Consolidation Therapy for Acute Myeloid Leukemia Guided by Leukemia Stem Cell Behavior

• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (Other)
• Overall Status: Terminated
• CT.gov ID: NCT01588951
• Collaborator: (none)
• Enrollment: 10
• Location: 1
• Arms: 3
• Actual Duration (Months): 40
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

The primary objective of the trial is to compare the two-year relapse-free survival (RFS) of patients with acute myeloid leukemia (AML), presumed to be at high risk for relapse due to the presence of leukemia stem cells (LSCs) in their bone marrow at first complete remission (CR1), who receive either standard cytarabine-based chemotherapy or allogeneic stem cell transplantation (SCT).

Condition or Disease	Intervention/Treatment	Phase
Acute Myeloid Leukemia	Drug: Cytarabine consolidation; Drug: Allogeneic transplant	Phase 2/Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 10 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Consolidation Therapy for Acute Myeloid Leukemia Guided by Leukemia Stem Cell Behavior
• Actual Study Start Date: Dec 1, 2013
• Actual Primary Completion Date: Apr 1, 2017
• Actual Study Completion Date: Apr 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: No Leukemia Stem Cells - Consolidation Without LSC, standard cytarabine consolidation	Drug: Cytarabine consolidation; Cytarabine-based consolidation per institutional standards.; Other Names: HiDAC
Experimental: Leukemia Stem Cells - Consolidation LSC present, randomized to cytarabine consolidation	Drug: Cytarabine consolidation; Cytarabine-based consolidation per institutional standards.; Other Names: HiDAC
Experimental: Leukemia Stem Cells - Transplant LSC present, randomized to allogeneic transplant	Drug: Allogeneic transplant; Allogeneic stem cell transplant per institutional standards.; Other Names: Bone marrow transplant Stem cell transplant Hematopoietic stem cell transplant BMT HSCT

Outcome Measures

Primary Outcome Measures

1. Relapse Free Survival [2 years]

   Percentage of participants alive and without relapsed disease at two years.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 100 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Age greater than or equal to 18 years

2. Able to give informed consent

3. New diagnosis of AML, other than APL or poor-risk AML, as defined in section 3.2

Exclusion criteria:

1. Has already had a bone marrow biopsy and aspirate to assess remission status after induction therapy

2. Any debilitating medical or psychiatric illness that would preclude ability to give informed consent or receive optimal treatment and follow-up

3. Pregnancy: Women of childbearing potential who are β- HCG+

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	Baltimore	Maryland	United States	21287

Sponsors and Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Investigators

• Study Chair: Margaret Showel, MD, JHU

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Jan 18, 2017

More Information

Publications

None provided.

• Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
• ClinicalTrials.gov Identifier: NCT01588951
• Other Study ID Numbers: J1227; NA_00071844
• First Posted: May 1, 2012
• Last Update Posted: Aug 14, 2018
• Last Verified: Jul 1, 2018

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Cytarabine

Study Results

Participant Flow

Arm/Group Title	No Leukemia Stem Cells - Consolidation	Leukemia Stem Cells - Consolidation	Leukemia Stem Cells - Transplant
Arm/Group Description	Without LSC, standard cytarabine consolidation Cytarabine consolidation: Cytarabine-based consolidation per institutional standards.	LSC present, randomized to cytarabine consolidation Cytarabine consolidation: Cytarabine-based consolidation per institutional standards.	LSC present, randomized to allogeneic transplant Allogeneic transplant: Allogeneic stem cell transplant per institutional standards.
Period Title: Overall Study
STARTED	2	2	6
COMPLETED	2	2	5
NOT COMPLETED	0	0	1

Baseline Characteristics

Arm/Group Title	No Leukemia Stem Cells - Consolidation	Leukemia Stem Cells - Consolidation	Leukemia Stem Cells - Transplant	Total
Arm/Group Description	Without LSC, standard cytarabine consolidation Cytarabine consolidation: Cytarabine-based consolidation per institutional standards.	LSC present, randomized to cytarabine consolidation Cytarabine consolidation: Cytarabine-based consolidation per institutional standards.	LSC present, randomized to allogeneic transplant Allogeneic transplant: Allogeneic stem cell transplant per institutional standards.	Total of all reporting groups
Overall Participants	2	2	6	10
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%	0 0%
Between 18 and 65 years	2 100%	1 50%	3 50%	6 60%
>=65 years	0 0%	1 50%	3 50%	4 40%
Sex: Female, Male (Count of Participants)
Female	2 100%	2 100%	1 16.7%	5 50%
Male	0 0%	0 0%	5 83.3%	5 50%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	1 50%	0 0%	0 0%	1 10%
Not Hispanic or Latino	1 50%	2 100%	6 100%	9 90%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%	0 0%
Asian	1 50%	0 0%	1 16.7%	2 20%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	1 16.7%	1 10%
White	0 0%	2 100%	4 66.7%	6 60%
More than one race	0 0%	0 0%	0 0%	0 0%
Unknown or Not Reported	1 50%	0 0%	0 0%	1 10%

Outcome Measures

1. Primary Outcome

Title	Relapse Free Survival
Description	Percentage of participants alive and without relapsed disease at two years.
Time Frame	2 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	No Leukemia Stem Cells - Consolidation	Leukemia Stem Cells - Consolidation	Leukemia Stem Cells - Transplant
Arm/Group Description	Without LSC, standard cytarabine consolidation Cytarabine consolidation: Cytarabine-based consolidation per institutional standards.	LSC present, randomized to cytarabine consolidation Cytarabine consolidation: Cytarabine-based consolidation per institutional standards.	LSC present, randomized to allogeneic transplant Allogeneic transplant: Allogeneic stem cell transplant per institutional standards.
Measure Participants	2	2	5
Number [percentage of participants]	0 0%	0 0%	0 0%

Adverse Events

Time Frame	Collected monthly for up to 3.5 years.
Adverse Event Reporting Description	Only unexpected adverse events, grades III-IV acute GVHD, and chronic GVHD were collected.
Arm/Group Title	No Leukemia Stem Cells - Consolidation		Leukemia Stem Cells - Consolidation		Leukemia Stem Cells - Transplant
Arm/Group Description	Without LSC, standard cytarabine consolidation Cytarabine consolidation: Cytarabine-based consolidation per institutional standards.		LSC present, randomized to cytarabine consolidation Cytarabine consolidation: Cytarabine-based consolidation per institutional standards.		LSC present, randomized to allogeneic transplant Allogeneic transplant: Allogeneic stem cell transplant per institutional standards.
All Cause Mortality
	No Leukemia Stem Cells - Consolidation		Leukemia Stem Cells - Consolidation		Leukemia Stem Cells - Transplant
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/2 (0%)		0/2 (0%)		1/6 (16.7%)
Serious Adverse Events
	No Leukemia Stem Cells - Consolidation		Leukemia Stem Cells - Consolidation		Leukemia Stem Cells - Transplant
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/2 (0%)		0/2 (0%)		0/6 (0%)
Other (Not Including Serious) Adverse Events
	No Leukemia Stem Cells - Consolidation		Leukemia Stem Cells - Consolidation		Leukemia Stem Cells - Transplant
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/2 (0%)		0/2 (0%)		0/6 (0%)

Limitations/Caveats

[Not Specified]

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.