Recruiting
Phase 2

Masitinib

Sponsor:

AB Science

Code:

NCT05449444

Conditions

Mast Cell Activation Syndrome

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

Masitinib 4.5 mg/kg/day

Placebo

Best supportive care

Masitinib 6.0 mg/kg/day

Study Details

Brief summary:

To evaluate the efficacy and safety of two dosing schemes of oral masitinib versus matching placebo in the treatment of patients suffering from severe MCAS with handicap unresponsive to optimal symptomatic treatment.

Conditions

Mast Cell Activation Syndrome

Study ID

NCT05449444

Start date

Jul 1, 2022

Status verified date

Feb, 2023

Completion date

Dec 31, 2024

Anticipated

Primary completion date

Dec 31, 2024

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Inclusion Criteria include:

  • Patient with mast cell activation syndrome (MCAS).
  • Patient with severe symptoms over the 14-day run-in period defined as at least one of the following: Pruritus score ≥ 9; Number of flushes per week ≥ 8; Hamilton rating scale for depression (HAMD-17) score ≥ 19
  • Patient with documented treatment failures of his/her handicap(s) (within last two years) with at least two of the symptomatic treatments used at optimized dose.
  • Patients must be on a stable dose of Anti-H1 for a minimum of 4 weeks before screening and should remain at a stable dose throughout the study period.

Exclusion Criteria include:

  • Previous treatment with any Tyrosine Kinase Inhibitor.
  • Any change in the symptomatic treatment of MCAS, including systemic corticosteroids, or administration of any new treatment for MCAS within 4 weeks prior to screening.
  • Patient with systemic indolent mastocytosis.
  • Female patients who are pregnant or are breastfeeding.

Study Design

Enrollment

72 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Masitinib (4.5) & BSC

Masitinib 4.5 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) for 4 weeks, given orally twice daily, with a dose escalation to 4.5 mg/kg/day for the remainder of the treatment period. Each ascending dose titration is subjected to a safety control.

experimental: Masitinib (6.0) & BSC

Masitinib 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) for 4 weeks, given orally twice daily, with a dose escalation to 4.5 mg/kg/day for4 weeks of treatment, then a second dose escalation to 6 mg/kg/day for the remainder of the treatment period. Each ascending dose titration is subjected to a safety control.

placebo comparator: Placebo & BSC

Placebo administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC). Participants receive a matched dose placebo, given orally twice daily.

Interventions

Masitinib 4.5 mg/kg/day

Masitinib 4.5 mg/kg/day

Placebo

Matching placebo

Best supportive care

Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, sodium cromoglicate, antidepressants, antileukotrienes, proton pump inhibitors, and corticosteroids.

Masitinib 6.0 mg/kg/day

Masitinib 6.0 mg/kg/day

Primary outcome measure

  • Confirmed response at 50% [ Time Frame: 24 weeks ]

Central Contacts and Locations

Central contacts

Clinical Study Coordinator

+33(0)147200014clinical@ab-science.com

Locations

St Charles Clinical Research

Recruiting

Weldon Spring, Missouri, United States, 63304

More Information

Sponsor

AB Science

Last update posted

Feb 6, 2023

Last verified

Feb, 2023

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by AB Science on 2023-02-06.