Immunochemotherapy and AlloSCT in Patients With High Risk CD33+ AML/MDS

Study Overview
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Brief Summary
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Targeted immune therapy with gemtuzumab ozogamicin (Mylotarg) in combination with chemotherapy followed by allogeneic stem cell transplantation will be given to patients with high risk acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).

Detailed Description
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Gemtuzumab ozogamicin in combination with busulfan and cyclophosphamide (immunochemotherapy) conditioning followed by allogeneic stem cell transplantation + anti-thymocyte globulin (unrelated donors only) in patients with high risk CD33+AML or MDS meeting eligibility criteria.

Official Title
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A Pilot Study of Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamide (Immunochemotherapy) and Allogeneic Stem Cell Transplantation in Patients With High Risk Acute Myelogenous Leukemia and Myelodysplastic Syndrome

Conditions
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Acute Myelogenous Leukemia, Myelodysplastic Syndrome

Intervention / Treatment
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* Drug: Gemtuzumab Ozogamicin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Disease Status: History of AML Induction/Reinduction Failure AML 1st Complete Remission (CR) with poor cytogenetics AML 2nd CR with minimal residual disease (MRD) AML 3rd CR AML in refractory relapse but ≤25% bone marrow leukemia blasts MDS with >6% bone marrow blasts at diagnosis Secondary MDS with ≤5% bone marrow myeloblasts at diagnosis Disease Immunophenotype: • Disease must express a minimum of >/= 10% CD33+ for patients with AML Organ Function: • Adequate renal function, adequate liver function, adequate cardiac function, adequate pulmonary function Age: ≤25 years of age Donor: matched family donor, unrelated cord blood donor, unrelated adult donor Exclusion Criteria: Patients with active central nervous system (CNS) AML/MDS disease at time of conditioning therapy Female patients who are pregnant Karnofsky <50% or Lansky <50% if 10 years or less Age >25 years Has received gemtuzumab in the previous 30 days or has not recovered from prior gemtuzumab therapy.

Ages Eligible for Study
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Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Gemtuzumab Ozogamicin<br>Conditioning therapy with Gemtuzumab Ozogamicin in combination with busulfan and cyclophosphamide chemotherapy followed by allogeneic stem cell transplantation. | Drug: Gemtuzumab Ozogamicin<br>* Gemtuzumab Ozogamicin 7.5 mg/m^2/dose given IV over 2 hours once during conditioning<br>* Other names: Mylotarg;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response rate | overall response rate (CR + PR) in patients receiving GO, busulfan and cyclophosphamide and AlloSCT in patients with measurable disease (relapse/refractory) with high risk CD33+ AML/MDS | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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targeted therapy, leukemia, MDS, immune therapy