MastocytosisPhase 2/3HCQMaNCT05084872
Hydroxychloroquine in Isolated Cutaneous Mastocytosis Patients or Indolent Systemic Mastocytosis With Associated Skin Involvement Patients
A Phase 2/3 study testing Hydroxychloroquine in people with Mastocytosis, run by University Hospital, Toulouse at 1 site in France.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mastocytosis
- What is being tested?
- Hydroxychloroquine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2021to Jan 2024
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 20Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Hydroxychloroquine in Isolated Cutaneous Mastocytosis Patients or Indolent Systemic Mastocytosis With Associated Skin Involvement Patients: Proof of Concept Study
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.
Read the 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.
- Conditions
- Mastocytosis
- Being tested
- Hydroxychloroquine
- Keywords
- Mastocytosis, mast cell activation symptoms, hydroxychloroquine
- Medical subject headings
- Mastocytosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day during 12 month
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day Receives: Drug: Hydroxychloroquine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: 12 month
Secondary outcomes (8)
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.
Measured: 12 month
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.
Measured: 12 month
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
Measured: 12 month
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.
Measured: 12 month
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.
Measured: 12 month
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
Measured: 12 month
The safety of hydroxychloroquine treatment.
The safety of hydroxychloroquine treatment will be done by evaluation of adverse events
Measured: 12 month
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.
Measured: 12 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2021
- Study startOct 2021 (estimated)
- Primary completionJan 2024 (estimated)
- Study completionJan 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for MastocytosisStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Maella Severino-Freire, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05084872
- Sponsor's protocol ID
- RC31/19/0504
- Other IDs
- 2020-003268-25
- Acronym
- HCQMa
- Status last verified
- Oct 2021
- First submitted
- 6 Oct 2021
- Last updated
- 20 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 31009132Severino 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.
PubMed 26899198
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydroxychloroquine in Isolated Cutaneous Mastocytosis Patients or Indolent Systemic Mastocytosis With Associated Skin Involvement Patients: Proof of Concept Study
- ClinicalTrials.gov ID
- NCT05084872
- Acronym / sponsor ID
- HCQMa / RC31/19/0504
- Conditions
- Mastocytosis
- Keywords
- Mastocytosis, mast cell activation symptoms, hydroxychloroquine
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- Oct 2021 (estimated)
- Primary completion
- Jan 2024 (estimated)
- Study completion
- Jan 2024 (estimated)
- First posted
- 20 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: HydroxychloroquinePatients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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:
- Skin KIT mutation known
- Performance scale: OMS/ECOG ≤ 1
Key exclusion criteria (20 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxychloroquinePatients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day during 12 month | Drug | Treatment |
Primary outcome
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.
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
8 secondary outcomes
Difference on mast cell burden - serum tryptase level
Time frame: 12 month
Difference on skin mast cell burden - mast cells/mm²
Time frame: 12 month
Difference of mast cell activation symptoms : diarrhea
Time frame: 12 month
Difference of mast cell activation symptoms : pollakiuria
Time frame: 12 month
Difference of mast cell activation symptoms : arthralgia
Time frame: 12 month
Difference of mast cell activation symptoms : discomfort
Time frame: 12 month
The safety of hydroxychloroquine treatment.
Time frame: 12 month
effectiveness of treatment
Time frame: 12 month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2021.
- Background: PubMed 31009132Fradet 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.
- Background: PubMed 26899198Severino 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maella Severino-Freire, MD principal investigator
University Hospital, Toulouse
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Hydroxychloroquine in people with Mastocytosis, run by University Hospital, Toulouse at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Maella Severino-Freire, MD (05 67 77 81 41 ext. +33, severino-freire.m@chu-toulouse.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of mast cell activation symptoms (time frame: 12 month). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05084872, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2021; AzTrials last refreshed its copy on 8 Nov 2026.