ATHN 2 – A Longitudinal, Observational Study of Previously Treated Hemophilia Patients (PTPs) Switching Factor Replacement Products
With nearly a dozen new factor replacement products available, many patients are likely to switch to a new product within the next few years. To better understand the effects of switching to any of these new factor products, ATHN is sponsoring ATHN 2, a new research study on the effects of switching factor products.
Unlike manufacturer-sponsored studies of individual factor products, ATHN 2 is designed to study all of the new factor products over many years, after they have been FDA approved. Patients who have a prior history of inhibitors to factors (but are free of an inhibitor now) are eligible to participate. All data collected will be entered into electronic case report forms (eCRFs) within the secure ATHN Study Manager system.
The multidisciplinary, multiregional ATHN 2 Steering Committee, including leading researchers and nurses from 9 ATHN-affiliated sites and a patient representative, guides the direction of this longitudinal, observational study.
Principal Investigator is Ellis Neufeld, MD, PhD, Boston Children’s Hospital
ATHN is the sponsor with product specific modules funded by specific industry partners (i.e., Biogen and Baxalta as of February 2016). The ATHN 2 Steering Committee is made up of representatives from 9 ATHN-affiliated sites and a patient, Ellis Neufeld, MD, PhD, Boston Children’s Hospital (Chair); Janna Journeycake, MD (supervising PI for product specific sub-studies)
Study Design: Longitudinal, observational study of multiple cohorts (one for each factor replacement product)
The primary objective is to assess inhibitor development and strength based on Bethesda Units (BU) within one (1) year or fifty (50) exposure days, whichever is first, after switching factor replacement products in previously treated patients with hemophilia A or B (PTPs).
- To determine the prospective incidence of inhibitor development after 10 exposure days to a new, novel recombinant factor following a switch from another clotting factor replacement product.
- To compare the prevalence of selected risk factors in patients with hemophilia who develop inhibitors following switching to a new product, to those found in subjects who do not develop inhibitors. In particular, the risk factor “history of prior inhibitors” will be tracked as a sub-cohort of the ATHN 2 population.
- To collect and summarize targeted post- approval safety and efficacy data for events related to clotting factor replacement products.
- To collect data on bleeding events following switching factor.
- To summarize factor replacement dosing regimens prescribed to the study population.
- To serve as a platform for product-specific substudies in cohorts of patients who switch or have switched to a particular factor replacement product.
If you are interested in participating in ATHN 2, contact your HTC staff. To learn more, download the ATHN 2 brochure.
ATHN-affiliated investigators interested in getting involved in ATHN 2 should contact email@example.com.