RecruitingNot Applicable

Treatment of Hemophilia A Patients With FVIII Inhibitors

United States, Germany120 participantsStarted 2020-03-17

Plain-language summary

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.

Who can participate

SexMALE

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Proportion of participants achieving inhibitor titer < 0.6 Bethesda units (BU)/mL L for at least 2 consecutive measurements

Timeframe: Up to 5 years

Proportion of participants achieving FVIII recovery ≥ 66% of the predefined reference value of 1.5% IU/kg body weight (Groups 1 and 2)

Timeframe: Up to 5 years

Proportion of participants achieving FVIII half-life ≥ 6 h (Groups 1 and 2)

Timeframe: Up to 5 years

Annualized bleeding rate

Timeframe: Up to 5 years

Trial details

NCT IDNCT04023019

SponsorEmory University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2028-12

Contact for this trial

Robert Sidonio, MD, MSc

404-785-1637 robert.sidonio.jr@emory.edu

View on ClinicalTrials.gov More Hemophilia A trials

Plain-language summary

Who can participate

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Proportion of participants achieving inhibitor titer < 0.6 Bethesda units (BU)/mL L for at least 2 consecutive measurements

Timeframe: Up to 5 years

Proportion of participants achieving FVIII recovery ≥ 66% of the predefined reference value of 1.5% IU/kg body weight (Groups 1 and 2)

Timeframe: Up to 5 years

Proportion of participants achieving FVIII half-life ≥ 6 h (Groups 1 and 2)

Timeframe: Up to 5 years

Annualized bleeding rate

Timeframe: Up to 5 years