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出境医 / 临床实验 / Observational Study on Chronic Myeloid Leukemia Patients in Any Phase Treated With Ponatinib (Iclusig®) at Any Dose (Ponderosa)

Observational Study on Chronic Myeloid Leukemia Patients in Any Phase Treated With Ponatinib (Iclusig®) at Any Dose (Ponderosa)

Study Description
Brief Summary:
Additional information is needed to characterize the safety profile of ponatinib as it is used in routine clinical practice in Europe. This observational cohort study will provide a real-life picture of ponatinib use in clinical practice and additional quantification and characterization of adverse events (AEs) and their outcomes in patients with Chronic Myeloid Leukemia in any phase treated with ponatinib.

Condition or disease
Chronic Myeloid Leukemia, Chronic Phase

Detailed Description:

This study is a multicentre, long-term, prospective and retrospective, observational cohort study of patients who are treated with ponatinib. The study is non-interventional; all Treatment decisions are made at the discretion of the patient's healthcare provider (HCP) and are not mandated by the study design or protocol.

A heterogeneous sample of oncology practice sites in Germany (both Hospitals and Field based Haematologists) treating adult patients with Chronic Myeloid Leukemia in any phase using ponatinib will be selected for participation in the study. Eligible, consenting patients initiating treatment with ponatinib will be enrolled into the prospective part of the study. Eligible consenting patients with Chronic Myeloid Leukemia in any Phase that have initiated ponatinib outside clinical trial after its approval in Germany will be enrolled in the retrospective part of the study.

Approximately 100 patients will be enrolled from approximately 50 sites. The patient enrolment period is estimated to be approximately two years. Patients who experience an Adverse Event (AE) during treatment or within the 30 days post discontinuation will be followed for 12 months to determine the consequences of the Adverse Event. Data on patient's history, patient's data at baseline, ponatinib treatment, Adverse Events, all medications received, and efficacy measures will be collected during the study in conjunction with routine care visits, anticipated to be approximately every 3 months.

Information on any new therapies, including treatment for Chronic Myeloid Leukemia and other medications will be collected during follow-up.

Study Design
Layout table for study information
Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Case-Only
Time Perspective: Retrospective
Official Title: Observational Study on Chronic Myeloid Leukemia Patients in Any Phase Treated With Ponatinib (Iclusig®) at Any Dose
Actual Study Start Date : July 8, 2015
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : December 31, 2022
Arms and Interventions
Outcome Measures
Primary Outcome Measures :
  1. Change in Cytogenetic response [ Time Frame: through study completion, an average of 3 years ]
    Achievement of cytogenetic Response (percentage of Ph+ metaphases)

  2. Molecular response [ Time Frame: through study completion, an average of 3 year ]
    BCR-ABL in % (lower is better)


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study will include patients with Chronic Myeloid Leukemia in any phase that are initiating or have initiated treatment with ponatinib and who meet the enrolment criteria and provide informed consent. A group of patients, for whom the decision of starting ponatinib has already been made, but in whom ponatinib has not yet been started, will be identified prospectively and invited to enrol into the study. The study enrolment date is the date the informed consent is signed. An additional group of patients who have received or are receiving ponatinib treatment outside clinical trials will be invited to join the retrospective part of the study.
Criteria

Inclusion Criteria:

  • Adult patients (age ≥18 years) with Chronic Myeloid Leukemia in any phase who are initiating ponatinib monotherapy, or for whom ponatinib monotherapy was initiated after ponatinib approval in Germany. [The decision to prescribe ponatinib must have been made prior to enrolment in the study. Patients enrolled in the retrospective part of the study may or may not be still on ponatinib treatment at the time informed consent is given. These retrospective patients should have started Treatment after 02.02.2015.].
  • Patients who have the ability to understand the requirements of the study, and provide written informed consent to comply with the study data collection procedures.
  • Patients with a minimum life expectancy of 3 months

Exclusion Criteria:

  • Patients previously treated with investigational ponatinib (within a clinical trial)
  • Patients receiving an investigational agent
  • Patients who are pregnant and/or breastfeeding
  • Patients who are pregnant and/or breastfeeding
Contacts and Locations

Contacts
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Contact: Andreas Hochhaus, Prof. Dr. +49 36419396670 ponderosa@med.uni-jena.de

