Clinical Trial: A Phase 3 Study to Compare Efficacy and Safety of Masitinib to Placebo in the Treatment of Patients With Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis With Handicap
Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional
Official Title: A 24-week With Possible Extension, Prospective, Multicentre, Randomized, Double Blind, Placebo-controlled, 2-parallel Group With a Randomization 1:1, Phase III Study to Compare Efficacy and Safety of
Brief 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 Summary:
Mastocytosis is a disease characterized by mast cell invasion in various organs. Mast cells are bone marrow derived cells which produce histamine and other substances causing allergic and anaphylactic reactions. Accumulation of mast cells in body organs can inhibit the functionality of the organ and eventually cause degeneration.
Mutations of the mast cell growth factor receptor (KIT protein, the product of the c-Kit proto-oncogene) might be found in patients with mastocytosis.
Masitinib (AB1010) is a tyrosine kinase inhibitor (TKI), selectively and effectively inhibiting c-kit. Its effect may include inhibition of cell proliferation, inhibition of cell cycle progression and induction of apoptosis resulting in the reduction of mast cell accumulation in body tissues. This drug is thereby specific to mastocytosis and active in slowing or reducing the number of mast cells particularly in aggressive forms of the disease.
Sponsor: AB Science
Current Primary Outcome: Responder rate at week 24 [ Time Frame: 24 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Response in mastocytosis variables (pruritus, flushes, depression or asthenia), mictions and stools per day, patient score and assessments, patients without handicap, mast cell infiltration , serum tryptase level, safety profile [ Time Frame: 24 weeks ]
Original Secondary Outcome: Same as current
Information By: AB Science
Dates:
Date Received: December 22, 2008
Date Started: December 2008
Date Completion: June 2013
Last Updated: September 25, 2012
Last Verified: September 2012
