Clinical trial · Interventional
Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease
Stem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency Diseases
- 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)
RATIONALE: Giving chemotherapy and total body irradiation before a donor bone marrow transplant or peripheral blood stem cell transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving antithymocyte globulin and removing the T cells from the donor cells before transplant may stop this from happening. PURPOSE: This phase I trial is studying the side effects and best dose of donor T cells and antithymocyte globulin when given together with chemotherapy and total-body irradiation in treating young patients who are undergoing T-cell depleted donor stem cell transplant for myelodysplastic syndrome, leukemia, bone marrow failure syndrome, or severe immunodeficiency disease.
Conditions
Conditions (4)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Congenital Amegakaryocytic Thrombocytopenia | — | UNRESOLVED | — |
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
| Severe Congenital Neutropenia | — | UNRESOLVED | — |
Interventions
Interventions (8)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| allogeneic bone marrow transplantation | Procedure | — | UNRESOLVED |
| allogeneic hematopoietic stem cell transplantation | Procedure | — | UNRESOLVED |
| anti-thymocyte globulin | Biological | — | UNRESOLVED |
| fludarabine phosphate | Drug | Fludarabine | ALIAS |
| in vitro-treated peripheral blood stem cell transplantation | Procedure | — | UNRESOLVED |
| therapeutic allogeneic lymphocytes | Biological | — | UNRESOLVED |
| thiotepa | Drug | Thiotepa | ALIAS |
| total-body irradiation | Radiation | — |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Single arm of transplant
- description
- Receiving haplocompatible T cell depleted peripheral blood stem cell transplant
- interventionNames
- Biological: anti-thymocyte globulin
- Biological: therapeutic allogeneic lymphocytes
- Drug: fludarabine phosphate
- Drug: thiotepa
- Procedure: allogeneic bone marrow transplantation
- Procedure: allogeneic hematopoietic stem cell transplantation
- Procedure: in vitro-treated peripheral blood stem cell transplantation
- Radiation: total-body irradiation
Primary outcomes (1)
- measure
- Engraftment at 4 weeks post bone marrow transplantation through 100 days
- timeFrame
- 100 days
Secondary outcomes (4)
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Year
- Maximum age
- 17 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Diagnosis of one of the following:
* Acute lymphoblastic leukemia in ≥ 2nd remission or delayed remission induction
* High-risk myelodysplastic syndromes
* Refractory anemia with excess blasts (RAEB)
* RAEB in transformation
* Chronic myelogenous leukemia in second chronic phase
* No accelerated phase (\> 5% blasts in marrow)
* Juvenile myelomonocytic leukemia
* Acute nonlymphoblastic leukemia in \> 1st remission or induction failure and \< 30% blasts in marrow
* Severe aplastic anemia, defined as absolute neutrophil count \< 500/mm\^3 and platelet and/or red blood cell transfusion dependent
* Unresponsive to immunosuppressive therapy
* No Fanconi's anemia
* Congenital marrow aplasias unresponsive to cytokines and transfusion dependent
* Inherited immunodeficiency disease involving neutrophils or lymphocytes, including any of the following:
* Chediak-Higashi disease
* Wiskott-Aldrich syndrome
* Combined immunodeficiency disease (Nezelof's)
* Hyper IgM syndrome
* No relapsed disease
* Haplocompatible related donor, including parent, cousin, aunt, uncle, grandparent, half-sibling, or sibling (≥ 12 years of age), available
* 2 or 3 HLA antigen mismatch
* At least a 3 HLA antigen genotypic match
* No closely matched related or unrelated donor available in sufficient time to do the transplant
PATIENT CHARACTERISTICS:
* No active hepatitis or cytomegalovirus infection
* Cardiac ejection fraction ≥ 30%
* Creatinine clearance ≥ 70 mL/min
* DLCO ≥ 70% of predicted
* No active infection
* No HIV positivity
PRIOR CONCURRENT THERAPY:
* See Disease CharacteristicsReferences
Publications (0)
Data not yet available