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Recruiting NCT04942080

Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)

No phase Interventional Myeloproliferative Neoplasm Essential Thrombocythemia Primary Myelofibrosis, Prefibrotic Stage Primary Myelofibrosis, Fibrotic Stage

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: CALR allele burden quantification.
Who it may be relevant to
Registry conditions: Myeloproliferative Neoplasm, Essential Thrombocythemia, Primary Myelofibrosis, Prefibrotic Stage, Primary Myelofibrosis, Fibrotic Stage. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
France
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Overview

Prospective study to evaluate the relevance of CALR allele burden monitoring as a molecular marker of disease progression.

Detailed description

A first local study on 45 patients showed the prognostic impact of CALR mutation quantification in follow-up, independently of the European LeukemiaNet (ELN) prognostic score validated in this group of patients.

This study aims to evaluate a multicenter cohort of 260 patients, including all types of CALR-mutated MPNs and several follow-up samples, to model the temporal evolution of CALR allele burden.

Blood of MPN patients will be collected, at the time of diagnosis and for 3 years (max 1 sample/year), for the quantification of the CALR allele burden. During follow-up, a clinicobiological score to define the progression or not of the disease for each patient will be evaluated in Essential Thrombocythemia (ET) and MyeloFibrosis (MF).

Interventions

  • Biological CALR allele burden quantification
    * DNA extraction from blood sample for CALR mutation quantification (fragment analysis) * at diagnosis and follow-up (inclusion period: 3 years) * max 1 sample/year * secondary outcome: mutational landscape by Next Generation Sequencing (NGS) analysis at diagnosis

Primary outcome measures

  • For each disease, Hazard Ratio of the different trajectories of CALR allele burden to explain the time to onset of disease progression by the clinicobiological score. [Time frame: 3 years follow-up]
Secondary outcome measures (1)
  • A multinomial logistic model will be performed to identify the characteristics associated with the different trajectories of CALR allele burden (pathology, treatment, additional mutations, type of CALR mutation). [Time frame: 3 years follow-up]

Eligibility criteria

Inclusion criteria

  • adults (age ≥18 years),
  • affiliated to the national social security system,
  • with CALR mutated myeloproliferative neoplasm diagnosed between 2006 - 2020,
  • for which at least one sample is available at the time of diagnosis or before cytoreductive treatment,
  • who signed the consent to participate in the study,
  • included, or consenting to be included, in the national clinical-biological database of France Intergroupe Syndrome Myéloprolifératifs (FIM).

Exclusion criteria

  • patient with another active hematological disease or cancer at the time of diagnosis,
  • person subject to legal protection scheme or incapable of giving consent.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Other

Study locations

France · 10 centers
  • CHU Angers — Angers
  • Chu Bordeaux — Bordeaux
  • Chu Brest — Brest
  • Ch Cholet — Cholet
  • Ch Le Mans — Le Mans
  • Ch Morlaix — Morlaix
  • AP-HP Henri Mondor — Paris
  • Chu Poitiers — Poitiers
  • … and 2 more centers

Publications

  • Cottin L, Riou J, Orvain C, Ianotto JC, Boyer F, Renard M, Truchan-Graczyk M, Murati A, Jouanneau-Courville R, Allangba O, Mansier O, Burroni B, Rousselet MC, Quintin-Roue I, Martin A, Sadot-Lebouvier S, Delneste Y, Chretien JM, Hunault-Berger M, Blanchet O, Lippert E, Ugo V, Luque Paz D. Sequential mutational evaluation of CALR -mutated myeloproliferative neoplasms with thrombocytosis reveals an PMID 31710700

Identifiers

NCT: NCT04942080 · 49RC21_0156

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