Evaluation of Pazopanib on Bleeding in Subjects With Hereditary Haemorrhagic Telangiectasia
Status:
Terminated
Trial end date:
2016-02-10
Target enrollment:
Participant gender:
Summary
This study will investigate whether pazopanib can reduce epistaxis and improve anaemia in
subjects with hereditary haemorrhagic telangiectasia (HHT) at a dose that is well tolerated.
The study will have 2 parts. Part A will be an open label, dose-escalation study in which up
to 4 cohorts of approximately 6 subjects each will receive increasing doses of pazopanib for
a maximum of 12 weeks. The dose in the first cohort will be 50mg per day and the maximum dose
in a cohort will be 400 mg per day. Dose escalation will not occur as planned if the
predefined safety stopping criteria are met or at least 4 subjects in a cohort have
demonstrated efficacy (as measured by epistaxis, haemoglobin, transfusion or iron infusion
requirements). If efficacy is demonstrated in Part A with an acceptable safety profile, Part
B will be initiated to further define the optimal dose(s) including dose duration/schedule
and to provide further support for the proof of mechanism. Approximately 15 subjects will
participate and will be randomised to active or placebo in a ratio of 3:2. This part of the
study will be double-blind.