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Italian Non-Interventional Study of FLT3 Mutated AML Patients

Conditions
AML
FLT3-TKD Mutation
FLT3-ITD Mutation
Interventions
Genetic: Clinical and Molecular data collection
Registration Number
NCT03547258
Lead Sponsor
Istituto Scientifico Romagnolo per lo Studio e la cura dei Tumori
Brief Summary

This is an observational study involving a retrospective and prospective collection of clinical and molecular data regarding patients with AML with FLT3+ mutations

Detailed Description

This is an observational study involving a retrospective and prospective collection of clinical and molecular data. Patients will follow their regular diagnostic and clinical practice. Thus, no additional procedure/blood withdrawal will be performed.

The study will be conducted as follows:

1. Retrospective phase: clinical and molecular data of AML patients with FLT3+ mutations detected at diagnosis or at any refractory/relapse state will be collected.

2. Prospective phase: clinical and molecular data of each new FLT3+ AML patient identified in participating centers at diagnosis or at any refractory/relapse state will be collected prospectively. Every effort will be done to include all consecutive patients, in order to avoid selection bias.

For patients with a mutation found at the time of disease relapse, any effort will be done to collect all the clinical and molecular information since the time of diagnosis.

The Primary objective of this study is to analyze how FLT3 mutational status evolve during the management of the disease looking at the percentage of patients with no FLT3 mutations at diagnosis who relapse with a new FLT3 mutation detected, and the percentage of FLT3 positive AML patients that after having obtained a Complete Remission relapse with FLT3 negative.

The secondary objective of the study is to investigate the association between different FLT3 mutations and the clinical, molecular and biological information.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
800
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
AML patientsClinical and Molecular data collectionClinical and Molecular data collection of AML Patients with FLT3 mutations (ITD or TKD)
Primary Outcome Measures
NameTimeMethod
percentage of patients FLT3 negative at diagnosis who relapse FLT3 positive;up to 24 months

description of how the FLT3 mutational status changes during the course and management of the disease

percentage of patients FLT3 positive at diagnosis who relapse FLT3 negativeup to 24 months

description of how the FLT3 mutational status changes during the course and management of the disease

Secondary Outcome Measures
NameTimeMethod
objective overall response rate (ORR)up to 24 months

the objective overall response rate (ORR) defined as the proportion of patients with a partial or response (CR, CRi, CRp) response, to initial treatment and in case of salvage;

disease-free survival (DFS)up to 24 months

disease-free survival (DFS) after first CR and after CR2/CR3, if applicable, defined as the time since CR (or CR2 or CR3) to disease relapse or death for any cause, whichever occurs first.

Percentage of AML patients with specific types of FLT3 mutationsup to 24 months

Percentage of AML patients with specific types of FLT3 mutations, at initial diagnosis and at disease relapse

retrospective collection of surrogate measures of QoLup to 24 months

the surrogate measures of QoL , as the number and days of hospitalizations per patient, number of clinical visits per patient, number of access in Day Hospital and Emergency Care Units per patient, and the use of antalgic drugs and neuro-active drugs, will be expressed in terms of mean values per patient or through proportions

overall survival (OS)up to 24 months

overall survival (OS) defined as the time since date of diagnosis until death for any cause or the last available patient contact.

distribution of specific FLT3 mutations in AML patientsup to 24 months

distribution of specific FLT3 mutations in AML patients according to: age, WBC, LDH, cytogenetics, NPM1, CEBPA alterations, IDH1/2, tp53, DNMT3A, secondary vs de novo AML;

frequency of the different methods used to evaluate (Minimal residual disease) MRDup to 24 months

the frequency of the different methods used to evaluate MRD such as, wt1 ratio or the fusion transcript level by Reverse transcription polymerase chain reaction (RT-PCR); percentage of patients performing FLT3 ITD analysis by Next Generation Sequencing (NGS);

transplantation percentageup to 24 months

percentage of FLT3 mutated AML patients undergoing transplantation

evaluation of modifications in terms of quality of life (QoL) of patients with FLT3 mutated AMLup to 24 months

scores for each patient and each scale as well as a summary QoL score will be computed according to the EORTC QLQ-C3 (quality of life questionnaire) manual

FLT3-ITD allelic ratioup to 24 months

FLT3-ITD allelic ratio defined as the ratio of the area under the curve of mutant and wild type alleles (mutant/totalFLT3) obtained after Fragment analysis for FLT3-ITD;

Trial Locations

Locations (33)

AOU Ospedali riuniti di Ancona

🇮🇹

Ancona, AN, Italy

AOU Policlinico Bari - Ematologia

🇮🇹

Bari, BA, Italy

Università di Bologna - DIMES

🇮🇹

Bologna, BO, Italy

Azienda Ospedaliera S. Croce e Carle

🇮🇹

Cuneo, CN, Italy

ASST Spedali di Brescia

🇮🇹

Brescia, BS, Italy

Azienda Ospedaliera G. Brotzu

🇮🇹

Cagliari, CA, Italy

Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IRST)

🇮🇹

Meldola, FC, Italy

IRCCS Casa sollievo della sofferenza

🇮🇹

San Giovanni Rotondo, FG, Italy

Ematologia - Azienda Ospedaliera "Pugliese Ciaccio" di Catanzaro

🇮🇹

Catanzaro, CZ, Italy

AOU Careggi

🇮🇹

Firenze, FI, Italy

Ospedale Vito Fazzi di Lecce

🇮🇹

Lecce, LE, Italy

Ospedale San Raffaele

🇮🇹

Milano, MI, Italy

Istituto Europeo di Oncologia

🇮🇹

Milano, MI, Italy

Ospedali Riuniti Villa Sofia-Cervello

🇮🇹

Palermo, PA, Italy

Ospedale "Guglielmo da Saliceto"

🇮🇹

Piacenza, PC, Italy

UO Ematologia, Ospedale S.Maria delle Croci

🇮🇹

Ravenna, RA, Italy

Azienda Ospedaliero-Universitaria di Parma - Ematologia e CTMO

🇮🇹

Parma, PR, Italy

Grande Ospedale Metropolitano

🇮🇹

Reggio Calabria, RC, Italy

Ospedale infermi di Rimini

🇮🇹

Rimini, RN, Italy

AOU San Giovannidi Dio e Ruggi - ematologia

🇮🇹

Salerno, SA, Italy

Università di Torino - Ospedale San Luigi Gonzaga

🇮🇹

Orbassano, TO, Italy

AOC di Ematologia - AOU Senese

🇮🇹

Siena, SI, Italy

A.O. Ordine Mauriziano Torino

🇮🇹

Torino, TO, Italy

AOU Città della Salute e della Scienza di Torino

🇮🇹

Torino, TO, Italy

Divisione di Ematologia - Università di Udine

🇮🇹

Udine, UD, Italy

AO Universitaria Federico II

🇮🇹

Napoli, Italy

Fondazione Policlinico Universitario Agostino Gemelli

🇮🇹

Roma, Italy

AOU Santa Maria della Misericordia - Ematologia

🇮🇹

Perugia, Italy

AOU Maggiore della carità

🇮🇹

Novara, Italy

AOU Universitaria Arcispedale Sant'Anna

🇮🇹

Cona, Ferrara, Italy

Irccs Crob

🇮🇹

Rionero In Vulture, Potenza, Italy

AOU Policlinico Gaetano Martino

🇮🇹

Messina, ME, Italy

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

🇮🇹

Milano, MI, Italy

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