WithdrawnNot Applicable

Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive

Stopped: complete per PI

0Started 2009-09-25

Plain-language summary

This is a prospective, controlled, randomized, open label study, aimed at comparing FVIII/VWF concentrates with FVIII concentrates at 200 IU/kg daily in their ability to induce immune tolerance in Haemophilia A patients with high responding inhibitors and poor prognosis for success.

Who can participate

SexMALE

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Inclusion criteria

✓. severe hemophilia A (FVIII\<1%);
✓. male, any age;
✓. high responders (peak inhibitor levels \> 5 BU);
✓. any inhibitor level at study enrolment;
✓. ability and willingness to participate in the study;
✓. at least one of the following risk factors for ITI failure:
✓. absence of high risk of cardiovascular, cerebrovascular or other thromboembolic events as deemed by the treating clinician.

Exclusion criteria

✕. concomitant systemic treatment with immunosuppressive drugs;
✕. concomitant experimental treatment;
✕. previous ITI attempt;
✕. previous history of myocardial infarction and/or cerebral stroke.

What they're measuring

Primary end point is the success in inducing immune tolerance, defined as: the abolition of the inhibitor to < 0.6 BU within 33 months of ITI with a factor VIII recovery ≥ 66% and half-life ≥ 6 hrs, and measured after a 72-hour washout period.

Timeframe: 33 months

Trial details

NCT IDNCT01051544

SponsorCity of Hope Medical Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2020-06-25

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