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Hydroxychloroquine in Isolated Cutaneous Mastocytosis Patients or Indolent Systemic Mastocytosis With Associated Skin Involvement Patients (HCQMa)

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ClinicalTrials.gov Identifier: NCT05084872
Recruitment Status : Not yet recruiting
First Posted : October 20, 2021
Last Update Posted : October 20, 2021
Sponsor:
Information provided by (Responsible Party):
University Hospital, Toulouse

Brief 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.


Condition or disease Intervention/treatment Phase
Mastocytosis Drug: Hydroxychloroquine Phase 2 Phase 3

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Hydroxychloroquine in Isolated Cutaneous Mastocytosis Patients or Indolent Systemic Mastocytosis With Associated Skin Involvement Patients: Proof of Concept Study
Estimated Study Start Date : October 2021
Estimated Primary Completion Date : January 2024
Estimated Study Completion Date : January 2024


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




Primary Outcome Measures :
  1. Change of mast cell activation symptoms [ Time Frame: 12 month ]
    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.

  2. Change of mast cell activation symptoms [ Time Frame: 12 month ]
    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


Secondary Outcome Measures :
  1. Difference on mast cell burden - serum tryptase level [ Time Frame: 12 month ]
    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.

  2. Difference on skin mast cell burden - mast cells/mm² [ Time Frame: 12 month ]
    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.

  3. Difference of mast cell activation symptoms : diarrhea [ Time Frame: 12 month ]
    The difference of diarrhea between the start of treatment and 12 months later evaluated by the absolute number of stools / day for diarrhea

  4. Difference of mast cell activation symptoms : pollakiuria [ Time Frame: 12 month ]
    The difference of pollakiuria between the start of treatment and 12 months later assessed by the absolute number of urinations / day for pollakiuria.

  5. Difference of mast cell activation symptoms : arthralgia [ Time Frame: 12 month ]
    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.

  6. Difference of mast cell activation symptoms : discomfort [ Time Frame: 12 month ]
    The difference of discomfort between the start of treatment and 12 months later evaluated by the absolute number of faintness / week

  7. The safety of hydroxychloroquine treatment. [ Time Frame: 12 month ]
    The safety of hydroxychloroquine treatment will be done by evaluation of adverse events

  8. effectiveness of treatment [ Time Frame: 12 month ]
    The correlation between the efficacy of treatment with the hydroxychloroquine and level of serum HCQ will be performed by the Bland-Altman test.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05084872


Contacts
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Contact: Maella Severino-Freire, MD 05 67 77 81 41 ext +33 severino-freire.m@chu-toulouse.fr
Contact: Cristina Bulai Livideanu, MD 05 67 77 81 35 ext +33 livideanu.c@chu-toulouse.fr

Locations
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France
Larrey Hospital - Toulouse University Hospital
Toulouse, France, 31059
Sponsors and Collaborators
University Hospital, Toulouse
Investigators
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Principal Investigator: Maella Severino-Freire, MD Toulouse University Hospital
Publications:
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Responsible Party: University Hospital, Toulouse
ClinicalTrials.gov Identifier: NCT05084872    
Other Study ID Numbers: RC31/19/0504
2020-003268-25 ( EudraCT Number )
First Posted: October 20, 2021    Key Record Dates
Last Update Posted: October 20, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University Hospital, Toulouse:
Mastocytosis
mast cell activation symptoms
hydroxychloroquine
Additional relevant MeSH terms:
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Mastocytosis
Mastocytosis, Systemic
Mastocytosis, Cutaneous
Mastocytoma
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Skin Diseases
Immune Complex Diseases
Hypersensitivity
Immune System Diseases
Hydroxychloroquine
Antimalarials
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antirheumatic Agents