Clinical trial NCT02448095: Retrospective Evaluation of CML Patients in the National Compassionate Program
Official title: Ponatinib Survey: Retrospective Evaluation of CML Patients in the National Compassionate Program According to the 648/96 Decree
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Chronic Myeloid Leukemia
Philadelphia Positive
Keywords: Chronic myeloid leukemia
Philadelphia positive
Compassionate use
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2015-05-19
Enrollment: 38
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: CML Ph+ patients in the Ponatinib compassionate use program

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.

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.

Eligibility 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

Primary outcomes:
measure: Number of adverse events
timeFrame: Maximum of 60 months from treatment start
Secondary outcomes:
measure: Number of patients with hematological complete response
timeFrame: Maximum of 60 months from treatment start
measure: Number of days from treatment start till response
timeFrame: Maximum of 60 months from treatment start
measure: Total number of patients alive
timeFrame: 12 months after patient enrollment in the study
measure: Number of patients in event free survival
timeFrame: 12 months after patient enrollment in the study
measure: Number of patients in progression free survival
timeFrame: 12 months after patient enrollment in the study
measure: Number of mutations
timeFrame: Maximum of 60 months from treatment start
measure: Number of units of Ponatinib administered per units of time
timeFrame: Maximum of 60 months from treatment start
Other outcomes:
Locations:
city: Bari
country: Italy
facility: UO Ematologia con trapianto-Università degli Studi di Bari Aldo Moro
city: Genova
country: Italy
facility: IRCCS_AOU San Martino-IST.Clinica Ematologica
city: Lecce
country: Italy
facility: ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano
city: Monza
country: Italy
facility: Azienda Ospedaliera "S.Gerardo"
city: Novara
country: Italy
facility: S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
city: Orbassano
country: Italy
facility: Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano
city: Pavia
country: Italy
facility: S.C. Ematologia - Fondazione IRCCS Policlinico S. Matteo
city: Reggio Calabria
country: Italy
facility: Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli"
city: Roma
country: Italy
facility: U.O.C. Ematologia - Ospedale S. Eugenio
Study officials:
affiliation: Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano
name: Carmen Fava
role: STUDY_CHAIR
References: