CompletedPhase 4

Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies

United States, Canada, Germany16 participantsStarted 2017-08-29

Plain-language summary

The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at tolerization.

Who can participate

SexMALE

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Inclusion criteria

✓. Signed and dated informed consent provided by the patient, or the patient's legally authorized representative for patients under the legal age. Assent should be obtained from pediatric patients according to local regulations
✓. Male patients of any age diagnosed with severe haemophilia A, as confirmed from the medical record
✓. Previously treated with any plasma-derived or recombinant conventional or extended half-life FVIII
✓. Diagnosed with high titer inhibitors (historical peak ≥5 Bethesda units (BU)/mL according to medical records)
✓. Inhibitor titer \>0.6 BU at screening
✓. Failed previous ITI attempt(s) with any plasma-derived or recombinant conventional or extended half-life FVIII including the use of immunosuppressant The attempt should be documented in the medical records and have the following characteristics:
✓. All patients must practice effective contraception during the study and for 3 months after their last dose of study treatment

Exclusion criteria

✕. Other coagulation disorder(s) in addition to haemophilia A
✕. History of hypersensitivity reactions associated with any rFVIIIFc administration
✕. High risk of cardiovascular, cerebrovascular, or other thromboembolic events, as judged by the investigator
✕. Planned major surgery to be deferred after study completion. Minor surgery such as tooth extraction or insertion/replacement of central venous access device is allowed.
✕. Concurrent systemic treatment with immunosuppressive drugs within 12 weeks prior to screening. Exceptions to this include: ribavirin for treatment of Hepatitis C virus (HCV), and/or systemic steroids (a total of 2 courses of pulse treatments lasting no more than 7 days within 12 weeks prior to Day 1) and/or inhaled steroids
✕. Abnormal renal function (serum creatinine \>2.0 mg/dL) as assessed by local lab
✕. Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>5 × upper limit of normal (ULN) as assessed by local lab
✕. Serum total bilirubin \>3 × ULN as assessed by local lab

What they're measuring

ITI Success

Timeframe: up to 60 weeks

Trial details

NCT IDNCT03103542

SponsorSwedish Orphan Biovitrum

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2019-09-04

Results submitted2021-08-03

View on ClinicalTrials.gov More Hemophilia A trials