Clinical trial NCT03547258: Italian Non-Interventional Study of FLT3 Mutated AML Patients
Official title: Italian Non-Interventional Study of FMS-like Tyrosine Kinase (FLT3) Mutated Acute Myeloid Leukemia (AML) Patients
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: AML
FLT3-ITD Mutation
FLT3-TKD Mutation
Keywords: AML
Italian observational study
FLT3-ITD Mutation
FLT3-TKD Mutation
Lead sponsor: Istituto Romagnolo per lo Studio dei Tumori Dino Amadori IRST S.r.l. IRCCS
Sponsor class: OTHER
First posted: 2018-06-06
Enrollment: 800
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: All consecutive patient with FLT3 mutated AML documented at diagnosis or at the time of relapse/refractory state treated in the participating center. With regards to the retrospective collection, data from the eligible and acconsenting patients will be retrieved since 2012, that is, when the evaluation of FLT3 mutation was almost routine in the centers considered in the study.

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.

Eligibility criteria: Inclusion criteria:

1. AML patients
2. Male or Female
3. Aged ≥ 18 years
4. FLT3 mutations (ITD or TKD) positive tests performed at diagnosis or at relapse.
5. Participant is willing and able to give informed consent for participation in the study.

Exclusion criteria:

1. To be currently involved in experimental clinical protocol, or have been treated with experimental drugs are not exclusion criteria.

Primary outcomes:
measure: percentage of patients FLT3 negative at diagnosis who relapse FLT3 positive;
timeFrame: up to 24 months
measure: percentage of patients FLT3 positive at diagnosis who relapse FLT3 negative
timeFrame: up to 24 months
Secondary outcomes:
measure: objective overall response rate (ORR)
timeFrame: up to 24 months
measure: disease-free survival (DFS)
timeFrame: up to 24 months
measure: overall survival (OS)
timeFrame: up to 24 months
measure: Percentage of AML patients with specific types of FLT3 mutations
timeFrame: up to 24 months
measure: distribution of specific FLT3 mutations in AML patients
timeFrame: up to 24 months
measure: frequency of the different methods used to evaluate (Minimal residual disease) MRD
timeFrame: up to 24 months
measure: FLT3-ITD allelic ratio
timeFrame: up to 24 months
measure: transplantation percentage
timeFrame: up to 24 months
measure: evaluation of modifications in terms of quality of life (QoL) of patients with FLT3 mutated AML
timeFrame: up to 24 months
measure: retrospective collection of surrogate measures of QoL
timeFrame: up to 24 months
Other outcomes:
Locations:
city: Ancona
country: Italy
facility: AOU Ospedali riuniti di Ancona
state: AN
city: Bari
country: Italy
facility: AOU Policlinico Bari - Ematologia
state: BA
city: Bologna
country: Italy
facility: Università di Bologna - DIMES
state: BO
city: Brescia
country: Italy
facility: ASST Spedali di Brescia
state: BS
city: Cagliari
country: Italy
facility: Azienda Ospedaliera G. Brotzu
state: CA
city: Cuneo
country: Italy
facility: Azienda Ospedaliera S. Croce e Carle
state: CN
city: Catanzaro
country: Italy
facility: Ematologia - Azienda Ospedaliera "Pugliese Ciaccio" di Catanzaro
state: CZ
city: Meldola
country: Italy
facility: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IRST)
state: FC
city: Cona
country: Italy
facility: AOU Universitaria Arcispedale Sant'Anna
state: Ferrara
city: San Giovanni Rotondo
country: Italy
facility: IRCCS Casa sollievo della sofferenza
state: FG
city: Firenze
country: Italy
facility: AOU Careggi
state: FI
city: Lecce
country: Italy
facility: Ospedale Vito Fazzi di Lecce
state: LE
city: Messina
country: Italy
facility: AOU Policlinico Gaetano Martino
state: ME
city: Milano
country: Italy
facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
state: MI
city: Milano
country: Italy
facility: Ospedale San Raffaele
state: MI
city: Milano
country: Italy
facility: Istituto Europeo di Oncologia
state: MI
city: Palermo
country: Italy
facility: Ospedali Riuniti Villa Sofia-Cervello
state: PA
city: Piacenza
country: Italy
facility: Ospedale "Guglielmo da Saliceto"
state: PC
city: Rionero In Vulture
country: Italy
facility: Irccs Crob
state: Potenza
city: Parma
country: Italy
facility: Azienda Ospedaliero-Universitaria di Parma - Ematologia e CTMO
state: PR
city: Ravenna
country: Italy
facility: UO Ematologia, Ospedale S.Maria delle Croci
state: RA
city: Reggio Calabria
country: Italy
facility: Grande Ospedale Metropolitano
state: RC
city: Rimini
country: Italy
facility: Ospedale infermi di Rimini
state: RN
city: Salerno
country: Italy
facility: AOU San Giovannidi Dio e Ruggi - ematologia
state: SA
city: Siena
country: Italy
facility: AOC di Ematologia - AOU Senese
state: SI
city: Orbassano
country: Italy
facility: Università di Torino - Ospedale San Luigi Gonzaga
state: TO
city: Torino
country: Italy
facility: AOU Città della Salute e della Scienza di Torino
state: TO
city: Torino
country: Italy
facility: A.O. Ordine Mauriziano Torino
state: TO
city: Udine
country: Italy
facility: Divisione di Ematologia - Università di Udine
state: UD
city: Napoli
country: Italy
facility: AO Universitaria Federico II
city: Novara
country: Italy
facility: AOU Maggiore della carità
city: Perugia
country: Italy
facility: AOU Santa Maria della Misericordia - Ematologia
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli
Study officials:
affiliation: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IRST)
name: Giovanni Martinelli, MD
role: STUDY_CHAIR
References: