DUPILUMAB for Indolent systemic mastocytosis with skin involvement

Inclusion criteria

• {"criterion_text":"- • Diagnosis of systemic mastocytosis with a slow course according to WHO criteria and skin involvement of the above-mentioned. criteria"}
• {"criterion_text":"- • Age 18-65 lat"}
• {"criterion_text":"- • There is a moderate additional risk of a disease impairing the quality of life in 2 of 6 abnormalities: 1. Extent of skin lesions - % of skin function (BSA-Body Surface Area according to the Wallace 9 principles); importance of the impact of skin lesions (>=30% BSA) 2. Intensity of itching on the VAS scale assessment of this result on a scale of 0-10 points severe or severe itching >=3 on the VAS scale 3. Quality of life index of the MC-QoL questionnaire (symptoms causing with the release of mast cell mediators) MC-QoL score >=50 points 4. Severity of the score (Hamilton Rating Scale for Depression) Hamilton Rating Scale score >=19 5. Severity determines (Fatigue Impact Scale) Fatigue Impact Scale score >=75 points 6. Assessment of the quality of life of patients treated for hematologic disease using the European Organization for Research and Treatment of Cancer EORTC QLQ C30 questionnaire <= 83 points"}

Exclusion criteria

• {"criterion_text":"- • Other cancer diseases, including one of the aggressive forms of mastocytosis, isolated cutaneous mastocytosis (without systemic mastocytosis)."}
• {"criterion_text":"- • Liver, kidney and heart failure, respiratory failure"}
• {"criterion_text":"- • Chronic infectious diseases and parasitic infections, pregnancy, lactation, lack of cooperation with the patient."}
• {"criterion_text":"- • Exacerbation of chronic diseases."}
• {"criterion_text":"- • Hypersensitivity to study drugs"}

Primary endpoints

• {"endpoint_text":"- 1. Reducing the incidence of symptoms caused by the release of mast cell mediators, mainly anaphylaxis (number of anaphylaxis and flushing/week)","definition_or_measurement_approach":"Incidence measured as number of anaphylaxis and flushing per week."}
• {"endpoint_text":"- 2. Reducing the extent of skin lesions (BSA-Body Surface Area assessed according to the principle Nines Wallace)","definition_or_measurement_approach":"Extent of skin lesions measured by Body Surface Area (BSA) assessed according to the Wallace 'Nines' principle."}
• {"endpoint_text":"- 3. Reduction of itch severity (VAS)","definition_or_measurement_approach":"Itch severity measured using Visual Analog Scale (VAS)."}
• {"endpoint_text":"- 4. Improving the quality of life (MC-QoL, EORTC QLQ C30)","definition_or_measurement_approach":"Quality of life measured by MC-QoL and EORTC QLQ-C30 questionnaires."}
• {"endpoint_text":"- 5. Reducing symptoms of depression (Hamilton Rating Scale for Depression) and fatigue (Fatigue Impact Scale)","definition_or_measurement_approach":"Depression measured by the Hamilton Rating Scale for Depression; fatigue measured by the Fatigue Impact Scale."}

Secondary endpoints

• {"endpoint_text":"- 1. Quality of Life in Mastocytosis (QLMS) Questionnaire","definition_or_measurement_approach":"Measured using the QLMS (Quality of Life in Mastocytosis) questionnaire."}
• {"endpoint_text":"- 2. The incidence of life-threatening anaphylactic reactions","definition_or_measurement_approach":"Measured as incidence/count of life-threatening anaphylactic reactions."}
• {"endpoint_text":"- 3. Decreased concentration of tryptase, IL4 and IL13 in peripheral blood serum.","definition_or_measurement_approach":"Measured as concentration levels of tryptase, IL-4 and IL-13 in peripheral blood serum."}

Poland

Earliest CTIS Part Ii Submission Date

06-02-2025

Latest Decision Or Authorization Date

30-06-2025

Processing Time Days

145

Number Of Sites

3

Number Of Participants

66

Primary sponsor

Full Name

Medical University Of Gdansk

Organisation Type

Educational Institution

Country Of Registered Address

Poland

Third parties

• {"country":"","full_name":"Medical Research Agency (Agencja Badań Medycznych)","duties_or_roles":"Source of monetary support","organisation_type":""}