← Back to Search

Tyrosine Kinase Inhibitor

Masitinib for Severe Mast Cell Activation Syndrome

Phase 2

Recruiting

Led By Julien Rossignol, MD

Research Sponsored by AB Science

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 8 to week 24

Awards & highlights

Study Summary

This trial is testing the effectiveness and safety of two different doses of masitinib, compared to placebo, in treating patients with severe MCAS who have not responded well to other treatments.

Who is the study for?

This trial is for patients with severe Mast Cell Activation Syndrome (MCAS) who haven't responded to other treatments. They must have had significant symptoms like intense itching, frequent flushes, or depression during a two-week period and failed at least two optimized dose treatments in the past two years. Participants should be on a stable anti-histamine regimen and not pregnant or breastfeeding.Check my eligibility

What is being tested?

The trial tests how effective and safe different doses of oral masitinib are compared to a placebo in treating severe MCAS unresponsive to optimal symptomatic treatment. Patients will receive either masitinib at 4.5 mg/kg/day, masitinib at 6.0 mg/kg/day, placebo, or best supportive care.See study design

What are the potential side effects?

Masitinib may cause side effects such as nausea, vomiting, diarrhea, rash, fatigue and edema. It can also affect blood counts leading to anemia or increase the risk of infections due to weakened immune responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have severe itching, frequent hot flashes, or significant depression.

Select...

I've tried at least two treatments for my condition without success in the last two years.

Select...

I have been diagnosed with mast cell activation syndrome.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 8 to week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 8 to week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 8 to week 24 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Confirmed response at 50%

Secondary outcome measures

Confirmed response (75%)

Cumulative response

Patient-Reported Outcome for Symptom Severity (PROSS)

Side effects data

From 2009 Phase 3 trial • 463 Patients • NCT00113763

66%

ERYTHEMA

58%

PRURITUS

57%

DERMATITIS ACNEIFORM

28%

ANOREXIA

26%

FATIGUE

25%

PARONYCHIA

24%

ABDOMINAL PAIN

22%

RASH

22%

NAUSEA

21%

DIARRHOEA

20%

SKIN FISSURES

19%

CONSTIPATION

18%

EXFOLIATIVE RASH

18%

VOMITING

16%

PYREXIA

16%

DYSPNOEA

15%

COUGH

14%

ACNE

13%

COLORECTAL CANCER METASTATIC

12%

OEDEMA PERIPHERAL

12%

ASTHENIA

10%

BACK PAIN

10%

NAIL DISORDER

10%

DRY SKIN

SKIN EXFOLIATION

ABDOMINAL PAIN UPPER

INSOMNIA

COLORECTAL CANCER

STOMATITIS

SKIN ULCER

GENERAL PHYSICAL HEALTH DETERIORATION

GROWTH OF EYELASHES

MUCOSAL INFLAMMATION

JAUNDICE

ANXIETY

HEPATOMEGALY

WEIGHT DECREASED

INTESTINAL OBSTRUCTION

ANAEMIA

ASCITES

HEPATIC FAILURE

DEHYDRATION

COMA

GASTROINTESTINAL OBSTRUCTION

RECTAL HAEMORRHAGE

HYPERBILIRUBINAEMIA

CACHEXIA

METASTASES TO LIVER

METASTASES TO LUNG

HEMIPARESIS

EPILEPSY

DEPRESSED LEVEL OF CONSCIOUSNESS

HEPATIC ENCEPHALOPATHY

PULMONARY EMBOLISM

DEEP VEIN THROMBOSIS

JUGULAR VEIN THROMBOSIS

100%

80%

60%

40%

20%

Study treatment Arm

Panitumumab Plus BSC

BSC Alone

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Masitinib (6.0) & BSCExperimental Treatment2 Interventions

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.

Group II: Masitinib (4.5) & BSCExperimental Treatment2 Interventions

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.

Group III: Placebo & BSCPlacebo Group2 Interventions

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.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

AB ScienceLead Sponsor

38 Previous Clinical Trials

15,650 Total Patients Enrolled

Julien Rossignol, MDPrincipal InvestigatorReference Centre for Mastocytosis (CEREMAST), Necker Hospital, Paris, France

Media Library

Masitinib (Tyrosine Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05449444 — Phase 2

Mast Cell Activation Syndrome Research Study Groups: Masitinib (6.0) & BSC, Placebo & BSC, Masitinib (4.5) & BSC

Mast Cell Activation Syndrome Clinical Trial 2023: Masitinib Highlights & Side Effects. Trial Name: NCT05449444 — Phase 2

Masitinib (Tyrosine Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05449444 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the FDA sanctioned Best supportive care?

"Best supportive care has been assigned a safety rating of 2, given that there is some evidence confirming it's security but none showing its effectiveness."

Answered by AI

Is it possible to still apply for participation in this research endeavor?

"Affirmative, clinicaltrials.gov confirms that this medical experiment was posted on July 1st 2022 and is currently recruiting for 72 participants from a single site."

Answered by AI

Am I eligible to enroll in this research study?

"This clinical trial is open to patients who have been diagnosed with mast cell activation syndrome and are between the ages of 18 and 75. A maximum of 72 individuals will be accepted into this medical experiment."

Answered by AI

Could persons of advanced age participate in this experiment?

"The participants of this trial must be aged between 18-75. For those under the age of 18, there are 377 studies available and for a person over 65 years old, 995 studies can be found."

Answered by AI

How many participants are being accepted into this research project?

"Affirmative. Per clinicaltrials.gov, this medical study is currently recruiting participants and was first posted on July 1st of 2022 with its last update occurring on the 18th of that same month. The goal is to enrol 72 patients at a single site."

Answered by AI

Recent research and studies

The LancetJournal

Masitinib for Treatment of Severely Symptomatic Indolent Systemic Mastocytosis: A Randomised, Placebo-controlled, Phase 3 Study

Lortholary, Olivier, Marie Olivia Chandesris, Cristina Bulai Livideanu, Carle Paul, Gérard Guillet, Ewa Jassem, Marek Niedoszytko, et al.. 2017. “Masitinib for Treatment of Severely Symptomatic Indolent Systemic Mastocytosis: A Randomised, Placebo-controlled, Phase 3 Study”. The Lancet. Elsevier BV. doi:10.1016/s0140-6736(16)31403-9.

clinicaltrials.govStudy

Masitinib for the Treatment of Severe Mast Cell Activation Syndrome

2022. "Masitinib for the Treatment of Severe Mast Cell Activation Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05449444.