by Janet Brewer, M.Ed ClinicalTrials.gov identifier (NCT number): NCT03619863
Published: Lifelines for Health Spring 2019
The American Thrombosis and Hemostasis Network has initiated the Natural History Study for the purpose of following patients with Hemophilia A/B with or without inhibitors for a period of 4-6 years who receive their care from hemophilia treatment center physicians across the United States. The primary purpose of the study is to assess the safety of FDA-approved treatment products and their outcomes to include: replacement clotting factor products, bypassing agents and non-factor products. Treatment regiments will be at the discretion of the patient and their hemophilia caregivers. There will be no treatment products or compensation offered; inhibitor testing however, will be provided by the CDC at no cost to patients.
Data collection 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. Patients will be 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.
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.
(https://clinicaltrials.gov/ct2/show/NCT03619863)
Secondary outcome measures will include:
• Effectiveness of non-factor products, bypassing agents and clotting factor replacement products based on the participant’s number of bleeding events and/or annualized bleed rate (ABR).
• Dosing regimens for hemophilia treatment products and total amount utilized for prophylaxis and treatment of bleeds.
• Target joint monitoring
Real world effectiveness of treatment products as measured by the number and types of medical visits and/or hospitalizations per year.
The study is currently open to 280 participants in 38 ATHN-affiliated HTCs. Patients will be seen at baseline, annually and at study exit.
Criteria:
No age restrictions
Open to men and women
Inclusion Criteria:
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 ATHN dataset
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
Contact:
Angela Riedel - Ph: 309.361.4375, ariedel@athn.org
Primary Investigators are:
Tyler Buckner, MD, MSc
Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical Campus
Nikki Hirsh, MS, CCRC - nhirsh@athn.org
Michael Recht, MD, PhD
The Hemophilia Center at Oregon Health & Science University
For a list of the 38 study locations please refer to:
Nikki Hirsh, MS, CCRC - nhirsh@athn.org
Michael Recht, MD, PhD
The Hemophilia Center at Oregon Health & Science University
https://clinicaltrials.gov/ct2/show/study/NCT03619863?show_locs=Y - locn
*EUHASS adverse events 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.