Retrospective Evaluation of CML Patients in the National Compassionate Program

Ponatinib Survey: Retrospective Evaluation of CML Patients in the National Compassionate Program According to the 648/96 Decree

This observational study aims at assessing the tolerability and safety profiles of Ponatinib, a drug used for Chronic Myeloid Leukemia patients who are Philadelphia positive. This drug is used during the chronic phase of the disease, according to the Italian national compassionate law 648/96.

Study Overview

• Status: Terminated
• Conditions: Chronic Myeloid Leukemia; Philadelphia Positive
• Intervention / Treatment: Drug: Ponatinib

Detailed Description

This observational study aims at assessing the tolerability and safety profiles of Ponatinib, a drug used for Chronic Myeloid Leukemia patients who are Philadelphia positive. This drug is used during the chronic phase of the disease, according to the Italian national compassionate law 648/96.

• Study Type: Observational
• Enrollment (Actual): 38

Contacts and Locations

Study Locations

• Italy
  • Bari, Italy
    • UO Ematologia con trapianto-Università degli Studi di Bari Aldo Moro
  • Genova, Italy
    • IRCCS_AOU San Martino-IST.Clinica Ematologica
  • Lecce, Italy
    • ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
  • Milano, Italy
    • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano
  • Monza, Italy
    • Azienda Ospedaliera "S.Gerardo"
  • Novara, Italy
    • S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
  • Orbassano, Italy
    • Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano
  • Pavia, Italy
    • S.C. Ematologia - Fondazione IRCCS Policlinico S. Matteo
  • Reggio Calabria, Italy
    • Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli"
  • Roma, Italy
    • U.O.C. Ematologia - Ospedale S. Eugenio

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

CML Ph+ patients in the Ponatinib compassionate use program

Description

Inclusion Criteria:

• CML Ph+ patients in chronic phase
• 18 years old or older
• Patients have received either Dasatinib or Nilotinib and resulted resistant or not tolerant to the drugs or have developed the T3151 mutation
• Patients have started Ponatinib at least 12 months before registration
• Informed consent signed at registration

Exclusion Criteria:

• CML Ph+ patients in accelerated or blast phase with acute lymphoblastic leukemia

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
CML ph+ patients; Chronic myeloid leukemia patients who are philadelphia positive	Drug: Ponatinib; Observation of tolerability and safety profiles

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse events	General adverse events and grade 3-4 adverse events according to NCI CTCAE version 4.0	Maximum of 60 months from treatment start

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with hematological complete response	Hematological complete response and related responses, such as major cytogenetic response, complete cytogenetic response and major molecular response.	Maximum of 60 months from treatment start
Number of days from treatment start till response		Maximum of 60 months from treatment start
Total number of patients alive	Overall survival	12 months after patient enrollment in the study
Number of patients in event free survival	Event free survival	12 months after patient enrollment in the study
Number of patients in progression free survival	Progression free survival	12 months after patient enrollment in the study
Number of mutations	Both at the beginning and at the end of treatment	Maximum of 60 months from treatment start
Number of units of Ponatinib administered per units of time	Chemotherapy dose intensity represents unit dose of chemotherapy administered per unit time.	Maximum of 60 months from treatment start

Collaborators and Investigators

• Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
• Investigators: Study Chair: Carmen Fava, Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano

Publications and helpful links

Helpful Links

• GIMEMA Foundation website

Study record dates

Study Major Dates

• Study Start (Actual): December 22, 2015
• Primary Completion (Actual): July 17, 2018
• Study Completion (Actual): July 17, 2018

Study Registration Dates

• First Submitted: May 15, 2015
• First Submitted That Met QC Criteria: May 15, 2015
• First Posted (Estimate): May 19, 2015

Study Record Updates

• Last Update Posted (Actual): October 26, 2022
• Last Update Submitted That Met QC Criteria: October 24, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Keywords: Chronic myeloid leukemia; Compassionate use; Philadelphia positive
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Protein Kinase Inhibitors; Ponatinib
• Other Study ID Numbers: CML1214

Plan for Individual participant data (IPD)

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