Chronic Myeloid LeukemiaNCT02448095
Retrospective Evaluation of CML Patients in the National Compassionate Program
An observational study of Chronic Myeloid Leukemia and Philadelphia Positive, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 10 sites in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloid Leukemia, Philadelphia Positive
- What is being tested?
- Ponatinib
- How many people
- 38 participants
- Where
- 10 sitesItaly
- Study dates
- 22 Dec 2015to 17 Jul 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: CML Ph+ patients in the Ponatinib compassionate use program
Who may be able to join
- 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
Who cannot join
- CML Ph+ patients in accelerated or blast phase with acute lymphoblastic leukemia
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
Italy10 sites
UO Ematologia con trapianto-Università degli Studi di Bari Aldo Moro
Bari
IRCCS_AOU San Martino-IST.Clinica Ematologica
Genova
ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
Lecce
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano
Milan
Azienda Ospedaliera "S.Gerardo"
Monza
S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
Novara
Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano
Orbassano
S.C. Ematologia - Fondazione IRCCS Policlinico S. Matteo
Pavia
Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli"
Reggio Calabria
U.O.C. Ematologia - Ospedale S. Eugenio
Roma
About this study
Official title: 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.
Read the 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.
- Conditions
- Chronic Myeloid Leukemia, Philadelphia Positive
- Being tested
- Ponatinib
- Keywords
- Chronic myeloid leukemia, Philadelphia positive, Compassionate use
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PonatinibDrug
Observation of tolerability and safety profiles
| Study group | Role | What they receive |
|---|---|---|
| CML ph+ patients | Chronic myeloid leukemia patients who are philadelphia positive Receives: Drug: Ponatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events
General adverse events and grade 3-4 adverse events according to NCI CTCAE version 4.0
Measured: Maximum of 60 months from treatment start
Secondary outcomes (7)
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.
Measured: Maximum of 60 months from treatment start
Number of days from treatment start till response
Measured: Maximum of 60 months from treatment start
Total number of patients alive
Overall survival
Measured: 12 months after patient enrollment in the study
Number of patients in event free survival
Event free survival
Measured: 12 months after patient enrollment in the study
Number of patients in progression free survival
Progression free survival
Measured: 12 months after patient enrollment in the study
Number of mutations
Both at the beginning and at the end of treatment
Measured: 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.
Measured: Maximum of 60 months from treatment start
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2015 (estimated)
- Study start22 Dec 2015
- Primary completion17 Jul 2018
- Study completion17 Jul 2018
Why it stopped: lack of interest
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'Adulto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Carmen Fava, Study chair, Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02448095
- Sponsor's protocol ID
- CML1214
- Status last verified
- Oct 2022
- First submitted
- 15 May 2015
- Last updated
- 26 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ponatinib Survey: Retrospective Evaluation of CML Patients in the National Compassionate Program According to the 648/96 Decree
- ClinicalTrials.gov ID
- NCT02448095
- Acronym / sponsor ID
- CML1214
- Conditions
- Chronic Myeloid Leukemia, Philadelphia Positive
- Keywords
- Chronic myeloid leukemia, Philadelphia positive, Compassionate use
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: lack of interest
- Enrolment
- 38 participants (actual)
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'AdultoUniversity, hospital or other
- Study start
- 22 Dec 2015 (actual)
- Primary completion
- 17 Jul 2018 (actual)
- Study completion
- 17 Jul 2018 (actual)
- First posted
- 19 May 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CML ph+ patients | – | Drug: PonatinibChronic myeloid leukemia patients who are philadelphia positive |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- CML Ph+ patients in the Ponatinib compassionate use program
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (1 listed)
- CML Ph+ patients in accelerated or blast phase with acute lymphoblastic leukemia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PonatinibObservation of tolerability and safety profiles | Drug | CML ph+ patients |
Primary outcome
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
7 secondary outcomes
Number of patients with hematological complete response
Time frame: Maximum of 60 months from treatment start
Number of days from treatment start till response
Time frame: Maximum of 60 months from treatment start
Total number of patients alive
Time frame: 12 months after patient enrollment in the study
Number of patients in event free survival
Time frame: 12 months after patient enrollment in the study
Number of patients in progression free survival
Time frame: 12 months after patient enrollment in the study
Number of mutations
Time frame: Maximum of 60 months from treatment start
Number of units of Ponatinib administered per units of time
Time frame: Maximum of 60 months from treatment start
Results and safety
The study was stopped early (reason given: lack of interest). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2022.
- GIMEMA Foundation websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carmen Fava study chair
Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Chronic Myeloid Leukemia and Philadelphia Positive, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 10 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: lack of interest). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse events (time frame: Maximum of 60 months from treatment start). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2022.