RECRUITING

Treatment of Hemophilia A Patients With FVIII Inhibitors

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a non-interventional, multicenter, observational, international study in male persons with haemophilia A who have developed inhibitors to any replacement coagulation factor VIII (FVIII) product. The purpose of the study is to capture different approaches in the management of persons with haemophilia A and FVIII inhibitors, document current immune tolerance induction approaches, and evaluate the efficacy and safety of immune tolerance induction, including the combination of FVIII and emicizumab. Patients will be assigned to 1 of 3 groups based on the treatments they receive, and may switch to another group if their treatment is changed. Participants will be followed after a maximum observational period of 5 years.

Official Title

MOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study

Quick Facts

Study Start:2020-03-17
Study Completion:2029-06
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04023019

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male persons with haemophilia A, of any severity, who have a historical inhibitor titer ≥ 0.6 BU/mL, including those who have failed previous immune tolerance induction (ITI) attempt(s)
  2. * Persons undergoing ITI with Nuwiq, octanate, or wilateor undergoing ITI with Nuwiq®, octanate® or wilate® and receiving prophylactic therapy with emicizumab, activated prothrombin complex concentrate (aPCC), or activated recombinant factor VII (rFVIIa)
  3. * Participants or participants' parent(s)/legal guardian(s) must be capable of giving signed informed consent and be able to understand the trial documents
  1. * Participants are excluded from the trial if any coagulation disorder other than haemophilia A is diagnosed
  2. * Partly retrospective patients will be excluded if detailed documentation on treatment, all bleeding episodes, inhibitor titers, and FVIII levels is not available for the retrospective period

Contacts and Locations

Study Contact

Robert Sidonio, MD, MSc
CONTACT
404-785-1637
robert.sidonio.jr@emory.edu
Carmen Escuriola-Ettingshausen, MD
CONTACT
+4961059638909
carmen.escuriola@hzrm.de

Principal Investigator

Robert Sidonio, MD, MSc
PRINCIPAL_INVESTIGATOR
Emory University

Study Locations (Sites)

Arthur M. Blank Hospital
Atlanta, Georgia, 30329
United States

Collaborators and Investigators

Sponsor: Emory University

  • Robert Sidonio, MD, MSc, PRINCIPAL_INVESTIGATOR, Emory University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2020-03-17
Study Completion Date2029-06

Study Record Updates

Study Start Date2020-03-17
Study Completion Date2029-06

Terms related to this study

Keywords Provided by Researchers

  • Blood disorders

Additional Relevant MeSH Terms

  • Hemophilia A