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ATHN 2: Factor Switching Study

This study is currently recruiting participants.
Verified October 2017 by American Thrombosis and Hemostasis Network
Sponsor:
ClinicalTrials.gov Identifier:
NCT02546622
First Posted: September 11, 2015
Last Update Posted: October 12, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
Shire
CSL Behring
Bioverativ Therapeutics Inc.
Information provided by (Responsible Party):
American Thrombosis and Hemostasis Network
  Purpose

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 Eloctate will be approached to participate in the 'Eloctate 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.


Condition Intervention
Hemophilia Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013 Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Other
Official Title: A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Factor Replacement Products

Resource links provided by NLM:


Further study details as provided by American Thrombosis and Hemostasis Network:

Primary Outcome Measures:
  • Inhibitor Development [ Time Frame: After 50 Exposure Days or 1 Year, whichever comes first ]
    Inhibitor development is the primary outcome. Subjects will be followed closely and tested at baseline, after 10 Exposure Days and 50 Exposure Days, and/or at 1 year. Specimens will be submitted to local laboratories and evaluated for inhibitor titers, and inhibitors will be confirmed by local laboratories and CDC.


Secondary Outcome Measures:
  • Inhibitor Development [ Time Frame: 10 days ]
    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.

  • Prevalence of risk factors for inhibitor development [ Time Frame: 1 year ]
    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.

  • Targeted post-marketing approval safety and efficacy data [ Time Frame: 1 year ]

    To collect and summarize targeted post-marketing approval safety and efficacy data for events related to clotting factor replacement products, specifically

    • To collect data on bleeding events following switching factor.
    • To summarize replacement factor product dosing regimens prescribed to the study population.

  • Platform for additional substudies [ Time Frame: 1 year ]
    To serve as a platform for product-specific questionnaires in cohorts of patients who switch to a particular product.


Biospecimen Retention:   None Retained
Plasma drawn at baseline, after 10 Exposure Days, after 50 Exposure Days, and at study end (1 year). If inhibitor suspected, sample will be drawn and tested locally, and within 2 weeks of first positive inhibitor, a confirmatory sample will be drawn and sent to CDC. Patients may be co-enrolled in other ATHN studies such as My Life Our Future, will performs genotype Samples may be linked in patient who have authorized

Estimated Enrollment: 600
Study Start Date: September 2015
Estimated Study Completion Date: January 2022
Estimated Primary Completion Date: August 2021 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Arm A Prospective

Patients who are switching to a new Factor VIII and Factor IX Replacement Product for Hemophilia A and B which was FDA approved after January 1, 2013.

These patients will be followed prospectively for up to 1 year.

Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013
Other Names:
  • Eloctate®
  • NovoEight®
  • Adynovate®
  • Nuwiq®
  • Afstyla®
Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013
Other Names:
  • Alprolix®
  • Rixubis®
  • IXinity®
  • Idelvion®
Arm B Retrospective

Patients who have recently switched to a new Factor VIII and Factor IX Replacement Product for Hemophilia A and B which was FDA approved after January 1, 2013.

Patients must have switched products within the past 50 weeks at the time of enrollment.

These patients will be assessed retrospectively and/or followed prospectively for up to 1 year.

Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013
Other Names:
  • Eloctate®
  • NovoEight®
  • Adynovate®
  • Nuwiq®
  • Afstyla®
Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013
Other Names:
  • Alprolix®
  • Rixubis®
  • IXinity®
  • Idelvion®

  Show Detailed Description

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

The study will enroll approximately 600 patients with hemophilia who meet the eligibility criteria and are receiving care from one of the ATHN-affiliated Hemophilia Treatment Centers (HTC). There will be 2 arms:

Arm A (Prospective) will include patients who are switching factor replacement products and will be followed prospectively for up to 1 year.

Arm B (Retrospective) will include patients who have switched factor replacement products previously (within the past 50 weeks at the time of enrollment). These patients will be assessed retrospectively and/or followed prospectively for up to 1 year.

Criteria

Inclusion Criteria

  • 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.

Exclusion Criteria

  • 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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02546622


Contacts
Contact: Ellen G McCarthy, MPH 800 360 2846 ext 115 emccarthy@athn.org
Contact: Crystal Watson, MS 800 360 2846 ext 102 cwatson@athn.org

  Show 30 Study Locations
Sponsors and Collaborators
American Thrombosis and Hemostasis Network
Shire
CSL Behring
Bioverativ Therapeutics Inc.
Investigators
Study Chair: Ellis J Neufeld, MD, PhD St. Jude Children's Research Hospital
  More Information

Additional Information:
Publications:
Responsible Party: American Thrombosis and Hemostasis Network
ClinicalTrials.gov Identifier: NCT02546622     History of Changes
Other Study ID Numbers: ATHN 2
First Submitted: September 9, 2015
First Posted: September 11, 2015
Last Update Posted: October 12, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Product-Specific Module data for subjects enrolled in each module will be shared with each sponsor, as appropriate.

Keywords provided by American Thrombosis and Hemostasis Network:
Factor
Coagulation
Inhibitor
HTC
Bleeding

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants