NCT02448095

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
38

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Dec 2015

Typical duration for all trials

Geographic Reach
1 country

10 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 15, 2015

Completed
4 days until next milestone

First Posted

Study publicly available on registry

May 19, 2015

Completed
7 months until next milestone

Study Start

First participant enrolled

December 22, 2015

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 17, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 17, 2018

Completed
Last Updated

October 26, 2022

Status Verified

October 1, 2022

Enrollment Period

2.6 years

First QC Date

May 15, 2015

Last Update Submit

October 24, 2022

Conditions

Chronic Myeloid LeukemiaPhiladelphia Positive

Keywords

Chronic myeloid leukemiaPhiladelphia positiveCompassionate use

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (7)

  • Number of patients with hematological complete 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

    12 months after patient enrollment in the study

  • Number of patients in event free survival

    12 months after patient enrollment in the study

  • Number of patients in progression free survival

    12 months after patient enrollment in the study

  • +2 more secondary outcomes

Study Arms (1)

CML ph+ patients

Chronic myeloid leukemia patients who are philadelphia positive

Drug: Ponatinib

Interventions

PonatinibDRUG

Observation of tolerability and safety profiles

CML ph+ patients

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

CML Ph+ patients in the Ponatinib compassionate use program

You may qualify if:

  • CML Ph+ patients in chronic phase
  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

UO Ematologia con trapianto-Università degli Studi di Bari Aldo Moro

Bari, Italy

Location

IRCCS_AOU San Martino-IST.Clinica Ematologica

Genova, Italy

Location

ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE

Lecce, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano

Milan, Italy

Location

Azienda Ospedaliera "S.Gerardo"

Monza, Italy

Location

S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro

Novara, Italy

Location

Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano

Orbassano, Italy

Location

S.C. Ematologia - Fondazione IRCCS Policlinico S. Matteo

Pavia, Italy

Location

Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli"

Reggio Calabria, Italy

Location

U.O.C. Ematologia - Ospedale S. Eugenio

Roma, Italy

Location

Related Links

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Interventions

ponatinib

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsMyeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Carmen Fava

    Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano

    STUDY CHAIR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 15, 2015

First Posted

May 19, 2015

Study Start

December 22, 2015

Primary Completion

July 17, 2018

Study Completion

July 17, 2018

Last Updated

October 26, 2022

Record last verified: 2022-10

Data Sharing

IPD Sharing
Will not share

Locations

IT(10)