Hydroxychloroquine in Isolated Cutaneous Mastocytosis Patients or Indolent Systemic Mastocytosis With Associated Skin Involvement Patients
NCT ID: NCT05084872
Last Updated: 2021-10-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE2/PHASE3
30 participants
INTERVENTIONAL
2021-10-31
2024-01-31
Brief Summary
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* Control of mast cell activation symptoms and
* The control of proliferation (accumulation) of mast cells.
There is no standard treatment and no treatment has a marketing authorization for the treatment of monoclonal indolent mastocytosis.
Detailed Description
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In adults, a classic distinction is made between isolated cutaneous forms (10 to 15%) and systemic forms (85 to 90%).
The treatment of systemic mastocytosis has two main axes:
* Control of mast cell activation symptoms and
* The control of proliferation (accumulation) of mast cells.
There is no standard treatment and no treatment has a marketing authorization for the treatment of monoclonal indolent mastocytosis.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Treatment
Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day
Hydroxychloroquine
Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day during 12 month
Interventions
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Hydroxychloroquine
Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day during 12 month
Eligibility Criteria
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Inclusion Criteria
2. Isolated Cutaneous mastocytosis or indolent systemic mastocytosis with associated skin lesions defined according to WHO criteria (and / or international standards for cutaneous mastocytosis)
3. Patient with at least one disability defined by the presence of the following symptoms assessed as moderate to severe:
1. Cutaneous pruritus with score ≥ 5 on a VAS scale from 0 to 10
2. Number of flushes / week ≥ 7
4. Skin KIT mutation known
5. Performance scale: OMS/ECOG ≤ 1
6. Woman and man of childbearing age\* under effective contraception during all the treatment by hydroxychloroquine, until 8 months after its cessation
Exclusion Criteria
* Advanced Systemic mastocytosis
* History of ophthalmic disease and / or cardiac conduction disorders, in particular the prolongation of the QT interval as well as the risk factors for prolongation of the QT interval, such as heart disease (heart failure, myocardial infarction), pro-arrhythmic conditions (eg bradycardia \<50 bpm), history of ventricular dysrhythmias, uncorrected hypokalemia and / or hypomagnesemia, concomitant treatment with interval prolonging agents QTagainst-indicating the use of hydroxychloroquine
* Treatment with citalopram, escitalopram, hydroxyzine, domperidone, piperaquine due to the increased risk of ventricular rhythm disorders, especially torsades de pointes
* Specific anti-tumor treatment (chemotherapy, radiotherapy) of less than 4 weeks before inclusion.
* Concomitant specific anti-mast cell treatment
* Contre-indication(s) to XYLOCAINE 10 mg/ml ADRENALINE 0,005 mg/ml, injectable solution: Known hypersensitivity to chlorhydrate de lidocaïne, to amide-type local anaesthetics or one of its excipients (sulfites), patients suffering from recurring porphyrias, coronary insufficiency, ventricular rhythm disorders, severe arterial hypertension, obstructive cardiomyopathy, hyperthyroidism.
* Inclusion in another trial with an experimental therapeutic molecule
* Change symptomatic treatment (including dosage) in the 4 weeks preceding the inclusion visit
* Moderate to severe renal or hepatic failure or diabetes
* History of organ transplant
* Inability to give informed consent
* Inability to undergo medical monitoring for geographical, social or psychic
* Patients with major surgery scheduled in the next two weeks screening
* Patient without health insurance
* Pregnancy, Breastfeeding
* Vulnerable Patient, defined as:
* Esperanzae survival \< 6 months
* Patient with another uncontrolled severe disease
* Patient under juridical protection
18 Years
ALL
No
Sponsors
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University Hospital, Toulouse
OTHER
Responsible Party
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Principal Investigators
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Maella Severino-Freire, MD
Role: PRINCIPAL_INVESTIGATOR
University Hospital, Toulouse
Locations
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Larrey Hospital - Toulouse University Hospital
Toulouse, , France
Countries
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Central Contacts
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References
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Fradet M, Negretto M, Tournier E, Laurent C, Apoil PA, Evrard S, Degboe Y, Del Mas V, Lamant L, Dubreuil P, Laroche M, Mailhol C, Hermine O, Paul C, Bulai Livideanu C. Frequency of isolated cutaneous involvement in adult mastocytosis: a cohort study. J Eur Acad Dermatol Venereol. 2019 Sep;33(9):1713-1718. doi: 10.1111/jdv.15638. Epub 2019 May 17.
Severino M, Chandesris MO, Barete S, Tournier E, Sans B, Laurent C, Apoil PA, Lamant L, Mailhol C, Laroche M, Fraitag S, Hanssens K, Dubreuil P, Hermine O, Paul C, Bulai Livideanu C. Telangiectasia macularis eruptiva perstans (TMEP): A form of cutaneous mastocytosis with potential systemic involvement. J Am Acad Dermatol. 2016 May;74(5):885-91.e1. doi: 10.1016/j.jaad.2015.10.050. Epub 2016 Feb 19.
Other Identifiers
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2020-003268-25
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
RC31/19/0504
Identifier Type: -
Identifier Source: org_study_id