Clinical trial NCT05193448: A Non-interventional Ambispective Real-world Cohort of rEfractory and reLapsed (R/R) FLT3 Mutated Acute MyEloid Leukemia (AML) Patients Treated With Gilteritinib in FrANCE
Official title: A Non-interventional Ambispective Real-world Cohort of rEfractory and reLapsed FLT3 Mutated Acute MyEloid Leukemia Patients Treated With Gilteritinib in FrANCE
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Refractory AML
Relapsed Adult AML
FLT3-TKD Mutation
FLT3-ITD
Lead sponsor: French Innovative Leukemia Organisation
Sponsor class: OTHER
Collaborators: name: Acute Leukemia French Association
First posted: 2022-01-14
Enrollment: 177
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: PROBABILITY_SAMPLE
Study population: ≥18 years of age FLT3-mutated R/R AML as defined by the WHO Classification between the periods of March 1st, 2019 to September 30, 2021 allowing 6-months follow-up for the last patient included (March, 19 2019 to September 18, 2019 for French "ATU nominative"; September 18, 2019 to January 08, 2020 for French "ATU de cohorte"; January 08, 2020 to March 30, 2021 for "post ATU" period) treated in virtually all AML center in France

Brief summary: Gilteritinib is available in early access in France through Temporary Authorisation of Use (or ATU program) since March 2019. The ATU program reflects a real-life treatment situation and the related clinical data would help to better understand the benefit/risk profile of gilteritinib and to better document gilteritinib efficacy and safety in patients who received midostaurine in First Line (1L) setting.

The main objective is to describe gilteritinib effectiveness in FLT3 (Fms Related Tyrosine Kinase 3) -mutated AML patients in Refractory/Relapsed(R/R) situation treated in the context of early access program to gilteritinib in France through Temporary Authorisation of Use, the so-called ATU program, and the post ATU period from marketing authorisation to launch when reimbursement and price are published.


Eligibility criteria: Inclusion Criteria:

* Adult patients ≥ 18 years at AML diagnosis
* Patients that started gilteritinib during ATU and post-ATU period from 19th March 2019 to 30th March2021
* Patients diagnosed with refractory or relapsed AML as defined by the World Health Organization (WHO) Classification
* Patients with FLT3 genetic testing performed at diagnosis and/or at R/R (if available)
* Gilteritinib with or without other drug (chemotherapy, hypomethylating agent, hydroxyurea, etc.)

Exclusion Criteria:

* Newly diagnosed AML patients
* Participant opposed to the collection and analysis of their medical data
* Prescription of gilteritinib out of the scope of its marketing authorisation approval such as post HSCT maintenance in patients in first complete remission after intensive chemotherapy
* persons placed in curatorship,guardianship or guardianship orders

Primary outcomes:
measure: Gilteritinib effectiveness in FLT3-mutated AML patients in R/R situation
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
Locations:
city: Amiens
country: France
facility: Amiens CHU
city: Angers
country: France
facility: Angers CHU
city: Avignon
country: France
facility: Avignon CH
city: Bayonne
country: France
facility: Bayonne CH
city: Besançon
country: France
facility: Besançon CHU
city: Brest
country: France
facility: Brest CHU
city: Caen
country: France
facility: Caen CHU
city: Cergy-Pontoise
country: France
facility: CERGY PONTOISE - CH René Dubos
city: Clermont-Ferrand
country: France
facility: CHU Estaing
city: Corbeil-Essonnes
country: France
facility: Corbeil-Essonnes - Ch Sud Francilien
city: Créteil
country: France
facility: Créteil CHU HENRI MONDOR
city: Dijon
country: France
facility: Dijon CHU
city: Grenoble
country: France
facility: Grenoble CHU
city: Le Mans
country: France
facility: Le Mans CH
city: Limoges
country: France
facility: Limoges CHU
city: Lyon
country: France
facility: Lyon sud CHU
city: Marseille
country: France
facility: Marseille IPC
city: Meaux
country: France
facility: Meaux CH de l'Est francilien
city: Metz
country: France
facility: METZ-THIONVILLE CHR- Hôpital de Mercy
city: Montpellier
country: France
facility: Montpellier - Chu Saint Eloi
city: Nantes
country: France
facility: Nantes CHU
city: Nice
country: France
facility: Nice CHU
city: Nîmes
country: France
facility: Nimes CHU
city: Paris
country: France
facility: Paris La Pitié salpetrière
city: Paris
country: France
facility: Paris Necker
city: Paris
country: France
facility: Paris Saint Louis
city: Pessac
country: France
facility: Bordeaux CHU
city: Reims
country: France
facility: Reims CHU
city: Rennes
country: France
facility: Rennes CHU
city: Roubaix
country: France
facility: roubaix CH
city: Saint-Priest-en-Jarez
country: France
facility: Institut de Cancérologie Lucien Neuwirth
city: Saint-Quentin
country: France
facility: Saint Quentin CH
city: Toulouse
country: France
facility: Toulouse - IUCT Oncopole - Service d'Hématologie
city: Tours
country: France
facility: Tours CHU
city: Troyes
country: France
facility: Troyes CH
city: vandoeuvre les Nancy
country: France
facility: Nancy CHU
city: Versailles
country: France
facility: Versailles CH
city: Villejuif
country: France
facility: Villejuif IGR
Study officials:
References: