Clinical trial · Interventional
Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Leukemia
Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia (AML/MDS/JMML)
NCT01020539CI-TRIAL-00053133AML/MDS/JMMLcompletedPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Myelogenous Leukemia | Acute Myeloid Leukemia | ALIAS | 0.90 |
| Juvenile Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | ONTOLOGY_EXACT | 0.98 |
| Myelodysplastic Syndrome | Myelodysplastic Syndrome | CURATED_BROADER | 0.80 |
Interventions
Interventions (6)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Anti-Thymocyte Globulin | Drug | — | UNRESOLVED |
| Busulfan | Drug | Busulfan | ALIAS |
| Fludarabine | Drug | Fludarabine | ALIAS |
| Gemtuzumab Ozogamicin | Drug | Gemtuzumab Ozogamicin | ALIAS |
| GVHD Prophylaxis | Drug | — | UNRESOLVED |
| Isotretinoin | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Matched Family Donor
- description
- Patients will receive a reduced intensity fludarabine (6 days)/busulfan (2 days) conditioning regimen followed by a stem cell transplant from a related family donor using bone marrow or cord blood stem cells. Then followed by Gemtuzumab Ozogamicin, once at about 2-6 months post alloSCT and once at least 2 months after the first dose. Patients with JMML will also receive cis-retinoic acid (Isotretinoin). During and following transplantation patients will receive methylprednisolone for immune suppression. Graft-versus-host-disease (GVHD) prophylaxis will consist of tacrolimus, mycophenolate mofetil and additionally methotrexate in recipients of unrelated adult transplants.
- interventionNames
- Drug: Fludarabine
- Drug: Busulfan
- Drug: GVHD Prophylaxis
- Drug: Gemtuzumab Ozogamicin
- type
- EXPERIMENTAL
- label
- Unrelated Donor
- description
- Patients will receive a reduced intensity fludarabine (6 days)/busulfan (2 days) conditioning regimen followed by a stem cell transplant from an unrelated donor using matched bone marrow or cord blood stem cells.Then followed by Gemtuzumab Ozogamicin, once at about 2-6 months post alloSCT and once at least 2 months after the first dose. Patients with JMML will also receive cis-retinoic acid (Isotretinoin). During and following transplantation patients will receive methylprednisolone for immune suppression and unrelated donor transplant recipients will additionally receive Anti-Thymocyte Globulin. GVHD prophylaxis will consist of tacrolimus, mycophenolate mofetil and additionally methotrexate in recipients of unrelated adult transplants.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Month
- Maximum age
- 30 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available
No reference posted for this study.