Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment

Sponsor
AB Science
Study ID
NCT04333108
Phase
PHASE3
Status
Unknown

Conditions

  • Indolent Systemic Mastocytosis

Eligibility Criteria

Sex
ALL
Age
18 Years - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • Masitinib — DRUG
    Masitinib 6 mg/kg/day
  • Placebo — OTHER
    Matching placebo
  • Best Supportive Care — OTHER
    Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, proton pump inhibitors (PPI), sodium cromoglycate, antidepressants, leukotriene antagonists and corticosteroids.

Study Details

The purpose of this study is to evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients suffering from smouldering or indolent systemic mastocytosis with severe symptoms of mast cell mediator release, unresponsive to optimal symptomatic treatment.

Key Dates

Start date
Jul 1, 2020
Status verified
May 2023
Primary completion
Dec 31, 2024
Completion
Jun 30, 2025

Study Design

Enrollment
140 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Masitinib & BSC
    Experimental Arm: Masitinib (titration to 6.0 mg/kg/day) administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC). Participants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment, followed by dose escalation to 6.0 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control.
  • Placebo Comparator: Placebo & BSC
    Placebo Comparator: Matching placebo administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC)

Primary Outcome Measure

Cumulative response (3R75%) [ Time Frame: 24 weeks ]

Central Contacts

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