Locations
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Germany
University Hospital Jena Recruiting
Jena, Germany, 07747
Contact: Andreas Hochhaus, Prof. Dr.         
Sponsors and Collaborators
University of Jena
Investigators
Layout table for investigator information
Principal Investigator: Andreas Hochhaus University Hospital Jena
Tracking Information
First Submitted Date April 8, 2019
First Posted Date May 1, 2019
Last Update Posted Date January 29, 2021
Actual Study Start Date July 8, 2015
Estimated Primary Completion Date December 31, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 29, 2019)
  • Change in Cytogenetic response [ Time Frame: through study completion, an average of 3 years ]
    Achievement of cytogenetic Response (percentage of Ph+ metaphases)
  • Molecular response [ Time Frame: through study completion, an average of 3 year ]
    BCR-ABL in % (lower is better)
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Observational Study on Chronic Myeloid Leukemia Patients in Any Phase Treated With Ponatinib (Iclusig®) at Any Dose
Official Title Observational Study on Chronic Myeloid Leukemia Patients in Any Phase Treated With Ponatinib (Iclusig®) at Any Dose
Brief Summary Additional information is needed to characterize the safety profile of ponatinib as it is used in routine clinical practice in Europe. This observational cohort study will provide a real-life picture of ponatinib use in clinical practice and additional quantification and characterization of adverse events (AEs) and their outcomes in patients with Chronic Myeloid Leukemia in any phase treated with ponatinib.
Detailed Description

This study is a multicentre, long-term, prospective and retrospective, observational cohort study of patients who are treated with ponatinib. The study is non-interventional; all Treatment decisions are made at the discretion of the patient's healthcare provider (HCP) and are not mandated by the study design or protocol.

A heterogeneous sample of oncology practice sites in Germany (both Hospitals and Field based Haematologists) treating adult patients with Chronic Myeloid Leukemia in any phase using ponatinib will be selected for participation in the study. Eligible, consenting patients initiating treatment with ponatinib will be enrolled into the prospective part of the study. Eligible consenting patients with Chronic Myeloid Leukemia in any Phase that have initiated ponatinib outside clinical trial after its approval in Germany will be enrolled in the retrospective part of the study.

Approximately 100 patients will be enrolled from approximately 50 sites. The patient enrolment period is estimated to be approximately two years. Patients who experience an Adverse Event (AE) during treatment or within the 30 days post discontinuation will be followed for 12 months to determine the consequences of the Adverse Event. Data on patient's history, patient's data at baseline, ponatinib treatment, Adverse Events, all medications received, and efficacy measures will be collected during the study in conjunction with routine care visits, anticipated to be approximately every 3 months.

Information on any new therapies, including treatment for Chronic Myeloid Leukemia and other medications will be collected during follow-up.
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Retrospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population The study will include patients with Chronic Myeloid Leukemia in any phase that are initiating or have initiated treatment with ponatinib and who meet the enrolment criteria and provide informed consent. A group of patients, for whom the decision of starting ponatinib has already been made, but in whom ponatinib has not yet been started, will be identified prospectively and invited to enrol into the study. The study enrolment date is the date the informed consent is signed. An additional group of patients who have received or are receiving ponatinib treatment outside clinical trials will be invited to join the retrospective part of the study.
Condition Chronic Myeloid Leukemia, Chronic Phase
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 29, 2019) 		100
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 31, 2022
Estimated Primary Completion Date December 31, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Adult patients (age ≥18 years) with Chronic Myeloid Leukemia in any phase who are initiating ponatinib monotherapy, or for whom ponatinib monotherapy was initiated after ponatinib approval in Germany. [The decision to prescribe ponatinib must have been made prior to enrolment in the study. Patients enrolled in the retrospective part of the study may or may not be still on ponatinib treatment at the time informed consent is given. These retrospective patients should have started Treatment after 02.02.2015.].
  • Patients who have the ability to understand the requirements of the study, and provide written informed consent to comply with the study data collection procedures.
  • Patients with a minimum life expectancy of 3 months

Exclusion Criteria:

  • Patients previously treated with investigational ponatinib (within a clinical trial)
  • Patients receiving an investigational agent
  • Patients who are pregnant and/or breastfeeding
  • Patients who are pregnant and/or breastfeeding
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Andreas Hochhaus, Prof. Dr. +49 36419396670 ponderosa@med.uni-jena.de
Listed Location Countries Germany
Removed Location Countries  
 
Administrative Information
NCT Number NCT03933852
Other Study ID Numbers Ponderosa
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Prof. Dr. med. Andreas Hochhaus, University of Jena
Study Sponsor University of Jena
Collaborators Not Provided
Investigators
Principal Investigator: Andreas Hochhaus University Hospital Jena
PRS Account University of Jena
Verification Date January 2021

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