NCT04205175Active, not recruitingPhase 4
A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab
A Single-center, Open-Label, Dose Escalation Study Evaluating the Safety of in Vivo Administration of FEIBA in Congenital Hemophilia A Patients With Inhibitors on Emicizumab
Source: ClinicalTrials.gov · Last updated 9 Mar 2022
Brief summary (registry, in English)
Hemophilia A is a severe, life-long, genetic bleeding disorder characterized by a deficiency of factor VIII (FVIII), a crucial cofactor of the coagulation system. The mainstay of hemophilia treatment is factor replacement therapy with FVIII clotting factor concentrates (CFC) and these can be given episodically in response to bleeding or prophylactically to prevent bleeding. The main adverse effect of FVIII CFC is the development of neutralizing anti-drug antibodies termed inhibitors, and these render replacement therapy less effective if they are low titer inhibitors or completely ineffective if they are of the high titer variety. These so-called 'inhibitor patients' cannot rely on FVIII CFC for their treatment and are treated with other CFC called bypassing agents such as activated prothrombin complex concentrate (aPCC/Feiba). While these agents can be effective in some patients for prophylaxis, they are not as effective for bleed prevention as FVIII CFC for patients without inhibitors.Recently, emicizumab (Hemlibra, Roche), was developed and licensed for the prevention of bleeding in patients with hemophilia A with and without inhibitors. However, patients in the clinical trials for emicizumab have developed thrombotic adverse events and only patients who received doses of Feiba of \>100 IU/kg/24 hours for more than 24 hours developed thrombosis. As a result of the above data, recommendations have been to either avoid altogether in patients on emicizumab, or to be very cautious about using it to treat breakthrough bleeding. With this in mind, we propose to study the in vivo combination of Feiba in patients with inhibitors on emicizumab.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 20 (estimated)
- First submitted
- 5 Dec 2019
- Start date
- 1 Jul 2020
- Primary completion date
- 31 Oct 2022
- Lead sponsor
- Children's Hospital Los Angeles
- Diseases
- Haemophilia
- NCT number
- NCT04205175
What you commit to
- Study type: Interventional · Phase 4
- Planned participants: 20
- From 1 Jul 2020 to the primary completion date, 31 Oct 2022
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Active bleed requiring factor therapy at screening.
- Treatment with rFVIIa or aPCC 7 days prior to screening.
- Surgical procedure 14 days prior to screening.
- Current use of any medication other than emicizumab that could affect the coagulation system e.g. aspirin, anticoagulants, etc.
Who the study is looking for
- Male patients with congenital hemophilia A of any severity and any age.
- History of high titer factor VIII inhibitor (Bethesda unit \>5)
- Currently prescribed bypassing agent therapy for bleed management.
- Current treatment with emicizumab for a minimum of 2 months without interruption.
Sex: Male · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Childrens Hospital Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.