Study of Sapacitabine in Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS)

Study Overview
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Brief Summary
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This is a combination study to evaluate sapacitabine administered in alternating cycles with decitabine in previously untreated Acute Myeloid Leukemia (AML) or concomitantly with venetoclax in previously treated AML or MDS

Detailed Description
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This is an open-label, single arm, study of sapacitabine administered in alternating cycles with decitabine in elderly patients with previously untreated AML (Part 1) or concomitantly with venetoclax in adult patients with relapsed or refractory AML or MDS (Part 2). Treatment will be administered on an outpatient basis. One treatment cycle is 4 weeks.

Official Title
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A Phase I/II Combination Study of Sapacitabine in Acute Myeloid Leukemia or Myelodysplastic Syndromes

Conditions
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Acute Myeloid Leukemia, Myelodysplastic Syndromes

Intervention / Treatment
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* Drug: sapacitabine and decitabine (Part 1 - completed)
* Drug: sapacitabine and venetoclax (Part 2 - recruiting)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Newly diagnosed AML based on WHO classification (Part 1) or previously treated AML or MDS (Part 2) Age 70 years or older for whom the treatment of choice is low-intensity therapy by investigator assessment or who has refused intensive induction therapy recommended by investigator (Part 1); age 18 years or older (Part 2) ECOG performance status 0-2 Adequate renal function Adequate liver function Able to swallow capsules Ability to understand and willingness to sign the informed consent form Exclusion Criteria: AML is of the sub-type of acute promyelocytic leukemia or extramedullary myeloid tumor without bone marrow involvement Known central nervous system (CNS) involvement by leukemia Uncontrolled intercurrent illness including Known hypersensitivity to decitabine (Part 1) or venetoclax (Part 2) Known to be HIV-positive

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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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Sapacitabine/decitabine (Part 1 - completed)<br>decitabine will be administered in alternating cycles with sapacitabine | Drug: sapacitabine and decitabine (Part 1 - completed)<br>* decitabine will be administered in alternating cycles with sapacitabine<br>|
| Experimental: sapacitabine/venetoclax (Part 2 - recruiting)<br>sapacitabine will be administered concomitantly with venetoclax | Drug: sapacitabine and venetoclax (Part 2 - recruiting)<br>* sapacitabine will be administered concomitantly with venetoclax<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of patients who achieve a clinical benefit response including CR, CRp, PR and major HI |  | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| response duration |  | 2 years | 
| transfusion requirements |  | 2 years | 
| hospitalized days |  | 2 years | 
| overall survival |  | 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Sapacitabine, decitabine, venetoclax, elderly, AML, MDS, Refractory, relapsed, untreated