A Phase II, single-blind, randomised, placebo-controlled trial to study the efficacy and safety of anti-von Willebrand factor Nanobody administered as adjunctive treatment to patients with acquired thrombotic thrombocytopenic purpura
This is a Phase ii multicentre, single-blinded, parallel design, randomised, placebo-controlled
after confirmation of eligibility to study participation, subjects will be randomised in a ratio of
1:1 to either receive aLX-0081 or placebo as adjunctive therapy to Pe. Subjects will be randomised prior to the start of Pe treatment . in exceptional cases however (due to need or ability to start Pe in a time frame which does not allow all required screening and/or baseline study procedures to be performed), a subject may be randomised after the first, single Pe session, but prior to the start of the second Pe session. This overall second Pe session should be started within 24 hours of the end of the very first Pe session, and will be considered the first Pe on study.
Subjects will receive a first intravenous (i.v.) bolus of 10 mg aLX-0081 or placebo via push injection within 6h, but not later than 15 minutes prior to the initiation of Pe on study (i.e., the very first Pe session, if the subject was randomised prior to the initiation of Pe, or the second Pe session, if the subject was randomised after one, single Pe session). This first Pe on study is followed by subcutaneous (s.c.) administration of 10 mg study drug.
Subsequently daily s.c. administrations of 10 mg aLX-0081 (Table 1) or placebo will follow each Pe session for the duration of Pe (including tapering and Pe given for exacerbations) and once daily for 30 days following the very last Pe (including tapering) (Table 1). The maximum total daily dose of study drug is 10 mg when administered in conjunction with Pe (20 mg only in case twice daily Pe sessions are needed) and 10 mg when in the period following the very last Pe (including tapering). Study drug administration will continue in case of re-initiation of Pe for an exacerbation of TTP, with a maximum total treatment duration limited to 90 days after first administration of study drug.
at [LessThanorequalTo] 30 days after the last day of study drug administration, subjects will be assessed for the primary and secondary endpoints of the study, and will be followed for a maximum of 1 year for relapses and other tertiary (longer-term) endpoints.
1. 18 years of age or older
2. Men or women willing to accept an acceptable contraceptive regimen
3. Patients with clinical diagnosis of TTP
4. Necessitating PE (one, single PE session prior to randomisation into the study is allowed)
5. Subject accessible to follow-up
6. Obtained, signed and dated informed consent