emory — Atlanta, Georgia, United States
• Bleeding and Clotting Disorders Institute — Peoria, Illinois, United States
• Indiana Hemophilia and Thrombosis Center (IHTC) — Indianapolis, Indiana, United States
• Louisiana Center for Bleeding and Clotting Disorders — New Orleans, Louisiana, United States
• Maine Hemophilia and Thrombosis Center — Scarborough, Maine, United States
• Johns Hopkins University Medical Center — Baltimore, Maryland, United States
• Boston Hemophilia Center at Children's Hospital of Boston — Boston, Massachusetts, United States
• University of Michigan Hemophilia and Coagulation Disorders Program — Ann Arbor, Michigan, United States
• Michigan State University Center for Bleeding and Clotting Disorders — East Lansing, Michigan, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• Dartmouth-Hitchcock Comprehensive Hemophilia and Thrombosis Center — Lebanon, New Hampshire, United States
• Weill Cornell Medicine — New York, New York, United States
• Mary M. Gooley Hemophilia Center — Rochester, New York, United States
• Oregon Health and Science University — Portland, Oregon, United States
• Children's Hospital of Philadelphia (CHOP) — Philadelphia, Pennsylvania, United States
• Pennsylvania Comprehensive Hemophilia and Thrombophilia Program / Hospital of the University of Pennsylvania — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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