NCT04709731

Brief Summary

This study will address the therapeutic activity and the safety/biological profile of Ponatinib when used as third line therapy of Chronic Myeloid Leukemia in Chronic Phase after the only two TKIs known for their cardiovascular safety, i.e. Imatinib and Bosutinib.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
50

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Feb 2021

Typical duration for phase_2

Geographic Reach
1 country

13 active sites

Status
unknown

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

January 12, 2021

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 14, 2021

Completed
18 days until next milestone

Study Start

First participant enrolled

February 1, 2021

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2024

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2025

Completed
Last Updated

February 3, 2021

Status Verified

January 1, 2021

Enrollment Period

3.7 years

First QC Date

January 12, 2021

Last Update Submit

January 31, 2021

Conditions

Keywords

Chronic Myeloid LeukemiaChronic PhasePonatinibBCR-ABL Positive (BCR-ABL+)Philadelphia Chromosome Positive (Ph+)Imatinib and/or Bosutinib IntoleranceImatinib and/or Bosutinib Resistance

Outcome Measures

Primary Outcomes (1)

  • Exposure adjusted Rate of Arterial Occlusive Events and Serious Arterial Occlusive Events at 1 year after study treatment initiation of each patient

    Exposure adjusted Rate of Arterial Occlusive Events (AOE) and Serious AOE (SOE) at 1 year after study treatment initiation of each patient

    1 year

Study Arms (1)

Total Patients

EXPERIMENTAL

Intolerant Group Ponatinib 15 mg tablet, taken orally once daily (QD) Resistant Group Ponatinib 30 mg tablet, taken orally once daily (QD) The dose will be reduced to 15mg once daily (QD) as soon as a Complete Cytogenetic Response will be obtained. In those patients showing Major Molecular Response or better, the dose could be further reduced to 15MG every other day (EOD), due to the prolonged half-life of the drug.

Drug: Ponatinib 15mg QDDrug: Ponatinib 30mg QD

Interventions

Ponatinib 15 mg tablet, taken orally once daily

Also known as: ICLUSIG, AP24534
Total Patients

Ponatinib 30 mg tablet, taken orally once daily

Also known as: ICLUSIG, AP24534
Total Patients

Eligibility Criteria

Age18 Years - 99 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Signed and dated Informed Consent approved by Local Ethical Committee before any protocol-specific screening procedures.
  • CML diagnosis, Chronic Phase (CP), treated with imatinib and bosutinib. Previous treatment with dasatinib or nilotinib will not be allowed.
  • Resistant or intolerant to imatinib and/or bosutinib.
  • Able to take oral therapy.
  • Female or male, 18 years of age or older.
  • ECOG performance status 0-2.
  • Minimum life expectancy of 3 months or more.
  • Adequate organ function as defined by the following criteria:
  • Serum aspartate transaminase (AST) and serum alanine transaminase (ALT) ≤ 2.5 x upper limit of normal (ULN) or AST and ALT ≤ 5 x ULN if liver function abnormalities are due to underlying malignancy
  • Total serum bilirubin ≤ 1.5 x ULN (except patients with documented Gilbert's syndrome)
  • Creatinine ≤ 1.5 x ULN
  • Prothrombin time (PT) \< 1.5 × ULN
  • Lipase ≤ 1.5 × ULN for institution
  • Amylase ≤ 1.5 × ULN for institution
  • Normal QTcF interval on screening electrocardiogram (ECG) evaluation, defined as QTcF of ≤ 450 ms in males or ≤ 470 ms in females.
  • +3 more criteria

You may not qualify if:

  • Current treatment on another therapeutic clinical trial.
  • Received TKI therapy within 7 days prior to receiving the first dose of ponatinib, or have not recovered (\> grade 1 by NCI CTCAE, v. 4.0) from AEs (except alopecia) due to agents previously administered.
  • Underwent autologous or allogeneic stem cell transplant \< 60 days prior to receiving the first dose of ponatinib; any evidence of on-going graft versus-host disease (GVHD), or GVHD requiring immunosuppressive therapy.
  • Take medications that are known to be associated with Torsades de Pointes.
  • Require concurrent treatment with immunosuppressive agents, other than corticosteroids prescribed for a short course of therapy.
  • Have previously been treated with ponatinib.
  • Have significant or active cardiovascular disease, specifically including, but not restricted to:
  • Myocardial infarction within 3 months prior to first dose of ponatinib,
  • History of clinically significant atrial arrhythmia or any ventricular arrhythmia,
  • Unstable angina within 3 months prior to first dose of ponatinib,
  • Congestive heart failure within 3 months prior to first dose of ponatinib.
  • Have a significant bleeding disorder unrelated to CML or Ph+ ALL.
  • Have a history of pancreatitis or alcohol abuse.
  • Have uncontrolled hypertriglyceridemia (triglycerides \> 450 mg/dL).
  • Have malabsorption syndrome or other gastrointestinal illness that could affect absorption of orally administered ponatinib.
  • +5 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

Presidio Ospedaliero "Oncologico Businco" - Cagliari (CA)

Cagliari, 09121, Italy

Location

AOU "Policlinico Vittorio Emanuele" - Catania (CT)

Catania, 95124, Italy

Location

Ospedale San Gerardo - Monza (MB)

Milan, 20090, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Milano (MI)

Milan, 20122, Italy

Location

Azienda Ospedaliera Universitaria "Federico II" - Napoli (NA)

Napoli, 80131, Italy

Location

Fondazione IRCCS Policlinico San Matteo - Pavia (PV)

Pavia, 27100, Italy

Location

Grande Ospedale Metropolitano "Bianchi-Melacrino-Morelli" - Reggio Calabria (RC)

Reggio Calabria, 89133, Italy

Location

AUSL Reggio Emilia (RE)

Reggio Emilia, 42122, Italy

Location

ASL Roma 2 "Ospedale S. Eugenio" - Roma (RM)

Roma, 00144, Italy

Location

AOU Policlinico Umberto I "Università La Sapienza" - Roma (RM)

Roma, 00161, Italy

Location

Azienda Ospedaliero-Universitaria Senese - Siena (SI)

Siena, 53100, Italy

Location

AOU Città della Salute e della Scienza - Torino (TO)

Torino, 10126, Italy

Location

AOU Integrata Verona "Ospedale Borgo Roma" - Verona (VN)

Verona, 37134, Italy

Location

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

  • Carlo Gambacorti

    Ospedale San Gerardo - Monza (MI)

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 12, 2021

First Posted

January 14, 2021

Study Start

February 1, 2021

Primary Completion

October 31, 2024

Study Completion

April 1, 2025

Last Updated

February 3, 2021

Record last verified: 2021-01

Data Sharing

IPD Sharing
Will not share

Locations