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Systemic mastocytosis – Trials in Disease

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Ongoing Clinical Trials for Systemic Mastocytosis

There are currently 5 clinical trials ongoing for systemic mastocytosis, a rare disorder where mast cells accumulate abnormally in various body tissues. These studies are exploring new treatment options including targeted therapies and combination approaches across multiple European countries.

Clinical trial locations

Study of Avapritinib for Adults with Systemic Mastocytosis Who Previously Participated in an Avapritinib Study

This trial provides continued access to avapritinib (also known as BLU-285) for patients who have already participated in a previous avapritinib study and are still benefiting from the treatment. The medication is taken orally as a film-coated tablet.

Main inclusion criteria: Patients must have participated in a previous Blueprint Medicines-sponsored study involving avapritinib and must still be receiving clinical benefit from the treatment, as determined by their doctor. Participants need to have completed their previous study according to protocol requirements. Female patients of childbearing potential must have a negative pregnancy test within 20 days before starting treatment, and all patients must agree to use effective birth control methods.

Main exclusion criteria: Patients who have not participated in a Blueprint Medicines sponsored study, those who are no longer benefiting from avapritinib treatment, individuals younger than 3 years old, and those not diagnosed with the condition cannot participate.

Trial focus: The study aims to ensure uninterrupted treatment access for patients who have shown positive results with avapritinib until the medication becomes commercially available. The trial involves regular monitoring and assessments to evaluate continued clinical benefit and safety.

Investigational drug: Avapritinib is a kinase inhibitor that works by blocking specific enzymes involved in cell growth and survival. It is designed to target the abnormal accumulation of mast cells characteristic of the condition.

Study on the Safety and Effectiveness of CGT9486 for Patients with Nonadvanced Systemic Mastocytosis

This trial is testing bezuclastinib (CGT9486) in patients with nonadvanced forms of the condition, including indolent, smoldering, and bone marrow variants. The study compares bezuclastinib with a placebo to evaluate safety and effectiveness.

Main inclusion criteria: Patients must be diagnosed with indolent systemic mastocytosis, smoldering systemic mastocytosis, or bone marrow mastocytosis. They need to have moderate-to-severe symptoms according to a specific questionnaire and be on stable treatment with at least two symptom-controlling medications for at least 14 days. An ECOG Performance Status of 0 to 2 is required, meaning participants should be able to care for themselves despite some symptoms.

Main exclusion criteria: Pregnant or breastfeeding women, individuals who participated in another clinical trial within the last 30 days, those with known allergies to the study medication, patients unable to take oral medication, those with recent drug or alcohol abuse, certain heart conditions, uncontrolled high blood pressure, active infections requiring treatment, and most cancer histories are excluded.

Trial focus: The study is divided into three parts: determining the best dose of bezuclastinib, assessing how well it works compared to placebo, and understanding the safety profile and patient tolerance. Participants are randomly assigned to receive either the medication or placebo in a double-blind design.

Investigational drug: Bezuclastinib is an oral medication that targets specific molecular pathways involved in the disease process. It is classified as a targeted therapy designed to reduce symptom burden and improve quality of life.

Study on Dupilumab and Fexofenadine for Treating Indolent Systemic Mastocytosis with Skin Involvement in Patients

This study examines the effectiveness of dupilumab compared to fexofenadine in treating patients with indolent systemic mastocytosis who have skin involvement. Dupilumab is administered as an injection, while fexofenadine is taken orally as a tablet.

Main inclusion criteria: Patients must be diagnosed with indolent systemic mastocytosis with a slow disease course and skin involvement. They should be between 18 and 65 years of age and have moderate additional disease burden in at least 2 out of 6 specific areas: extensive skin lesions (30% or more body surface area), significant itching (VAS score of 3 or more), reduced quality of life (MC-QoL score of 50 points or more), depression (Hamilton Rating Scale score of 19 or more), severe fatigue (Fatigue Impact Scale score of 75 points or more), or impaired quality of life (EORTC QLQ C30 score of 83 points or less).

Main exclusion criteria: Patients with conditions other than indolent systemic mastocytosis with skin involvement, those outside the specified age range, and individuals not meeting the gender requirements cannot participate.

Trial focus: The study compares dupilumab’s ability to reduce symptoms such as skin lesions, itching, and overall quality of life improvements against the standard antihistamine treatment fexofenadine. Some participants may receive a placebo for comparison purposes.

Investigational drugs: Dupilumab is a monoclonal antibody that blocks specific proteins causing inflammation, primarily used for conditions like eczema and asthma. Fexofenadine is a second-generation antihistamine that blocks histamine to relieve allergy symptoms.

Study of Avapritinib for Patients with Indolent Systemic Mastocytosis Not Adequately Managed by Standard Therapy

This trial evaluates avapritinib for patients with indolent systemic mastocytosis whose symptoms are not adequately controlled by standard treatments. The medication is a tyrosine kinase inhibitor that targets specific KIT gene mutations.

