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

Phase 1

• Conditions: Acute Myeloid Leukemia; Myelodysplastic Syndromes
• Interventions: Drug: sapacitabine and venetoclax (Part 2 - recruiting); Drug: sapacitabine and decitabine (Part 1 - completed)

• Registration Number: NCT01211457
• Lead Sponsor: Cyclacel Pharmaceuticals, Inc.

Brief Summary

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

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.

Study Timeline

• Study Start: 2010-06-17
• Study First Submitted: 2010-09-27
• Study First Submitted QC: 2010-09-28
• Study First Posted: 2010-09-29
• Status Verified: 2018-10
• Last Update Submitted: 2019-07-12
• Last Update Posted: 2019-07-16
• Primary Completion: 2020-06-20
• Study Completion: 2020-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 65

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
sapacitabine/venetoclax (Part 2 - recruiting)	sapacitabine and venetoclax (Part 2 - recruiting)	sapacitabine will be administered concomitantly with venetoclax
Sapacitabine/decitabine (Part 1 - completed)	sapacitabine and decitabine (Part 1 - completed)	decitabine will be administered in alternating cycles with sapacitabine

Primary Outcome Measures

Name	Time	Method
Number of patients who achieve a clinical benefit response including CR, CRp, PR and major HI	2 years

Secondary Outcome Measures

Name	Time	Method
response duration	2 years
transfusion requirements	2 years
hospitalized days	2 years
overall survival	2 years

Trial Locations

Locations (3)

Roswell Park Cancer Institute; 🇺🇸 Buffalo, New York, United States

Rush University Medical Center; 🇺🇸 Chicago, Illinois, United States

MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States