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

NCT05084872 | Unknown Phase 2

• 2 other identifiers: RC31/19/05042020-003268-25
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

Mastocytosis

Keywords

Mastocytosis; mast cell activation symptoms; hydroxychloroquine

Outcome Measures

Primary Outcomes (2)

• 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

• 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 Outcomes (8)

• Difference on mast cell burden - serum tryptase level

  12 month

• Difference on skin mast cell burden - mast cells/mm²

  12 month

• Difference of mast cell activation symptoms : diarrhea

  12 month

• Difference of mast cell activation symptoms : pollakiuria

  12 month

• Difference of mast cell activation symptoms : arthralgia

  12 month

Study Arms (1)

Treatment

EXPERIMENTAL

Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day

Drug: Hydroxychloroquine

Interventions

Hydroxychloroquine DRUG

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

Treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \> 18 years
• Isolated Cutaneous mastocytosis or indolent systemic mastocytosis with associated skin lesions defined according to WHO criteria (and / or international standards for cutaneous mastocytosis)
• Patient with at least one disability defined by the presence of the following symptoms assessed as moderate to severe:
• Cutaneous pruritus with score ≥ 5 on a VAS scale from 0 to 10
• Number of flushes / week ≥ 7
• Skin KIT mutation known
• Performance scale: OMS/ECOG ≤ 1
• Woman and man of childbearing age\* under effective contraception during all the treatment by hydroxychloroquine, until 8 months after its cessation

You may not qualify if:

• 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
• 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.
• 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

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (1)

Larrey Hospital - Toulouse University Hospital

Toulouse, 31059, France

Location

Related 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.

  PMID: 31009132 BACKGROUND

• 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.

  PMID: 26899198 BACKGROUND

MeSH Terms

Conditions

Mastocytosis

Interventions

Hydroxychloroquine

Condition Hierarchy (Ancestors)

Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Mast Cell Activation Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Chloroquine; Aminoquinolines; Quinolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Maella Severino-Freire, MD

  University Hospital, Toulouse

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Maella Severino-Freire, MD

CONTACT

05 67 77 81 41; severino-freire.m@chu-toulouse.fr

Cristina Bulai Livideanu, MD

CONTACT

05 67 77 81 35; livideanu.c@chu-toulouse.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2021
• First Posted: October 20, 2021
• Study Start: October 1, 2021
• Primary Completion: January 1, 2024
• Study Completion: January 1, 2024
• Last Updated: October 20, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)