Indolent Systemic Mastocytosis Clinical Trial
Official title:
Randomized, Placebo-controlled, Phase 3 Study to Compare Efficacy and Safety of Masitinib at 6 mg/kg/Day to Placebo in Treatment of Patients With Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis With Handicap
The objective of this study is to compare the safety and efficacy of masitinib (AB1010) to placebo in patients with mastocytosis with handicap.
This was a prospective, multicenter, randomized, placebo-controlled, parallel-group, phase 3
study, conducted in 15 countries, evaluating the efficacy and safety of masitinib (6
mg/kg/day administered orally in two daily intakes over 24-weeks with a double-blind
extension period possible) for the treatment of indolent systemic mastocytosis, smoldering
mastocytosis or cutaneous mastocytosis, in patients with mast cells mediator release symptoms
that are refractory to conventional symptomatic treatment.
A study protocol amendment restricted enrolment to patients with severe indolent and
smoldering systemic mastocytosis. The objective of this phase 3 study was therefore to
evaluate masitinib efficacy and safety in severe systemic mastocytosis patients, with or
without D816V mutation of c-Kit. The primary objective of the phase 3 study was to detect a
statistically significant difference between masitinib (plus optimal concomitant symptomatic
treatments) and placebo (plus optimal concomitant symptomatic treatments) in cumulative
response on four severe symptoms, referred to also as handicaps.
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