Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ AML/MDS/JMML

• Status: Terminated
• Trial end date: 2013-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The addition of gemtuzumab ozogamicin (GO) in combination with Busulfan/Cyclophosphamide followed by AlloSCT in patients with high risk CD33+ AML/JMML/MDS will be safe and well tolerated.

This study will attempt to determine the maximum tolerated dose of the immune therapy (gemtuzumab) when given in combination with the myeloablative (high dose) drugs used in this study for allogeneic stem cell transplant. (Part A)

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: New York Medical College
• Treatments: Busulfan; Cyclophosphamide; Gemtuzumab; Methotrexate; Mycophenolate mofetil; Mycophenolic Acid; Tacrolimus; Thymoglobulin

Criteria

Eligibility

Inclusion Criteria:

Disease Status

• AML Induction Failure

• AML in 1st, 2nd, or 3rd Relapse (>10% bone marrow blasts)

• AML greater than or equal to 3rd CR

• MDS with >6% bone marrow blasts at diagnosis

• Secondary MDS with less than or equal to 5% bone marrow myeloblasts at diagnosis

• JMML with >6% bone marrow myeloblasts at diagnosis

Disease Immunophenotype Patients (AML only) receiving gemtuzumab ozogamicin must express minimum of >10% or =10% CD33 positivity. Patients with <10% CD33 positivity will not receive gemtuzumab ozogamicin.

Organ Function

Patients must have adequate organ function as defined below:

• Adequate renal function defined as:

• Serum creatinine <1.5 x normal, or

• Creatinine clearance or radioisotope GFR 40 ml/min/m2 or >60 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range

• Adequate liver function defined as:

• Total bilirubin 1.5 x normal, or SGOT (AST) or SGPT (ALT) <2.0 x normal or =2.0 x normal

• Adequate cardiac function defined as:

• Shortening fraction of >27% by echocardiogram, or

• Ejection fraction of >47% by radionuclide angiogram or echocardiogram

• Adequate pulmonary function defined as:

• DLCO >55% or =55% by PFT

• For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry >94% on room air

Exclusion Criteria:

• Patients with active CNS AML/JMML/MDS disease at time of conditioning therapy

• Female patients who are pregnant (positive HCG)

• Karnofsky <50% or Lansky <50% if 10 years or less

• Age >65 years

• Has received gemtuzumab in the previous 30 days or has not recovered from prior gemtuzumab therapy.