Back to resultsInterest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI) (CALRSUIVI)
Primary Purpose
Myeloproliferative Neoplasm, Essential Thrombocythemia, Primary Myelofibrosis, Prefibrotic Stage
Status
Recruiting
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
CALR allele burden quantification
Sponsored by
About this trial
This is an interventional other trial for Myeloproliferative Neoplasm focused on measuring CALR, Myeloproliferative Neoplasm, Essential Thrombocythemia, Primary Myelofibrosis, Thrombocytosis
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.
Sites / Locations
- CHU AngersRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CALRSUIVI cohort
Arm Description
Outcomes
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.
Clinicobiological score :
For ET, disease progression if ≥ 1 of:
leukocytosis > 12 G/L or myelemia > 10% or erythroblasts > 1%,
anemia or thrombocytopenia not related to drug toxicity,
development or worsening of splenomegaly,
development of thrombocytosis (platelets > 450 G/L) on cytoreductive therapy,
poor disease control (at least one change in therapy for reasons other than adverse events),
hematologic transformation or death related to hematologic pathology.
For MF, disease progression if ≥ 1 of:
anemia < 100 g/L, neutropenia < 1 G/L, thrombocytopenia < 100 G/L and/or development of general signs not previously present or recurring after improvement,
increase in leukocytosis > 25 G/L not previously present,
appearance or aggravation of transfusion dependence,
appearance (> 5 cm) or aggravation (> 50%) of splenomegaly,
leukemic transformation or death related to the hematologic pathology.
Secondary Outcome Measures
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).
Full Information
NCT ID
NCT04942080
First Posted
June 9, 2021
Last Updated
November 2, 2022
Sponsor
University Hospital, Angers
Collaborators
Ligue contre le cancer, France
1. Study Identification
Unique Protocol Identification Number
NCT04942080
Brief Title
Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)
Acronym
CALRSUIVI
Official Title
Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)
Study Type
Interventional
2. Study Status
Record Verification Date
November 2022
Overall Recruitment Status
Recruiting
Study Start Date
October 28, 2021 (Actual)
Primary Completion Date
April 28, 2023 (Anticipated)
Study Completion Date
April 28, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Angers
Collaborators
Ligue contre le cancer, France
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myeloproliferative Neoplasm, Essential Thrombocythemia, Primary Myelofibrosis, Prefibrotic Stage, Primary Myelofibrosis, Fibrotic Stage
Keywords
CALR, Myeloproliferative Neoplasm, Essential Thrombocythemia, Primary Myelofibrosis, Thrombocytosis
7. Study Design
Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
260 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
CALRSUIVI cohort
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
CALR allele burden quantification
Intervention Description
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 Measure Information:
Title
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.
Description
Clinicobiological score :
For ET, disease progression if ≥ 1 of:
leukocytosis > 12 G/L or myelemia > 10% or erythroblasts > 1%,
anemia or thrombocytopenia not related to drug toxicity,
development or worsening of splenomegaly,
development of thrombocytosis (platelets > 450 G/L) on cytoreductive therapy,
poor disease control (at least one change in therapy for reasons other than adverse events),
hematologic transformation or death related to hematologic pathology.
For MF, disease progression if ≥ 1 of:
anemia < 100 g/L, neutropenia < 1 G/L, thrombocytopenia < 100 G/L and/or development of general signs not previously present or recurring after improvement,
increase in leukocytosis > 25 G/L not previously present,
appearance or aggravation of transfusion dependence,
appearance (> 5 cm) or aggravation (> 50%) of splenomegaly,
leukemic transformation or death related to the hematologic pathology.
Time Frame
3 years follow-up
Secondary Outcome Measure Information:
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Laurane COTTIN, Doctor
Phone
+33 2 41 35 53 53
Email
Laurane.Cottin@chu-angers.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Emma BLANCHET
Phone
+33 2 41 35 63 38
Email
EmBlanchet@chu-angers.fr
Facility Information:
Facility Name
CHU Angers
City
Angers
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Laurane COTTIN, Dr
12. IPD Sharing Statement
Citations:
PubMed Identifier
31710700
Citation
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 association between CALR allele burden evolution and disease progression. Br J Haematol. 2020 Mar;188(6):935-944. doi: 10.1111/bjh.16276. Epub 2019 Nov 11.
Results Reference
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Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)
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