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

Phase 2

Completed

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

• Registration Number: NCT02221310
• Lead Sponsor: New York Medical College

Brief Summary

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

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.

Study Timeline

• Study Start: 2011-12-07
• Study First Submitted: 2014-07-31
• Study First Submitted QC: 2014-08-18
• Study First Posted: 2014-08-20
• Primary Completion: 2023-04-04
• Study Completion: 2023-04-04
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-24
• Last Update Posted: 2023-10-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Gemtuzumab Ozogamicin	Gemtuzumab Ozogamicin	Conditioning therapy with Gemtuzumab Ozogamicin in combination with busulfan and cyclophosphamide chemotherapy followed by allogeneic stem cell transplantation.

Primary Outcome Measures

Name	Time	Method
Response rate	1 year	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

Trial Locations

Locations (1)

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