Main inclusion criteria: Patients must be 18 years or older with confirmed systemic mastocytosis based on bone marrow biopsy review. They need to have moderate-to-severe symptoms with a minimum symptom score of 28 over a 14-day period. Participants must have tried at least two different standard treatments (such as antihistamines, stomach acid reducers, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab) without adequate symptom control. Current treatments must be stable for at least 14 days before assessment. An ECOG Performance Status of 0 to 2 is required.

Main exclusion criteria: Patients without indolent systemic mastocytosis, those outside the specified age range, individuals unwilling or unable to follow study procedures, those with interfering medical conditions or medications, pregnant or breastfeeding women, and recent participants in other clinical trials are excluded.

Trial focus: The study is divided into three parts: determining the appropriate avapritinib dose, comparing symptom changes between avapritinib and placebo groups, and assessing long-term safety and effectiveness. Regular health assessments including vital signs, heart tests, and laboratory work monitor participant safety.

Investigational drug: Avapritinib is a tyrosine kinase inhibitor specifically designed to target KIT mutations found in patients with the condition. It aims to reduce symptoms not well controlled by conventional therapies.

Study of BLU-263 and Azacitidine for Patients with Advanced Systemic Mastocytosis and Other KIT Altered Blood Cancers

This trial studies elenestinib (BLU-263) both alone and in combination with azacitidine for patients with advanced systemic mastocytosis and other blood cancers involving KIT gene alterations.

Main inclusion criteria: Participants must have an ECOG performance status of 0-2 and provide a new bone marrow biopsy or use a sample taken within 56 days before starting. For single-drug treatment, patients must have aggressive systemic mastocytosis, systemic mastocytosis with an associated blood cancer not suitable for standard therapy, mast cell leukemia, or other KIT-altered blood conditions. For combination therapy, patients must have systemic mastocytosis with an associated blood cancer. Previous treatment with one selective KIT inhibitor is allowed if specific mutation criteria are met and sponsor approval is obtained.

Main exclusion criteria: Patients with conditions other than advanced systemic mastocytosis, those outside the specified age range, individuals unable to follow study procedures, those with interfering medical conditions, pregnant or breastfeeding women, current participants in other trials, recent major surgery recipients, those with allergic reactions to study medications, and individuals with certain abnormal laboratory results are excluded.

Trial focus: The study determines the best dose of BLU-263 alone and combined with azacitidine, then assesses safety and effectiveness. The trial includes dose escalation and expansion phases lasting until 2030, with regular monitoring including bone marrow biopsies.

Investigational drugs: BLU-263 (elenestinib) is an oral tyrosine kinase inhibitor that blocks signals needed for abnormal cell growth. Azacitidine is commonly used for blood disorders and cancers, helping to slow abnormal blood cell growth.

Summary

The five ongoing clinical trials for systemic mastocytosis represent diverse approaches to treating different forms of this rare condition. Three trials focus on avapritinib, highlighting significant research interest in this tyrosine kinase inhibitor across continuation, nonadvanced, and indolent disease variants. Two studies explore entirely new therapeutic approaches: bezuclastinib for nonadvanced cases and the combination of BLU-263 with azacitidine for advanced disease.

Geographically, trials are concentrated in Western and Northern Europe, with the Netherlands, Belgium, Norway, Germany, France, Italy, and Spain hosting multiple studies. The bezuclastinib trial has the broadest geographic reach across 12 countries, while the dupilumab study is limited to Poland. This distribution reflects both the rarity of the condition and the concentration of specialized treatment centers in certain European regions.

The trials address the full spectrum of disease severity, from skin involvement and nonadvanced forms to aggressive variants. Most studies require patients to have inadequately controlled symptoms despite standard treatments, emphasizing the need for new therapeutic options. The inclusion of placebo-controlled designs in several trials ensures rigorous evaluation of treatment effectiveness.

Ongoing Clinical Trials on Systemic mastocytosis

  • Study on Dupilumab and Fexofenadine for Treating Indolent Systemic Mastocytosis with Skin Involvement in Patients

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Poland

  • Study of Avapritinib for Adults with Systemic Mastocytosis Who Previously Participated in an Avapritinib Study

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Italy The Netherlands Norway

  • Study of BLU-263 and Azacitidine for Patients with Advanced Systemic Mastocytosis and Other KIT Altered Blood Cancers

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany The Netherlands Norway Spain

  • Study on the Safety and Effectiveness of CGT9486 for Patients with Nonadvanced Systemic Mastocytosis

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia France Germany Greece +6

  • Study of Avapritinib for Patients with Indolent Systemic Mastocytosis Not Adequately Managed by Standard Therapy

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Belgium Denmark France Germany Italy The Netherlands +3

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