HYDROXYCHLOROQUINE SULFATE for Cutaneous mastocytosis|Indolent systemic mastocytosis

Inclusion criteria

• {"criterion_text":"- Age > 18 years"}
• {"criterion_text":"- Isolated Cutaneous mastocytosis or indolent systemic mastocytosis with associated skin lesions defined according to WHO criteria (and / or international standards for cutaneous mastocytosis)"}
• {"criterion_text":"- Patient with at least one disability defined by the presence of the following symptoms assessed as moderate to severe: a. Cutaneous pruritus with score ≥ 3 on a VAS scale from 0 to 10 b. Number of flushs / week ≥ 5"}
• {"criterion_text":"- Skin KIT mutation known"}
• {"criterion_text":"- Performance scale: OMS/ECOG ≤ 1"}
• {"criterion_text":"- Woman and man of childbearing age under effective contraception during treatment with hydroxychloroquine and up to 8 months after stopping it"}

Exclusion criteria

• {"criterion_text":"- Non-symptomatic mastocytosis and / or without skin involvement"}
• {"criterion_text":"- Change symptomatic treatment (including dosage) in the 4 weeks preceding the inclusion visit"}
• {"criterion_text":"- Inability to give informed consent"}
• {"criterion_text":"- Inability to undergo medical monitoring for geographical, social or psychic"}
• {"criterion_text":"- Patients with major surgery scheduled in the next two weeks screening"}
• {"criterion_text":"- Patient without health insurance"}
• {"criterion_text":"- Pregnancy, Breastfeeding"}
• {"criterion_text":"- Vulnerable Patient, defined as: - Esperance survival < 6 months - Patient with another uncontrolled severe disease"}
• {"criterion_text":"- Patient under juridical protection"}
• {"criterion_text":"- Systemic mastocytosis with associated hematologic neoplasm (SMAHN)"}
• {"criterion_text":"- Aggressive systemic mastocytosis"}
• {"criterion_text":"- Mast cell leukemia"}
• {"criterion_text":"- History of ophthalmic disease and / or cardiac conduction disorders against-indicating the use of hydroxychloroquine"}
• {"criterion_text":"- Treatment with citalopram, escitalopram, hydroxyzine, domperidone, piperaquine due to the increased risk of ventricular rhythm disorders, especially torsades de pointes"}
• {"criterion_text":"- Specific anti-tumor treatment (chemotherapy, radiotherapy) of less than 4 weeks before inclusion."}
• {"criterion_text":"- Concomitant specific anti-mast cell treatment"}
• {"criterion_text":"- Inclusion in another trial with an experimental therapeutic molecule"}

Primary endpoints

• {"endpoint_text":"- The primary endpoint of this study is the change of mast cell activation symptoms as pruritus and/or flushes between the start of treatment and 12 months later.","definition_or_measurement_approach":"Change between start of treatment and 12 months in mast cell activation symptoms (pruritus and/or flushes)."}

Secondary endpoints

• {"endpoint_text":"- - The difference on mast cell mass between the start of treatment and 12 months later","definition_or_measurement_approach":"Difference in mast cell mass measured between start of treatment and 12 months."}
• {"endpoint_text":"- The difference of other mast cell activation symptoms such as diarrhea, pollakiuria, arthralgia and discomfort between the start of treatment and 12 months later.","definition_or_measurement_approach":"Change in other mast cell activation symptoms (diarrhea, pollakiuria, arthralgia, discomfort) between start and 12 months."}
• {"endpoint_text":"- Tolerance by clinical examination and if necessary paraclinical according to the investigator's decision; tolerance will be assessed during the study period","definition_or_measurement_approach":"Safety/tolerability assessed by clinical examination and paraclinical tests as needed throughout the study per investigator's judgment."}
• {"endpoint_text":"- The correlation of the effectiveness of treatment with the hydroxychloroquine and level of serum HCQ","definition_or_measurement_approach":"Correlation analysis between treatment effectiveness and serum hydroxychloroquine (HCQ) levels."}

France

Earliest CTIS Part Ii Submission Date

15-10-2024

Latest Decision Or Authorization Date

03-10-2025

Processing Time Days

353

Number Of Sites

2

Number Of Participants

30

Primary sponsor

Full Name

Centre Hospitalier Universitaire De Toulouse

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France