Retrospective Evaluation of CML Patients in the National Compassionate Program

• Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT02448095
• Collaborator: (none)
• Enrollment: 80
• Locations: 10
• Anticipated Duration (Months): 83.3
• Patients Per Site: 8
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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	Phase
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.

Study Design

• Study Type: Observational
• Anticipated 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
• Actual Study Start Date: Dec 22, 2015
• Anticipated Primary Completion Date: Dec 1, 2022
• Anticipated Study Completion Date: Dec 1, 2022

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Number of adverse events [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 [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 [Maximum of 60 months from treatment start]

3. Total number of patients alive [12 months after patient enrollment in the study]

   Overall survival

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

   Event free survival

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

   Progression free survival

6. Number of mutations [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 [Maximum of 60 months from treatment start]

   Chemotherapy dose intensity represents unit dose of chemotherapy administered per unit time.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

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

Sponsors and Collaborators

• 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

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• GIMEMA Foundation website

Publications

None provided.

• Responsible Party: Gruppo Italiano Malattie EMatologiche dell'Adulto
• ClinicalTrials.gov Identifier: NCT02448095
• Other Study ID Numbers: CML1214
• First Posted: May 19, 2015
• Last Update Posted: Jan 5, 2022
• Last Verified: Jan 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• 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: Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Ponatinib

Study Results

No Results Posted as of Jan 5, 2022