Extension Study With Tosedostat in Relapsed/Refractory Acute Myeloid Leukemia

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the long-term efficacy and safety profile of tosedostat in elderly patients suffering from refractory or relapsed Acute Myeloid Leukemia.

Detailed Description
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Extension protocol to the OPAL Study (CHR-2797-038). Study mimics normal clinical practice; few procedures and visits are therefore mandated by the protocol. Timing of bone marrow assessment is also left at the investigator's discretion.

Official Title
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The TOPAZ Study: A Long-Term Extension Study in Elderly Subjects With Relapsed/Refractory Acute Myeloid Leukemia to Allow Continued Therapy With Tosedostat

Conditions
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Acute Myeloid Leukemia

Intervention / Treatment
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* Drug: CHR-2797


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed, informed consent Completion of Visit 11 in the OPAL Study (Month 6 Visit) Investigator's opinion that the subject would benefit from continued therapy with tosedostat. Exclusion Criteria: Any co-existing medical condition that in the Investigator's opinion will substantially increase the risk associated with the subject's participation in the study Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary studies Administration of any (other) investigational agent within 14 days of entry into TOPAZ.

Ages Eligible for Study
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Minimum Age: 60 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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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Tosedostat<br> | Drug: CHR-2797<br>* 120mg once daily oral for 48 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and Tolerability of extended treatment with tosedostat | Primary outcome will be assessed using the following procedures/data: Physical exams Vital signs Electrocardiography Laboratory parameters (hematology, chemistry, urinalysis) Adverse events Serious adverse events | Protocol mandated visits every 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy of extended treatment with tosedostat | The secondary outcome will be assessed using the following parameters: Overall survival Relapse-free survival Event-free survival Clinical responses (Complete Remission, Complete Remission with Incomplete Platelet Recovery, Morphological Leukemia-Free State, Partial Remission, Stable Disease, Progressive Disease) including best response and time to response Duration of clinical responses. | Protocol-mandated visits every 12 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Relapse Refractory Acute Myeloid Leukemia