Treatment of severe indolent systemic mastocytosis with masitinib
Phase 3 Study to Compare Oral Masitinib to Placebo in Treatment of Patients With Smouldering or Indolent Severe Systemic Mastocytosis, Unresponsive to Optimal Symptomatic Treatment
PHASE3 · AB Science · NCT04333108
This study is testing if a new medication called masitinib can help people with severe symptoms of systemic mastocytosis who haven't found relief from other treatments.
Quick facts
| Phase | PHASE3 |
|---|---|
| Study type | Interventional |
| Enrollment | 140 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | AB Science (industry) |
| Drugs / interventions | masitinib |
| Locations | 17 sites (Amiens and 16 other locations) |
| Trial ID | NCT04333108 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the efficacy and safety of oral masitinib compared to a placebo in patients with severe symptoms of indolent or smoldering systemic mastocytosis who have not responded to optimal symptomatic treatments. The study is a multicenter, randomized, double-blind, placebo-controlled trial that involves a 24-week treatment period. Participants will receive escalating doses of masitinib, a selective tyrosine kinase inhibitor, aimed at modulating mast cell activity. The trial focuses on patients experiencing significant symptoms related to mast cell mediator release.
Who should consider this trial
Good fit: Ideal candidates are patients with documented smoldering or indolent systemic mastocytosis and severe symptoms unresponsive to previous treatments.
Not a fit: Patients with cutaneous mastocytosis or those who have not experienced treatment failure with prior therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly alleviate severe symptoms for patients suffering from systemic mastocytosis.
How similar studies have performed: Other studies have shown promise with similar approaches targeting mast cell activity, but this specific application of masitinib is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Patient with one of the following documented mastocytosis subtypes (variants): Smouldering Systemic Mastocytosis, Indolent Systemic Mastocytosis 2. An excess of mast cells or a presence of abnormal mast cells in at least two organs (among skin, bone-marrow and GI Tract). 3. Patient with documented systemic mastocytosis and evaluable disease based upon histological criteria 4. Patient with documented treatment failure of his/her symptom(s) (within the past 2 years) with at least two of the symptomatic treatments used at optimized dose: Anti H1, Anti H2, Proton pump inhibitor, Antidepressants, Cromoglycate Sodium, Antileukotriene. 5. Patient with severe symptoms of mastocytosis over the 14-day run-in period including at least one among pruritus, flushes, and depression: pruritus score ≥ 9, number of flushes per week ≥ 8, Hamilton rating scale for depression (HAMD-17) score ≥ 19. Exclusion Criteria: 1. Patient with one of the following mastocytosis: Cutaneous Mastocytosis, Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD), Mast cell leukemia (MCL), Aggressive systemic mastocytosis (ASM) 2. Previous treatment with any Tyrosine Kinase Inhibitor 3. Treatment with any investigational agent within 8 weeks prior to screening.
Where this trial is running
Amiens and 16 other locations
- Centre Hospitalier Universitaire d'Amiens — Amiens, France (RECRUITING)
- Hospital Jean-Minjoz — Besançon, France (RECRUITING)
- Grenoble University Hospital — Grenoble, France (RECRUITING)
- Hospital Claude Huriez — Lille, France (RECRUITING)
- Marseille University Hospital Timone — Marseille, France (RECRUITING)
- Centre de référence de Mastocytose (CEREMAST) — Paris, France (RECRUITING)
- Poitiers University Hospital — Poitiers, France (RECRUITING)
- Centre Hospitalier Universitaire — Toulouse, France (RECRUITING)
- University Hospital Charité — Berlin, Germany (NOT_YET_RECRUITING)
- Erasmus University Medical Center — Rotterdam, Netherlands (RECRUITING)
- Medical University of Gdańsk — Gdańsk, Poland (RECRUITING)
- The University Hospital in Krakow (Szpital Uniwersytecki w Krakowie) — Kraków, Poland (RECRUITING)
- University Hospital in Bucharest (Spitalul Universitar de Urgență București) — Bucharest, Romania (RECRUITING)
- Almazov National Medical Research Centre — Saint Petersburg, Russian Federation (RECRUITING)
- Dnipropetrovsk Clinical Association of Emergency Medical Care of Dnipropetrovsk Regional — Dnipropetrovs'k, Ukraine (RECRUITING)
- Private Enterprise Private Manufacturing Company Acinus — Poltava, Ukraine (RECRUITING)
- Guy's and St Thomas' NHS Foundation Trust — London, United Kingdom (RECRUITING)
Study contacts
- Principal investigator: Cristina Bulai Livideanu, MD, MSc — Centre Hospitalier Universitaire, Service de Dermatologie, Toulouse -France
- Study coordinator: Clinical Study Coordinator
- Email: clinical@ab-science.com
- Phone: +33(0)147200014
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Indolent Systemic Mastocytosis, Mastocytosis with handicap, Mastocytosis, Mast cell, Mast cell infiltration, Skin, Bone marrow, Pruritus