Italian Observational Study of Patients With Acute Myeloid Leukemia Treated With Small Molecule Inhibiting BCL-2 (AVALON)

Italian Observational Study of Patients With Acute Myeloid Leukemia Treated With Small Molecule Inhibiting B-cell Lymphoma 2 (BCL-2)

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.

Study Overview

• Status: Unknown
• Conditions: Acute Myeloid Leukemia

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.

• Study Type: Observational
• Enrollment (Anticipated): 70

Contacts and Locations

• Study Contact: Name: Oriana Nanni; Phone Number: +390543739266; Email: oriana.nanni@irst.emr.it
• Study Contact Backup: Name: Bernadette Verogen, DR; Phone Number: +390544286058; Email: cc.ubsc@irst.emr.it

Study Locations

• Italy
  • Milano, Italy
    • Not yet recruiting
    • Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico
  • Milano, Italy
    • Recruiting
    • European Institute of Oncology
    • Contact: Elisabetta Todisco, MD
  • Roma, Italy, 00144
    • Recruiting
    • Ospedale Roma S.Eugenio
    • Contact: Elisabetta Abruzzese, MD, PhD
  • CR
    • Cremona, CR, Italy, 26100
      • Recruiting
      • ASST Cremona
      • Contact: Alfredo Molteni
  • CZ
    • Catanzaro, CZ, Italy, 88100
      • Recruiting
      • A.O. Pugliese Ciaccio
      • Contact: Stefano Molica
  • FC
    • Meldola, FC, Italy, 47014
      • Recruiting
      • Irst Irccs
      • Contact: Claudio Cerchione, MD
  • FI
    • Firenze, FI, Italy
      • Recruiting
      • AOU Careggi
      • Contact: Matteo Piccini
  • FR
    • Frosinone, FR, Italy, 03100
      • Recruiting
      • Ospedale F. Spaziani
      • Contact: Vincenza Martini
  • Foggia
    • San Giovanni Rotondo, Foggia, Italy, 71013
      • Recruiting
      • IRCCS Casa Sollievo sofferenza
  • LE
    • Lecce, LE, Italy, 73100
      • Recruiting
      • P.O. Vito Fazzi
  • LT
    • Latina, LT, Italy, 04100
      • Recruiting
      • Ospedale Santa Maria Goretti
      • Contact: Giuseppe Cimino
  • MB
    • Monza, MB, Italy, 20900
      • Recruiting
      • ASST Monza
      • Contact: Monica Fumagalli
  • MI
    • Milano, MI, Italy, 20162
      • Recruiting
      • ASST Grande Ospedale Metropolitano Niguarda
      • Contact: Roberto Cairoli
  • PE
    • Pescara, PE, Italy, 65124
      • Recruiting
      • Ospedale S.Spirito - ASL Pescara
      • Contact: Prassede Salutari
  • PG
    • Perugia, PG, Italy, 06156
      • Recruiting
      • Ospedale Santa Maria della Misericordia
      • Contact: Maria Paola Martelli
  • SA
    • Salerno, SA, Italy, 84131
      • Recruiting
      • AOU San Giovanni di Dio e Ruggi d'Aragona
  • SI
    • Siena, SI, Italy, 53100
      • Recruiting
      • Policlinico Santa Maria alle Scotte
      • Contact: Monica Bocchia
  • TO
    • Torino, TO, Italy, 10126
      • Recruiting
      • AOU Citta della Salute e della Scienza di Torino
      • Contact: Ernesta Audisio, MD
    • Torino, TO, Italy, 10128
      • Recruiting
      • A.O. Ordine Mauriziano
      • Contact: Alessandro Cignetti
  • TV
    • Treviso, TV, Italy, 31100
      • Recruiting
      • Ospedale Ca' Foncello
      • Contact: Michele Gottardi, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events of grade 3 and 4 (NCTCAE version 5.0)	To evaluate the toxicity profile of the therapy with anti-BCL-2 in patients with AML.	up to 18 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	to describe the clinical outcomes in terms of Overall Survival (OS) defined as the number of days between the first study drug administration and death from any cause	18 months
Disease Free Survival (DFS)	to describe Disease Free Survival, defined as the number of days between the first study drug administration and any event including disease progression or death from any cause (both median and restricted mean) whichever occurs first.	18 months
Complete Remission	to describe the Complete Remission (CR), in terms of proportions, in response to therapy	18 months
Complete Remission with incomplete hematologic recovery	to describe Complete Remission with incomplete hematologic recovery (CRi) in terms of proportions, in response to therapy	18 months
Minimal Residual Disease	to describe Minimal Residual Disease, in terms of proportions, in response to therapy	18 months
Incidence of successful bridge to allogeneic transplant	to describe the incidence of successful bridge to allogeneic transplant, in terms of proportions, in response to therapy	18 months
Collection of number of hospitalizations	to describe healthcare resource utilization in terms of number of hospitalizations per patient	18 months
Collection of days of hospitalizations	to describe healthcare resource utilization in terms of days of hospitalizations per patient	18 months
Collection of number of clinical visits per patient	to describe healthcare resource utilization in terms of number of clinical visits per patient	18 months
Collection of number of accesses Day Hospital per patient	to describe healthcare resource utilization in terms of number of accesses in Day Hospital per patient	18 months
Collection of number of accesses in Emergency Care Units per patient	to describe healthcare resource utilization in terms of number of accesses in Emergency Care Units per patient	18 months

Collaborators and Investigators

• Sponsor: Istituto Scientifico Romagnolo per lo Studio e la cura dei Tumori
• Collaborators: European Institute of Oncology
• Investigators: Principal Investigator: Giovanni Martinelli, MD, Irst Irccs

Study record dates

Study Major Dates

• Study Start (Actual): August 23, 2019
• Primary Completion (Anticipated): December 1, 2020
• Study Completion (Anticipated): December 1, 2020

Study Registration Dates

• First Submitted: August 23, 2019
• First Submitted That Met QC Criteria: August 26, 2019
• First Posted (Actual): August 28, 2019

Study Record Updates

• Last Update Posted (Actual): April 21, 2020
• Last Update Submitted That Met QC Criteria: April 20, 2020
• Last Verified: April 1, 2020

More Information

Terms related to this study

• Keywords: AML; clinical practice; BCL-2; anti BCL2 inhibitors
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: IRST204.04

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No