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Mastocytosis, Indolent SystemicDALY-MASTNCT05923372

Evaluation of the DALY on a Cohort of Patients With Indolent Systemic Mastocytosis

An observational study of Mastocytosis, Indolent Systemic, run by University Hospital, Toulouse at 1 site in France.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

Mastocytosis is a group of pathologies characterized by the accumulation and/or clonal proliferation of abnormal mast cells in various organs. The impact of mastocytosis on health, quality of life, the psychoaffective sphere and on professional life constitutes the burden of this disease. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Mastocytosis, Indolent Systemic
How many people
160 participants
Where
1 siteFrance
Study dates
1 May 2023to 1 May 2024
Phase
Observational
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

Study population: All patients with MSI, included in the CEREMAST register and who have given their consent to participate

Who may be able to join

  • Having expressed their non opposition to participate in this study
  • Diagnosed with indolent mastocytosis and cared for in the CEREMAST reference center in Toulouse

Who cannot join

  • Patient with a form of mastocytosis other than MSI
  • Patient under legal protection, guardianship or curatorship

Trial locations and contacts

1 site in 1 country.

Cristina BULAI LIVIDEANU, MD contact

0567778138

livideanu.c@chu-toulouse.fr

France1 site, 1 recruiting

  • Centre de Référence des Mastocytoses, Service de Dermatologie, Hôpital Larrey, CHU Toulouse

    Toulouse, Haute-Garonne, 31059

    Cristina BULAI LIVIDEANU, PH, 0567778138, livideanu.c@chu-toulouse.fr

    Recruiting

Other trials in France

About this study

Official title: Evaluation of the Disability-Adjusted Life Year (DALY) on a Cohort of Patients With Indolent Systemic Mastocytosis (ISM)

Mastocytosis is a group of pathologies characterized by the accumulation and/or clonal proliferation of abnormal mast cells in various organs. The impact of mastocytosis on health, quality of life, the psychoaffective sphere and on professional life constitutes the burden of this disease. The DALY is the disability-adjusted life expectancy, which takes into account the life expectancy and the number of years "lost" due to illness, disability or early death. Due to the major impact of the disease this study evaluates the DALY in indolent mastocytosis patients

Read the detailed description

Mastocytosis is a group of pathologies characterized by the accumulation and/or clonal proliferation of abnormal mast cells in various organs. Mastocytosis is a rare disease with an estimated prevalence of between 1/20,000 and 1/40,000. The impact of mastocytosis on health, quality of life, the psychoaffective sphere and on professional life constitutes the burden of this disease. This is probably important but very little studied so far. The DALY is the disability-adjusted life expectancy. It is calculated by subtracting from life expectancy the number of years "lost" due to illness, disability or early death.

Ignorance of the disease and diagnostic error cause significant distress in patients. In our clinical experience, the impact of the disease on the personal, social, and professional life of our patients is major. It therefore seems important to better specify the impact on the quality of life and the costs of this pathology. The main objective of this study is to evaluate the DALY in indolent mastocytosis patients from the CEREMAST reference center of the Toulouse University Hospital.

Keywords
DALY, Mastocytosis, Quality of life
Medical subject headings
Mastocytosis, Systemic, Mastocytosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT05923372
Study groupRoleWhat they receive
Patients with indolent systemic mastocytosis (IMS)Patients with IMS, included in the CEREMAST registry and who have given their consent to participate

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. DALY

    DALY (Disability Adjusted Life Year): Its calculation consists of subtracting from life expectancy the number of years "lost" due to illness, disability or early death.

    Measured: Day 1

Secondary outcomes (2)

  1. Quality of life based on the MC QOL scale

    The quality of life of the included patients will be described with the The Mastocytosis Quality of Life Questionnaire (MC-QoL) which has scores from 0 to 100, where higher scores indicate higher health-related quality-of-life impairment

    Measured: Day 1

  2. Frequency of symptoms

    The frequency of various symptoms of mast cell activation will also be described.

    Measured: Day 1

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Jun 2023
  2. Study start1 May 2023
  3. Primary completion1 May 2024 (estimated)
  4. Study completion1 May 2024 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Other
Enrolment
160 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05923372
Sponsor's protocol ID
RC31/23/0011
Other IDs
2023-A00178-37
Acronym
DALY-MAST
Status last verified
Jul 2023
First submitted
19 Jun 2023
Last updated
12 Jul 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Siebenhaar F, Fortsch A, Krause K, Weller K, Metz M, Magerl M, Martus P, Church MK, Maurer M. Rupatadine improves quality of life in mastocytosis: a randomized, double-blind, placebo-controlled trial. Allergy. 2013 Jul;68(7):949-52. doi: 10.1111/all.12159. Epub 2013 Jun 4.

    PubMed 23734572

  • Siebenhaar F, von Tschirnhaus E, Hartmann K, Rabenhorst A, Staubach P, Peveling-Oberhag A, Wagner N, Martus P, Carter MC, Metcalfe DD, Church MK, Maurer M, Weller K. Development and validation of the mastocytosis quality of life questionnaire: MC-QoL. Allergy. 2016 Jun;71(6):869-77. doi: 10.1111/all.12842. Epub 2016 Feb 15.

    PubMed 26797792

  • van Anrooij B, Kluin-Nelemans JC, Safy M, Flokstra-de Blok BM, Oude Elberink JN. Patient-reported disease-specific quality-of-life and symptom severity in systemic mastocytosis. Allergy. 2016 Nov;71(11):1585-1593. doi: 10.1111/all.12920. Epub 2016 Jun 23.

    PubMed 27089859

  • Jennings SV, Slee VM, Zack RM, Verstovsek S, George TI, Shi H, Lee P, Castells MC. Patient Perceptions in Mast Cell Disorders. Immunol Allergy Clin North Am. 2018 Aug;38(3):505-525. doi: 10.1016/j.iac.2018.04.006.

    PubMed 30007467

  • Jennings S, Russell N, Jennings B, Slee V, Sterling L, Castells M, Valent P, Akin C. The Mastocytosis Society survey on mast cell disorders: patient experiences and perceptions. J Allergy Clin Immunol Pract. 2014 Jan-Feb;2(1):70-6. doi: 10.1016/j.jaip.2013.09.004. Epub 2013 Dec 3.

    PubMed 24565772

  • Nowak A, Gibbs BF, Amon U. Pre-inpatient evaluation on quality and impact of care in systemic mastocytosis and the influence of hospital stay periods from the perspective of patients: a pilot study. J Dtsch Dermatol Ges. 2011 Jul;9(7):525-32. doi: 10.1111/j.1610-0387.2011.07638.x. Epub 2011 Mar 24. English, German.

    PubMed 21992665

  • Sanchez-Munoz L, Morgado JM, Alvarez-Twose I, Matito A, Garcia-Montero AC, Teodosio C, Jara-Acevedo M, Mayado A, Mollejo M, Caldas C, Gonzalez de Olano D, Escribano L, Orfao A. Diagnosis and classification of mastocytosis in non-specialized versus reference centres: a Spanish Network on Mastocytosis (REMA) study on 122 patients. Br J Haematol. 2016 Jan;172(1):56-63. doi: 10.1111/bjh.13789. Epub 2015 Oct 12.

    PubMed 26456532

  • Jackson CW, Pratt CM, Rupprecht CP, Pattanaik D, Krishnaswamy G. Mastocytosis and Mast Cell Activation Disorders: Clearing the Air. Int J Mol Sci. 2021 Oct 19;22(20):11270. doi: 10.3390/ijms222011270.

    PubMed 34681933

  • Valent P, Horny HP, Escribano L, Longley BJ, Li CY, Schwartz LB, Marone G, Nunez R, Akin C, Sotlar K, Sperr WR, Wolff K, Brunning RD, Parwaresch RM, Austen KF, Lennert K, Metcalfe DD, Vardiman JW, Bennett JM. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res. 2001 Jul;25(7):603-25. doi: 10.1016/s0145-2126(01)00038-8.

    PubMed 11377686

  • Afrin LB. Mast cell activation disease and the modern epidemic of chronic inflammatory disease. Transl Res. 2016 Aug;174:33-59. doi: 10.1016/j.trsl.2016.01.003. Epub 2016 Jan 18.

    PubMed 26850903

  • Hermouet S, Bigot-Corbel E, Gardie B. Pathogenesis of Myeloproliferative Neoplasms: Role and Mechanisms of Chronic Inflammation. Mediators Inflamm. 2015;2015:145293. doi: 10.1155/2015/145293. Epub 2015 Oct 11.

    PubMed 26538820

Questions about this trial

What is this clinical trial studying?

An observational study of Mastocytosis, Indolent Systemic, 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 2 inclusion and 2 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 12 Jul 2023. 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.

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 Cristina BULAI LIVIDEANU, MD (0567778138, livideanu.c@chu-toulouse.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

DALY (time frame: Day 1). 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 NCT05923372, which also lists 11 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 12 Jul 2023.

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