Overview

Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Leukemia

Status:
Completed
Trial end date:
2020-12-01
Target enrollment:
0
Participant gender:
All
Summary
Allogeneic stem cell transplantation (AlloSCT) followed by targeted immune therapy Gemtuzumab Ozogamicin patients with acute myeloid leukemia (AML)/juvenile myelomonocytic leukemia (JMML)/myelodysplastic syndromes (MDS) will be safe and well tolerated.
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Columbia University
Treatments:
Antilymphocyte Serum
Busulfan
Fludarabine
Gemtuzumab
Isotretinoin
Thymoglobulin
Criteria
Inclusion Criteria:

Disease Status

- AML 1st complete remission (CR) with a matched family donor (excluding Downs Syndrome,
acute promyelocytic leukaemia (APL), poor cytogenetics (12p, 5q, -7 and FMS-like
tyrosine kinase 3 (FLT3) mutations or duplication t(9;11) and others)) and patients
consented to and registered on an upfront AML COG study with a matched family donor)

- AML 1st CR (excluding Downs Syndrome, APL, and chromosome translocation (8;21) or
inversion (16) or poor cytogenetics (12p, 5q, -7, FLT3 mutation or duplication t(9;11)
and others)) with unrelated donor

- AML 2nd CR

- Myelodysplastic Syndrome (MDS) and ≤ 5% bone marrow myeloblasts at diagnosis (de novo
patients only)

- Juvenile Myelomonocytic Leukemia (JMML) and ≤ 5% bone marrow myeloblasts at diagnosis

In regards to disease immunophenotype, disease must express a minimum of ≥10% Cell
Differential 33 (CD33) positivity for patients with AML

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 glomerular filtration rate (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 2.0 x upper limit of normal (ULN), or
serum glutamic-oxaloacetic transaminase (SGOT)(aspartate aminotransferase (AST)) or serum
glutamic-pyruvic transaminase (SGPT)(alanine aminotransferase (ALT)) < 5.0 x ULN

Adequate cardiac function defined as:

- Shortening fraction of ≥ 25% by echocardiogram, or

- Ejection fraction of ≥ 45% by radionuclide angiogram or echocardiogram

Adequate pulmonary function defined as:

- Diffusion capacity of the lung for carbon monoxide (DLCO) ≥ 40% by pulmonary function
tests (PFT) (Uncorrected)

- 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 central nervous system (CNS) AML/JMML disease at time of
preparative regimen

- Secondary MDS

- Poor cytogenetics (12p, 5q, -7, FLT3 mutation or duplication)

- Female patients who are pregnant (positive human chorionic gonadotropin(hCG))

- Karnofsky <70% or Lansky <50% if 10 years or less

- Age >30 years

- Seropositive for Human Immunodeficiency Virus (HIV)

- Patients consented to and registered on an upfront Children's Oncology Group (COG) AML
study with a matched family donor