Observing treatment approaches for Hemophilia A patients with inhibitors
MOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study
Emory University · NCT04023019
This study looks at different treatment methods for men with Hemophilia A who have developed inhibitors to see how well they work and how safe they are over five years.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 120 (estimated) |
| Sex | Male |
| Sponsor | Emory University (other) |
| Drugs / interventions | emicizumab |
| Locations | 2 sites (Atlanta, Georgia and 1 other locations) |
| Trial ID | NCT04023019 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on male patients with Hemophilia A who have developed inhibitors to factor VIII. It aims to document various management strategies, including immune tolerance induction and the use of bypassing agents. Participants will be categorized into treatment groups based on their current therapies and may switch groups if their treatment changes. The study will follow participants for up to five years to evaluate the efficacy and safety of these approaches.
Who should consider this trial
Good fit: Ideal candidates include male patients with Hemophilia A who have a historical inhibitor titer of 0.6 BU/mL or higher.
Not a fit: Patients with other coagulation disorders or those lacking detailed treatment documentation will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could improve treatment strategies for Hemophilia A patients with inhibitors, potentially enhancing their quality of life.
How similar studies have performed: Previous studies on immune tolerance induction have shown promising results, indicating that this approach is well-established in the field.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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) * 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) * Participants or participants' parent(s)/legal guardian(s) must be capable of giving signed informed consent and be able to understand the trial documents Exclusion Criteria: * Participants are excluded from the trial if any coagulation disorder other than haemophilia A is diagnosed * 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
Where this trial is running
Atlanta, Georgia and 1 other locations
- Arthur M. Blank Hospital — Atlanta, Georgia, United States (RECRUITING)
- HZRM Hämophilie-Zentrum Rhein Main — Mörfelden-Walldorf, Germany (RECRUITING)
Study contacts
- Principal investigator: Robert Sidonio, MD, MSc — Emory University
- Study coordinator: Robert Sidonio, MD, MSc
- Email: robert.sidonio.jr@emory.edu
- Phone: 404-785-1637
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Hemophilia A, Blood disorders