Clinical trial NCT04070807: Italian Observational Study of Patients With Acute Myeloid Leukemia Treated With Small Molecule Inhibiting BCL-2
Official title: Italian Observational Study of Patients With Acute Myeloid Leukemia Treated With Small Molecule Inhibiting B-cell Lymphoma 2 (BCL-2)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Acute Myeloid Leukemia
Keywords: AML
BCL-2
anti BCL2 inhibitors
clinical practice
Lead sponsor: Istituto Romagnolo per lo Studio dei Tumori Dino Amadori IRST S.r.l. IRCCS
Sponsor class: OTHER
Collaborators: name: European Institute of Oncology
First posted: 2019-08-28
Enrollment: 70
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: AML according to World Health Organization (WHO) 2016 classification who have received any anti-BCL-2 treatment as single agent or in combination with other drugs from 1 Jan 2015 to 1 Apr 2019 outside clinical trials

Brief summary: This is a multi-center retrospective observational study. Every patient with Acute Myeloid Leukemia (AML) treated with anti-B-cell lymphoma 2 (BCL2) treatment outside clinical trial from 1st January 2015 up to 01 April 2019 may be included in this study. No additional drug/procedures/patient visits in comparison with the usual clinical practice are planned for the study. The decision to treat patient with ant-BCL2 inhibitors is made by the physician based on his clinical judgment, independently from the decision to include the patient in this study.

Detailed description: In phase 1-2 studies, anti BCL-2 treatment has shown evidence of anti-leukemic activity as single agent and in combination and proved to be particularly effective in providing a deep response, with an acceptable safety profile. Since 2015 anti-BCL2 treatment has been available in other indications and in off-label use in Italy.

In this non-interventional retrospective study, toxicity, effectiveness and costs assessment data will be collected from patients with AML, to improve the knowledge about anti-BCL2 treatment in clinical practice. Collecting and analyzing data from a large unbiased patient-set receiving anti-BCL2, would enlarge our knowledge on therapies inhibiting BCL2.

This is a multi-center retrospective observational study. Every patient with AML treated with anti-BCL2 treatment outside clinical trial from 1st January 2015 up to 01 April 2019 may be included in this study. No additional drug/procedures/patient visits in comparison with the usual clinical practice are planned for the study. The decision to treat patient with ant-BCL2 inhibitors is made by the physician based on his clinical judgment, independently from the decision to include the patient in this study. As this study is intended to be purely observational (not interventional), the patient's medical records will be the source of all data to be recorded. No additional procedures/patient visits should be planned in the study with respect to clinical practice.

Clinical data (treatment, survival, adverse events) will be collected for patients enrolled.

Eligibility criteria: Inclusion Criteria:

* Patient with AML according to World Health Organization (WHO) 2016 classification
* Patient who have received any anti-BCL-2 treatment as single agent or in combination with other drugs from 1 Jan 2015 to 1 Apr 2019 outside clinical trials

Exclusion Criteria:

• Patient who have received any anti-BCL-2 treatment within a clinical trials

Primary outcomes:
measure: Incidence of Adverse Events of grade 3 and 4 (NCTCAE version 5.0)
timeFrame: up to 18 months
Secondary outcomes:
measure: Overall Survival
timeFrame: 18 months
measure: Disease Free Survival (DFS)
timeFrame: 18 months
measure: Complete Remission
timeFrame: 18 months
measure: Complete Remission with incomplete hematologic recovery
timeFrame: 18 months
measure: Minimal Residual Disease
timeFrame: 18 months
measure: Incidence of successful bridge to allogeneic transplant
timeFrame: 18 months
measure: Collection of number of hospitalizations
timeFrame: 18 months
measure: Collection of days of hospitalizations
timeFrame: 18 months
measure: Collection of number of clinical visits per patient
timeFrame: 18 months
measure: Collection of number of accesses Day Hospital per patient
timeFrame: 18 months
measure: Collection of number of accesses in Emergency Care Units per patient
timeFrame: 18 months
Other outcomes:
Locations:
city: Cremona
country: Italy
facility: ASST Cremona
state: CR
city: Catanzaro
country: Italy
facility: A.O. Pugliese Ciaccio
state: CZ
city: Meldola
country: Italy
facility: Irst Irccs
state: FC
city: Firenze
country: Italy
facility: AOU Careggi
state: FI
city: San Giovanni Rotondo
country: Italy
facility: IRCCS Casa Sollievo sofferenza
state: Foggia
city: Frosinone
country: Italy
facility: Ospedale F. Spaziani
state: FR
city: Lecce
country: Italy
facility: P.O. Vito Fazzi
state: LE
city: Latina
country: Italy
facility: Ospedale Santa Maria Goretti
state: LT
city: Monza
country: Italy
facility: Asst Monza
state: MB
city: Milano
country: Italy
facility: ASST Grande Ospedale Metropolitano Niguarda
state: MI
city: Pescara
country: Italy
facility: Ospedale S.Spirito - ASL Pescara
state: PE
city: Perugia
country: Italy
facility: Ospedale Santa Maria della Misericordia
state: PG
city: Salerno
country: Italy
facility: AOU San Giovanni di Dio e Ruggi D'Aragona
state: SA
city: Siena
country: Italy
facility: Policlinico Santa Maria alle Scotte
state: SI
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
state: TO
city: Treviso
country: Italy
facility: Ospedale Ca' Foncello
state: TV
city: Milano
country: Italy
facility: European Institute of Oncology
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda OSPEDALE MAGGIORE POLICLINICO
city: Roma
country: Italy
facility: Ospedale Roma S.Eugenio
Study officials:
affiliation: IRST IRCCS
name: Giovanni Martinelli, MD
role: PRINCIPAL_INVESTIGATOR
References: