Study of Vosaroxin and Cytarabine for the Treatment of Adults 60 Years of Age or Older With Previously Untreated AML

• Sponsor: Hamid Sayar (Other)
• Overall Status: Terminated
• CT.gov ID: NCT02485353
• Collaborator: (none)
• Enrollment: 2
• Locations: 2
• Arm: 1
• Actual Duration (Months): 15.9
• Patient Per Site: 1
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

This is a pilot efficacy assessment clinical trial of vosaroxin and cytarabine for the treatment of adults 60 years of age or older with previously untreated acute myeloid leukemia. A total of 17 evaluable patients are planned to be treated on the study.

Condition or Disease	Intervention/Treatment	Phase
Leukemia, Myeloid, Acute	Drug: Vosaroxin and Cytarabine	N/A

Detailed Description

Primary objective: rate of complete remission (morphologic complete remission or morphologic complete remission with incomplete blood count recovery) Secondary objectives: (1) safety of this combination in induction therapy of older patients previously unexposed to intensive chemotherapy; (2) progression-free survival; (3) length of stay in hospital for induction; (4) 30- and 60-day mortality rate.

The treatment period will include up to 4 cycles of treatment (induction 1, induction 2, consolidation 1, and consolidation 2). In each cycle, the first day of study treatment will be considered day 1.

• Each cycle will include vosaroxin treatment on days 1 and 4 (total of 2 days) and cytarabine treatment on days 1-5 (total of 5 days) followed by a variable interval required to achieve hematologic recovery, defined as absolute neutrophil count (ANC) > 1000 cells/L.

• Up to 2 cycles of consolidation treatment may be completed by eligible patients in remission (morphologic complete remission or morphologic complete remission with incomplete blood count recovery) if safety parameters are met.

Study Design

• Study Type: Interventional
• Actual Enrollment: 2 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Pilot Study of Vosaroxin and Cytarabine for the Treatment of Adults 60 Years of Age or Older With Previously Untreated AML
• Actual Study Start Date: Oct 20, 2015
• Actual Primary Completion Date: May 2, 2016
• Actual Study Completion Date: Feb 16, 2017

Arms and Interventions

Arm	Intervention/Treatment
Other: Vosaroxin and Cytarabine	Drug: Vosaroxin and Cytarabine

Outcome Measures

Primary Outcome Measures

1. Rate of Complete Remission [2 months]

   Percentage of patients who have complete remission as defined by the International Working Group for AML: morphologic complete remission (CR) or morphologic complete remission with incomplete blood count recovery (CRi or CRp)

Secondary Outcome Measures

1. Procedure or Procedure Related Adverse Events [Up to 1 year]

   Number of unique patients who had a procedure or treatment related (possible, probable or definite) adverse events. (graded per NCI CTC v4.0)

Eligibility Criteria

Criteria

• Ages Eligible for Study: 60 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Age > 60 years

• A diagnosis of AML based on WHO classification (>20% myeloblasts in peripheral blood or bone marrow)

• No previous treatment with chemotherapy for AML, other than hydroxyurea. Previous treatment with hypomethylating agents is acceptable

• Patients must have an ECOG (Zubrod) performance status of 0-2 (see Appendix I).

• Patients must have adequate hepatic function with a total bilirubin < 1.5 times upper limit normal (ULN) other than cases of Gilbert disease, and ALT and AST < 2.5 times ULN; and adequate renal function as defined by a serum creatinine < 2 times ULN.

• Clinically significant nonhematologic toxicity after prior therapy has recovered to grade 1 per Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 or newer

• Patients must have the ability to understand and the willingness to sign a written informed consent. A signed informed consent must be obtained prior to initiation of treatment

• Left ventricular ejection fraction (LVEF) at least 40% by multiple gated acquisition (MUGA) scan or echocardiogram (ECHO)

Exclusion Criteria:

• Patients with known central nervous system (CNS) leukemia by spinal fluid cytology, flow cytometry or imaging. A lumbar puncture is not required unless CNS involvement is clinically suspected. Patients with signs or symptoms of leukemic meningitis or a history of leukemic meningitis must have a negative lumbar puncture within 2 weeks of study enrollment.

• A diagnosis of acute promyelocytic leukemia (APL). The study does not require to rule APL out for every subject. However, if there is clinical suspicion for APL, such diagnosis has to be ruled out before initiation of treatment.

• Active malignancy currently undergoing chemo or radiation therapy. Hormone therapy is acceptable.

• Active serious infection that at the discretion of treating physician makes patient ineligible for chemotherapy.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Indiana University Health Hospital	Indianapolis	Indiana	United States	46202
2	Indiana University Health Melvin and Bren Simon Cancer Center	Indianapolis	Indiana	United States	46202

Sponsors and Collaborators

• Hamid Sayar

Investigators

• Principal Investigator: Hamid Sayar, MD, Indiana University School of Medicine

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Hamid Sayar, Assistant Professor of Clinical Medicine, Hematology/Oncology, Indiana University School of Medicine
• ClinicalTrials.gov Identifier: NCT02485353
• Other Study ID Numbers: IUSCC-0525
• First Posted: Jun 30, 2015
• Last Update Posted: Sep 20, 2017
• Last Verified: Aug 1, 2017

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Hamid Sayar, Assistant Professor of Clinical Medicine, Hematology/Oncology, Indiana University School of Medicine: Vosaroxin; Cytarabine; Remission Induction; Safety; Progression-Free Survival; Mortality Rate; Length of Stay
• Additional relevant MeSH terms: Leukemia, Myeloid; Leukemia, Myeloid, Acute; Cytarabine

Study Results

Participant Flow

Recruitment Details	This protocol was based on enrolling 20 patients. Due to the death of 2 patients which raised safety concerns, the study was closed with 2 patients enrolled.

Arm/Group Title	Vosaroxin and Cytarabine
Arm/Group Description	Each cycle will include vosaroxin treatment on days 1 and 4 (total of 2 days) and cytarabine treatment on days 1-5 (total of 5 days) followed by a variable interval required to achieve hematologic recovery, defined as absolute neutrophil count (ANC) > 1000 cells/L
Period Title: Overall Study
STARTED	2
COMPLETED	0
NOT COMPLETED	2

Baseline Characteristics

Arm/Group Title	Vosaroxin and Cytarabine
Arm/Group Description	Each cycle will include vosaroxin treatment on days 1 and 4 (total of 2 days) and cytarabine treatment on days 1-5 (total of 5 days) followed by a variable interval required to achieve hematologic recovery, defined as absolute neutrophil count (ANC) > 1000 cells/L
Overall Participants	2
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	0 0%
>=65 years	2 100%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	72.9 (1.03)
Sex: Female, Male (Count of Participants)
Female	0 0%
Male	2 100%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	1 50%
Not Hispanic or Latino	0 0%
Unknown or Not Reported	1 50%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	1 50%
More than one race	0 0%
Unknown or Not Reported	1 50%

Outcome Measures

1. Primary Outcome

Title	Rate of Complete Remission
Description	Percentage of patients who have complete remission as defined by the International Working Group for AML: morphologic complete remission (CR) or morphologic complete remission with incomplete blood count recovery (CRi or CRp)
Time Frame	2 months

Outcome Measure Data

Analysis Population Description

All patients receiving at least one dose of study drug and having at least one evaluable post-baseline visit

Arm/Group Title	Vosaroxin and Cytarabine
Arm/Group Description	Each cycle will include vosaroxin treatment on days 1 and 4 (total of 2 days) and cytarabine treatment on days 1-5 (total of 5 days) followed by a variable interval required to achieve hematologic recovery, defined as absolute neutrophil count (ANC) > 1000 cells/L
Measure Participants	2
Number [percentage of patients]	0

2. Secondary Outcome

Title	Procedure or Procedure Related Adverse Events
Description	Number of unique patients who had a procedure or treatment related (possible, probable or definite) adverse events. (graded per NCI CTC v4.0)
Time Frame	Up to 1 year

Outcome Measure Data

Analysis Population Description

All patients enrolled and received treatment

Arm/Group Title	Vosaroxin and Cytarabine
Arm/Group Description	Each cycle will include vosaroxin treatment on days 1 and 4 (total of 2 days) and cytarabine treatment on days 1-5 (total of 5 days) followed by a variable interval required to achieve hematologic recovery, defined as absolute neutrophil count (ANC) > 1000 cells/L
Measure Participants	2
Count of Participants [Participants]	1 50%

Adverse Events

Time Frame	Up to 1 year
Arm/Group Title	Vosaroxin and Cytarabine
Arm/Group Description	Each cycle will include vosaroxin treatment on days 1 and 4 (total of 2 days) and cytarabine treatment on days 1-5 (total of 5 days) followed by a variable interval required to achieve hematologic recovery, defined as absolute neutrophil count (ANC) > 1000 cells/L
All Cause Mortality
	Vosaroxin and Cytarabine
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Vosaroxin and Cytarabine
	Affected / at Risk (%)	# Events
Total	2/2 (100%)
Infections and infestations
Infections and infestations - Other	1/2 (50%)
Sepsis	1/2 (50%)
Other (Not Including Serious) Adverse Events
	Vosaroxin and Cytarabine
	Affected / at Risk (%)	# Events
Total	2/2 (100%)
Gastrointestinal disorders
Mucositis oral	2/2 (100%)
General disorders
Fatigue	1/2 (50%)
Fever	2/2 (100%)
Infections and infestations
Upper respiratory infection	1/2 (50%)
Metabolism and nutrition disorders
Hypoalbuminemia	2/2 (100%)
Nervous system disorders
Diziness	1/2 (50%)
Psychiatric disorders
Insomnia	1/2 (50%)
Renal and urinary disorders
Urinary retention	1/2 (50%)
Respiratory, thoracic and mediastinal disorders
Hiccups	2/2 (100%)
Respiratory, thoracic and mediastinal disorders - Other	1/2 (50%)
Sore throat	1/2 (50%)
Wheezing	1/2 (50%)
Skin and subcutaneous tissue disorders
Alopecia	1/2 (50%)

Limitations/Caveats

Due to the death of 2 patients which raised safety concerns, the study was closed with 2 patients enrolled.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.