NCT00814073

Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 135 (actual)
• Sponsor: AB Science · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The objective of this study is to compare the safety and efficacy of masitinib (AB1010) to placebo in patients with mastocytosis with handicap.

Detailed description

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.

Conditions

• Indolent Systemic Mastocytosis

Interventions

Type	Name	Description
DRUG	Masitinib	Masitinib 6 mg/kg/day
DRUG	Placebo	Matching placebo
OTHER	Best Supportive Care	Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, proton pump inhibitors (PPI), sodium cromoglicate, antidepressants, leukotriene antagonists, interferon-alpha, 2-CdA, and corticosteroids.

Timeline

• Start date: 2008-12-01
• Primary completion: 2015-11-01
• Completion: 2015-11-01
• First posted: 2008-12-23
• Last updated: 2019-12-03

Locations

24 sites across 2 countries: United States, France

Regulatory

• FDA-regulated drug study