SCT Plus Immune Therapy in Average Risk AML/MDS

Phase 2

Completed

• Conditions: Acute Myelogenous Leukemia; Myelodysplastic Syndrome
• Interventions: Drug: Gemtuzumab Ozogamicin

• Registration Number: NCT02117297
• Lead Sponsor: New York Medical College

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.

Study Timeline

• Study Start: 2012-01-12
• Study First Submitted: 2014-04-08
• Study First Submitted QC: 2014-04-15
• Study First Posted: 2014-04-17
• Primary Completion: 2023-03-30
• Study Completion: 2023-03-30
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-24
• Last Update Posted: 2023-10-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 26

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Gemtuzumab Ozogamicin	Gemtuzumab Ozogamicin	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.

Primary Outcome Measures

Name	Time	Method
to evaluate incidence of graft failure	Day +42	If three or more of the first ten patients experience primary or secondary graft failure, we will discontinue the study.
to evaluate survival rates	1 year	Event-free survival and overall survival after RI AlloSCT and targeted immunotherapy in patients with average risk AML/MDS.
to determine toxicity	1 year	to monitor for serious adverse events related to protocol investigational therapy

Secondary Outcome Measures

Name	Time	Method
Minor histocompatibility antigen	1 year	To measure the minor histocompatibility antigen expression on AML tissue, donor and recipient, and the development of MHA specific CTLs post AlloSCT.
Chimerism	1 year	To determine the degree of mixed/complete donor chimerism after RI AlloSCT in patients with average risk AML/MDS.
Graft-versus-host disease	1 Year	To estimate the risk of acute and chronic GVHD following RI AlloSCT and FK506/MMF GVHD prophylaxis in patients with average risk AML/MDS.

Trial Locations

Locations (1)

New York Medical College; 🇺🇸 Valhalla, New York, United States