Clinical trial · Interventional
HLA-Nonidentical Stem Cell and Natural Killer Cell Transplantation for Children Less the Two Years of Age With Hematologic Malignancies
- 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)
Recent studies of conventional chemotherapy for infants with high-risk hematologic malignancies show that the long-term disease-free survival is low. Although blood and marrow stem cell transplantation using an HLA identical sibling has improved the outcome for these children, less than 25% have this donor source available. Another option is haploidentical transplantation using a partially matched family member donor (i.e. parental donor). Although haploidentical transplantation has proven curative for some patients, this procedure has been hindered by significant complications, primarily regimen-related toxicity including infection and graft versus host disease (GVHD). Building on prior institutional trials, this study will provide patients a haploidentical graft depleted of T lymphocytes using the investigational device, CliniMACS selection system. One week after the transplant procedure, patients will also receive an infusion of additional donor derived white blood cells called Natural Killer (NK) cells in an effort to decrease risks for rejection of the graft, disease relapse, and regimen related toxicity. The primary objective of the study is to evaluate 1 year survival in infants with high risk hematologic malignancies who receive this study treatment.
Conditions
Conditions (5)
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 Lymphocytic Leukemia | Acute Lymphoblastic Leukemia | ALIAS | 0.90 |
| Acute Myeloid Leukemia | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| Chronic Myeloid Leukemia | Chronic Myeloid Leukemia, BCR-ABL1 Positive | ALIAS | 0.90 |
| Histiocytosis | — | UNRESOLVED | — |
| Myelodysplasia | Myelodysplastic Syndrome | ALIAS | 0.90 |
Interventions
Interventions (3)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Allogeneic stem cell transplantation | Procedure | — | UNRESOLVED |
| Chemotherapy and antibodies | Drug | — | UNRESOLVED |
| Miltenyi Biotec CliniMACS | Device | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Study Participants
- description
- Participants who meet the eligibility criteria for this study. Donor cells will be obtained using the Miltenyi Biotec CliniMACS device. Interventions: Chemotherapy and antibodies, allogeneic stem cell transplantation.
- interventionNames
- Drug: Chemotherapy and antibodies
- Device: Miltenyi Biotec CliniMACS
- Procedure: Allogeneic stem cell transplantation
Primary outcomes (1)
- measure
- One-year Survival
- timeFrame
- One year after transplant
- description
- The one-year survival of infants with high-risk hematologic malignancies who receive a haploidentical transplant procedure using a total body irradiation (TBI)-excluding conditioning regimen followed by an HLA-nonidentical family donor hematopoietic stem cell (HSC) graft depleted of T cells ex vivo using the CliniMACS CD34+ selection system, with a subsequent infusion of donor NK cells purified ex vivo using the CliniMACS CD3+ depletion and CD56+ enrichment system. The Kaplan-Meier estimate for one-year survival is reported.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 24 Months
Show eligibility criteria text
Inclusion Criteria: Must have one of the following diagnosis: * AML in remission or relapse (e.g., FAB M7 or biphenotypic leukemia) * High-risk ALL in first remission (e.g., poor responder to prednisone, Ph+ ALL) * ALL beyond first remission * Secondary leukemia * Primary myelodysplasia (including RAEB, RAEB-T, CMML, JCML, and JMML) * Chronic myeloid leukemia * Histiocytoses (including multi-system Langerhans' cell histiocytosis and hemophagocytic lymphohistiocytosis Inclusion criteria Donor research participants * HIV negative (date). * Hepatitis B surface antigen negative (date). * Hepatitis C antibody negative (date). * Syphilis negative (date). * Donor is equal to or greater than 3 on 6 HLA match (date). * Not pregnant (negative pregnancy test). * Not lactating. * At least 18 years of age. Exclusion Criteria * Patients greater than 24 months of age at the time of transplant. * HLA-identical sibling donor is available. * Cardiac function: shortening fraction \<25%. * Pulse oximetry oxygen saturation \<92% on room air. * Glomerular filtration rate less than 40 ml/min/1.73 m2 (may use Technetium-99 result for GFR). * Direct bilirubin \> 3 mg/dl. * SGPT \> 500 U/L. * Patients with previous allergy to mouse proteins. * Patients with previous allergy to rabbit serum products. * Patients with Down's syndrome
References
Publications (0)
Data not yet available