SCT Plus Immune Therapy in Average Risk AML/MDS
Primary Purpose
Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Gemtuzumab Ozogamicin
Sponsored by
About this trial
This is an interventional treatment trial for Acute Myelogenous Leukemia
Eligibility Criteria
Inclusion Criteria:
Disease Status:
- AML 1st CR with a matched family donor
- AML 1st CR with unrelated donor
- AML 2nd CR or CRP
- MDS and < or = 5% bone marrow myeloblasts at diagnosis
Disease Immunophenotype:
- Disease must express a minimum of > or = 10% CD33 positivity for patients with AML
Organ Function:
- Adequate renal function, adequate liver function, adequate cardiac function, adequate pulmonary function
Exclusion Criteria:
- Patients with active CNS AML disease at time of preparative regimen
- Secondary MDS
- Poor cytogenetics
- Female patients who are pregnant
- Karnofsky <70% or Lansky <50% if 10 years or less
- Age >25 years
- Seropositive for HIV
Sites / Locations
- New York Medical College
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Gemtuzumab Ozogamicin
Arm Description
Consolidation therapy with GO will be administered between days 60 and 180 post transplantation when the ANC is >1000/mm3 and platelet count is >40,000/mm3 untransfused x 3 days after AlloSCT and again at minimum 8 weeks later.
Outcomes
Primary Outcome Measures
to evaluate incidence of graft failure
If three or more of the first ten patients experience primary or secondary graft failure, we will discontinue the study.
to evaluate survival rates
Event-free survival and overall survival after RI AlloSCT and targeted immunotherapy in patients with average risk AML/MDS.
to determine toxicity
to monitor for serious adverse events related to protocol investigational therapy
Secondary Outcome Measures
Minor histocompatibility antigen
To measure the minor histocompatibility antigen expression on AML tissue, donor and recipient, and the development of MHA specific CTLs post AlloSCT.
Chimerism
To determine the degree of mixed/complete donor chimerism after RI AlloSCT in patients with average risk AML/MDS.
Graft-versus-host disease
To estimate the risk of acute and chronic GVHD following RI AlloSCT and FK506/MMF GVHD prophylaxis in patients with average risk AML/MDS.
Full Information
NCT ID
NCT02117297
First Posted
April 8, 2014
Last Updated
September 26, 2022
Sponsor
New York Medical College
1. Study Identification
Unique Protocol Identification Number
NCT02117297
Brief Title
SCT Plus Immune Therapy in Average Risk AML/MDS
Official Title
Allogeneic Stem Cell Transplantation Following by Targeted Immune Therapy) (Gemtuzumab Ozogamicin) in Average Risk Acute Myelogenous Leukemia and Myelodysplastic Syndrome (AML/MDS)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 2011 (undefined)
Primary Completion Date
December 2022 (Anticipated)
Study Completion Date
December 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
New York Medical College
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Allogeneic stem cell transplantation followed by targeted immune therapy with Gemtuzumab Ozogamicin (Mylotarg) will be given to patients with average risk AML or MDS.
Detailed Description
Reduced intensity conditioning regimen of Busulfan (Bu) and Fludarabine (Flu) + Anti-Thymocyte Globulin (ATG ) (unrelated donors only) or reduced toxicity conditioning regimen of Bu/Flu/alemtuzumab, or reduced hepatic toxicity regimen of melphan/Flu/alemtuzumab and AlloSCT, followed by Gemtuzumab Ozogamicin consolidation in patients with average risk AML/MDS meeting eligibility criteria.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myelogenous Leukemia, Myelodysplastic Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
26 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Gemtuzumab Ozogamicin
Arm Type
Experimental
Arm Description
Consolidation therapy with GO will be administered between days 60 and 180 post transplantation when the ANC is >1000/mm3 and platelet count is >40,000/mm3 untransfused x 3 days after AlloSCT and again at minimum 8 weeks later.
Intervention Type
Drug
Intervention Name(s)
Gemtuzumab Ozogamicin
Other Intervention Name(s)
Mylotarg
Intervention Description
Gemtuzumab, 9.0 mg/m2, will be given IV over 2 hours two times post allogeneic transplantation.
Primary Outcome Measure Information:
Title
to evaluate incidence of graft failure
Description
If three or more of the first ten patients experience primary or secondary graft failure, we will discontinue the study.
Time Frame
Day +42
Title
to evaluate survival rates
Description
Event-free survival and overall survival after RI AlloSCT and targeted immunotherapy in patients with average risk AML/MDS.
Time Frame
1 year
Title
to determine toxicity
Description
to monitor for serious adverse events related to protocol investigational therapy
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Minor histocompatibility antigen
Description
To measure the minor histocompatibility antigen expression on AML tissue, donor and recipient, and the development of MHA specific CTLs post AlloSCT.
Time Frame
1 year
Title
Chimerism
Description
To determine the degree of mixed/complete donor chimerism after RI AlloSCT in patients with average risk AML/MDS.
Time Frame
1 year
Title
Graft-versus-host disease
Description
To estimate the risk of acute and chronic GVHD following RI AlloSCT and FK506/MMF GVHD prophylaxis in patients with average risk AML/MDS.
Time Frame
1 Year
10. Eligibility
Sex
All
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Disease Status:
AML 1st CR with a matched family donor
AML 1st CR with unrelated donor
AML 2nd CR or CRP
MDS and < or = 5% bone marrow myeloblasts at diagnosis
Disease Immunophenotype:
Disease must express a minimum of > or = 10% CD33 positivity for patients with AML
Organ Function:
Adequate renal function, adequate liver function, adequate cardiac function, adequate pulmonary function
Exclusion Criteria:
Patients with active CNS AML disease at time of preparative regimen
Secondary MDS
Poor cytogenetics
Female patients who are pregnant
Karnofsky <70% or Lansky <50% if 10 years or less
Age >25 years
Seropositive for HIV
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mitchell S. Cairo, M.D.
Organizational Affiliation
New York Medical College
Official's Role
Principal Investigator
Facility Information:
Facility Name
New York Medical College
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
SCT Plus Immune Therapy in Average Risk AML/MDS
We'll reach out to this number within 24 hrs