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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT05084872
Other study ID # RC31/19/0504
Secondary ID 2020-003268-25
Status Not yet recruiting
Phase Phase 2/Phase 3
First received
Last updated
Start date October 2021
Est. completion date January 2024

Study information

Verified date October 2021
Source University Hospital, Toulouse
Contact Maella Severino-Freire, MD
Phone 05 67 77 81 41
Email severino-freire.m@chu-toulouse.fr
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Description:

Mastocytosis is an orphan disease related to the accumulation and / or the proliferation of abnormal mast cells in different tissues. 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.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 30
Est. completion date January 2024
Est. primary completion date January 2024
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: 1. Age > 18 years 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: - Non-symptomatic mastocytosis and / or without skin involvement - 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Hydroxychloroquine
Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day during 12 month

Locations

Country Name City State
France Larrey Hospital - Toulouse University Hospital Toulouse

Sponsors (1)

Lead Sponsor Collaborator
University Hospital, Toulouse

Country where clinical trial is conducted

France, 

References & Publications (2)

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. — View Citation

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. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Change of mast cell activation symptoms The primary endpoint of this study is the change of mast cell activation symptoms as pruritus between the start of treatment and 12 months later. Skin pruritus will be assessed by the visual analogue scale from 0 to 10 at each visit. 12 month
Primary Change of mast cell activation symptoms The primary endpoint of this study is the change of mast cell activation symptoms as flushes between the start of treatment and 12 months later. The skin flush will be evaluated according to the absolute number of flushes / week at each visit 12 month
Secondary Difference on mast cell burden - serum tryptase level The difference on mast cell burden between the start of treatment and 12 months later will be evaluated by variation of the level serum tryptase l expressed in µg / L. 12 month
Secondary Difference on skin mast cell burden - mast cells/mm² The difference on mast cell burden between the start of treatment and 12 months later will be assessed by variation of the number of mast cells / mm² identified on the skin biopsies. 12 month
Secondary Difference of mast cell activation symptoms : diarrhea The difference of diarrhea between the start of treatment and 12 months later evaluated by the absolute number of stools / day for diarrhea 12 month
Secondary Difference of mast cell activation symptoms : pollakiuria The difference of pollakiuria between the start of treatment and 12 months later assessed by the absolute number of urinations / day for pollakiuria. 12 month
Secondary Difference of mast cell activation symptoms : arthralgia The difference of arthralgia between the start of treatment and 12 months later evaluated by the absolute number of painful joints / day and the intensity of joint pain assessed by the visual analogue scale from 0 to 10 for arthralgia. 12 month
Secondary Difference of mast cell activation symptoms : discomfort The difference of discomfort between the start of treatment and 12 months later evaluated by the absolute number of faintness / week 12 month
Secondary The safety of hydroxychloroquine treatment. The safety of hydroxychloroquine treatment will be done by evaluation of adverse events 12 month
Secondary effectiveness of treatment The correlation between the efficacy of treatment with the hydroxychloroquine and level of serum HCQ will be performed by the Bland-Altman test. 12 month
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