A Study to Evaluate Effectiveness and Safety of Ponatinib in Patients With BCR-ABL Positive ALL in Standard Clinical Practice in Europe - POSEIDON

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the effectiveness and safety of ponatinib (Iclusig®) in patients with BCR-ABL positive acute lymphoblastic leukemia (ALL) in standard clinical practice in Europe.

Official Title
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A Postmarketing Observational Cohort Study to Evaluate Effectiveness and Safety of Ponatinib (Iclusig®) in Patients With BCR-ABL Positive ALL in Standard Clinical Practice in Europe - POSEIDON

Conditions
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BCR-ABL Positive Acute Lymphoblastic Leukemia

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult patients with BCR-ABL (Ph+) positive ALL who are initiating Iclusig®, or for whom Iclusig® was initiated after January 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. Exclusion Criteria: Patients previously treated with investigational ponatinib. Patients who are pregnant and/or breastfeeding. Concurrent treatment with another tyrosine kinase inhibitor for Ph+ALL.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Ph+ ALL Patients<br>Patients with Ph+ ALL being treated with Iclusig®. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Complete hematological remission (CR) rate | CR defined as leukemic cells not detectable by light microscopy in bone marrow (BM), peripheral blood (PB), or cerebrospinal fluid (CSF) (BM < 5% blasts). | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| CR rate | CR defined as leukemic cells not detectable by light microscopy in BM, PB, or CSF (BM < 5% blasts). | 1, 3, 9, and 12 months | 
| CR rate | CR defined as leukemic cells not detectable by light microscopy in BM, PB, or CSF (BM < 5% blasts). | 1, 3, 6, 9, and 12 months | 
| Time to CR | Defined as time from enrollment to first CR. | Up to 12 months | 
| Minimal residual disease (MRD) level | Minimal residual disease level. | 3, 6, 9, and 12 months | 
| Best MRD (MolR) level rate | MolR defined as molecular/MRD response, less than MolCR. | 12 months | 
| Time to best MRD (MolR) level | MolR defined as molecular/MRD response, less than MolCR. | Up to 12 months | 
| Duration of molecular response | Measured by MRD log reduction (MolR). | Up to 12 months | 
| Duration of best molecular response (MolCR) | MolCR defined as complete molecular remission/MRD negativity. | Up to 12 months | 
| Time to progression | Defined as the time to molecular relapse or hematological relapse. | Up to 12 months | 
| Time to death | Defined as time from enrollment to death due to any cause. | Up to 12 months | 
| Prescribed dose | Prescribed dose of Iclusig® in milligrams. | Up to 12 months | 
| Daily average dose | The average daily dose of Iclusig® in milligrams. | Up to 12 months | 
| Number of serious adverse events (SAEs) | Defined as an adverse event that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, is a congenital anomaly/birth defect, or is an important medical event. | 12 months | 
| Number of adverse events of special interest (AESI) | AESI defined as vascular occlusive events, including arterial and venous events, as defined in the protocol. | 12 months | 
| Amount of hospital days | Defined as overnight stay(s), each night in the hospital will be counted as 1 day. | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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acute lymphoblastic leukemia, ponatinib, tyrosine kinase inhibitor