What this study is
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.
What's being tested
- Nuwiq
Intervention · BIOLOGICAL
Nuwiq is a recombinant FVIII concentrate from a human cell line. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
- Octanate
Intervention · BIOLOGICAL
Octanate is a high-purity human Factor VIII / von Willebrand Factor (VWF) concentrate. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
- Wilate
Intervention · BIOLOGICAL
Wilate is a high-purity human von Willebrand Factor (VWF)/Factor VIII concentrate. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
- Emicizumab
Intervention · BIOLOGICAL
Emicizumab is a therapeutic antibody which brings activated factor IX and factor X together It is administered via subcutaneous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
- Recombinant factor VIIa (rFVIIa)
Intervention · BIOLOGICAL
Recombinant factor VIIa (rFVIIa) is a blood factor VII manufactured using recombinant technology. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
- Activated prothrombin complex concentrate (aPCC)
Intervention · BIOLOGICAL
Activated prothrombin complex concentrate (aPCC) is an anti-inhibitor coagulant complex acting on multiple pathways to facilitate coagulation. It is administered via intravenous injection at a dosage determined by the investigator's discretion, in consideration of the participants' clinical condition and prescribing information.
Study details
- Official title
- MOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study
- Sponsor
- Emory University
- Conditions
- Hemophilia A
- Enrolling
- 120 people
- Starts
- 2020-03-17
- Est. completion
- 2029-06
- Record
- View on ClinicalTrials.gov ↗
Who can join
Sex MALEHealthy volunteers Not acceptedYou may qualify if
- 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
You can't join if
- 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
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (2)
2 openArthur M. Blank Hospital
Atlanta, Georgia, United States
- Robert Sidonio, MD, MSc · Contact404-785-1637robert.sidonio.jr@emory.edu
- Robert Sidonio, MD, MSc · Principal investigator
Recruiting
1 site outside the US
HZRM Hämophilie-Zentrum Rhein Main
Mörfelden-Walldorf, Germany
- Carmen Escuriola-Ettingshausen, MD · Contact+4961059638909carmen.escuriola@hzrm.de
- Carmen Escuriola-Ettingshausen, MD · Principal investigator
Recruiting
Study team
Sponsor
Emory University
Study ID IRB00113316
Principal investigator
Robert Sidonio, MD, MSc
Emory University
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.