Myelodysplastic Syndrome Clinical Trial
— AML/MDS/JMMLOfficial title:
Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia (AML/MDS/JMML)
Verified date | August 2021 |
Source | Columbia University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 18 |
Est. completion date | December 2020 |
Est. primary completion date | July 20, 2011 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month to 30 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Columbia University Medical Center | New York | New York |
Lead Sponsor | Collaborator |
---|---|
Columbia University |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Maximal tolerated and/or biologically active dose of Gemtuzumab Ozogamicin (GO) | To determine the maximal tolerated and/or biologically active dose of Gemtuzumab Ozogamicin (GO) (anti CD33 immunotoxin) therapy following reduced intensity (RI) allogeneic stem cell transplantation (alloSCT) in patients with average risk AML/JMML/MDS. | Up to 2 years | |
Secondary | Change of minimal residual disease | To measure the changes, if applicable, of minimal residual disease prior to and after consolidation therapy with targeted immunotherapy in average risk AML/JMML/MDS post RI AlloSCT. | Day 60, Day 100, Day 180, 1 year, 2 years | |
Secondary | Incidence of minor histocompatibility antigen (MHA) expression on acute myeloid leukemia (AML) tissue | To measure the minor histocompatibility antigen expression on AML tissue, donor and recipient, and the incidence of development of MHA specific cytotoxic T-lymphocytes (CTLs). | Up to 2 years | |
Secondary | Degree of mixed/complete donor chimerism | To determine the degree of mixed/complete donor chimerism after RI AlloSCT in patients with average risk AML/JMML/MDS. | Up to 2 years | |
Secondary | Event free survival (EFS) rate | To determine event free survival (EFS) after RI AlloSCT and targeted immunotherapy in patients with average risk AML/JMML/MDS. | Up to 2 years | |
Secondary | Overall survival (OS) rate | To determine overall survival (OS) after RI AlloSCT and targeted immunotherapy in patients with average risk AML/JMML/MDS. | Up to 2 years |
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