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Retrospective Evaluation of CML Patients in the National Compassionate Program

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ClinicalTrials.gov Identifier: NCT02448095
Recruitment Status : Unknown
Verified October 2018 by Gruppo Italiano Malattie EMatologiche dell'Adulto.
Recruitment status was:  Recruiting
First Posted : May 19, 2015
Last Update Posted : October 23, 2018
Sponsor:
Information provided by (Responsible Party):
Gruppo Italiano Malattie EMatologiche dell'Adulto

Brief Summary:
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.

Condition or disease Intervention/treatment
Chronic Myeloid Leukemia Philadelphia Positive 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.

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Study Type : Observational
Estimated Enrollment : 80 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Ponatinib Survey: Retrospective Evaluation of CML Patients in the National Compassionate Program According to the 648/96 Decree
Study Start Date : December 2015
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018


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




Primary Outcome Measures :
  1. Number of adverse events [ Time Frame: Maximum of 60 months from treatment start ]
    General adverse events and grade 3-4 adverse events according to NCI CTCAE version 4.0


Secondary Outcome Measures :
  1. Number of patients with hematological complete response [ Time Frame: Maximum of 60 months from treatment start ]
    Hematological complete response and related responses, such as major cytogenetic response, complete cytogenetic response and major molecular response.

  2. Number of days from treatment start till response [ Time Frame: Maximum of 60 months from treatment start ]
  3. Total number of patients alive [ Time Frame: 12 months after patient enrollment in the study ]
    Overall survival

  4. Number of patients in event free survival [ Time Frame: 12 months after patient enrollment in the study ]
    Event free survival

  5. Number of patients in progression free survival [ Time Frame: 12 months after patient enrollment in the study ]
    Progression free survival

  6. Number of mutations [ Time Frame: Maximum of 60 months from treatment start ]
    Both at the beginning and at the end of treatment

  7. Number of units of Ponatinib administered per units of time [ Time Frame: Maximum of 60 months from treatment start ]
    Chemotherapy dose intensity represents unit dose of chemotherapy administered per unit time.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
CML Ph+ patients in the Ponatinib compassionate use program
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02448095


Contacts
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Contact: Paola Fazi p.fazi@gimema.it
Contact: Enrico Crea e.crea@gimema.it

Locations
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Italy
UO Ematologia con trapianto-Università degli Studi di Bari Aldo Moro Recruiting
Bari, Italy
Contact: Giorgina Specchia         
Principal Investigator: Giorgina Specchia         
IRCCS_AOU San Martino-IST.Clinica Ematologica Recruiting
Genova, Italy
Contact: Marco Gobbi         
Principal Investigator: Marco Gobbi         
ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE Recruiting
Lecce, Italy
Contact: Nicola Di Renzo         
Principal Investigator: Nicola Di Renzo         
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano Recruiting
Milano, Italy
Contact: Carmen Fava         
Principal Investigator: Carmen Fava         
Azienda Ospedaliera "S.Gerardo" Recruiting
Monza, Italy
Contact: Carlo Gambacorti Passerini         
Principal Investigator: Carlo Gambacorti Passerini         
S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro Recruiting
Novara, Italy
Contact: Gianluca Gaidano         
Principal Investigator: Gianluca Gaidano         
Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano Recruiting
Orbassano, Italy
Contact: Carmen Fava         
S.C. Ematologia - Fondazione IRCCS Policlinico S. Matteo Recruiting
Pavia, Italy
Contact: Ester Orlandi         
Principal Investigator: Ester Orlandi         
Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli" Recruiting
Reggio Calabria, Italy
Contact: Bruno Martino         
Principal Investigator: Bruno Martino         
U.O.C. Ematologia - Ospedale S. Eugenio Recruiting
Roma, Italy
Contact: Elisabetta Abruzzese         
Principal Investigator: Elisabetta Abruzzese         
Sponsors and Collaborators
Gruppo Italiano Malattie EMatologiche dell'Adulto
Investigators
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Study Chair: Carmen Fava Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano
Additional Information:
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Responsible Party: Gruppo Italiano Malattie EMatologiche dell'Adulto
ClinicalTrials.gov Identifier: NCT02448095    
Other Study ID Numbers: CML1214
First Posted: May 19, 2015    Key Record Dates
Last Update Posted: October 23, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:
Chronic myeloid leukemia
Philadelphia positive
Compassionate use
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia
Neoplasms by Histologic Type
Neoplasms
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Ponatinib
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action