Clinical trial NCT01211457: Study of Sapacitabine in Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS)
Official title: A Phase I/II Combination Study of Sapacitabine in Acute Myeloid Leukemia or Myelodysplastic Syndromes
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Acute Myeloid Leukemia
Myelodysplastic Syndromes
Keywords: Sapacitabine
decitabine
venetoclax
elderly
AML
MDS
Refractory
relapsed
untreated
Lead sponsor: Cyclacel Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: M.D. Anderson Cancer Center
First posted: 2010-09-29
Enrollment: 65
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of patients who achieve a clinical benefit response including CR, CRp, PR and major HI
timeFrame: 2 years
Secondary outcomes:
measure: response duration
timeFrame: 2 years
measure: transfusion requirements
timeFrame: 2 years
measure: hospitalized days
timeFrame: 2 years
measure: overall survival
timeFrame: 2 years
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Hagop M Kantarjian, MD
role: STUDY_CHAIR
References:
citation: Pooled Analysis of Elderly Patients with Newly Diagnosed AML Treated with Sapacitabine and Decitabine Administered in Alternating Cycles. Farhad Ravandi, Tapan M. Kadia, Gautam Borthakur, William G. Wierda, Stuart L. Goldberg, Meir Wetzler, Parameswaran Venugopal, Karen Seiter, Judy Chiao and Hagop M. Kantarjian. Blood 2012 120:2630.