Clinical trial · Interventional
Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ AML/MDS/JMML
Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamid and Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): PI left institution
Summary
Brief summary (as posted)
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)
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 Myeloid Leukemia | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| Juvenile Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | ONTOLOGY_EXACT | 0.98 |
| Myelodysplastic Syndrome | Myelodysplastic Syndrome | CURATED_BROADER | 0.80 |
Interventions
Interventions (7)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Busulfan | Drug | Busulfan | ALIAS |
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Gemtuzumab Ozogamicin | Drug | Gemtuzumab Ozogamicin | ALIAS |
| Methotrexate | Drug | Methotrexate | ALIAS |
| Mycophenolate Mofetil | Drug | — | UNRESOLVED |
| Tacrolimus | Drug | — | UNRESOLVED |
| Thymoglobulin | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- study 515
- interventionNames
- Drug: Gemtuzumab Ozogamicin
- Drug: Busulfan
- Drug: Cyclophosphamide
- Drug: Thymoglobulin
- Drug: Tacrolimus
- Drug: Mycophenolate Mofetil
- Drug: Methotrexate
Primary outcomes (1)
- measure
- Maximal tolerated dose or tolerable dose of Gemtuzumab Ozogamicin (anti-CD33 immunotoxin) therapy combined with Busulfan/ Cyclophosphamide in the conditioning regimen prior to AlloSCT in patients with high risk CD33+ AML/JMML/MDS
- timeFrame
- 1 year
Secondary outcomes (3)
- measure
- Changes, if applicable, of minimal residual disease (cytogenetics, FISH, RT-PCR) in patients with high risk CD33+ AML/JMML/MDS after AlloSCT.
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 30 Years
Show eligibility criteria text
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.
References
Publications (0)
Data not yet available