Clinical trial · Interventional
Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission
Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission
- 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: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells. A donor peripheral blood, bone marrow , or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving antithymocyte globulin before the transplant may stop this from happening. PURPOSE: This phase I/II trial is studying the side effects of busulfan, antithymocyte globulin, and fludarabine when given together with a donor stem cell transplant in treating young patients with blood disorders, bone marrow disorders, chronic myelogenous leukemia in first chronic phase, or acute myeloid leukemia in first remission.
Conditions
Conditions (6)
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 | — |
| Diamond-blackfan Anemia | — | UNRESOLVED | — |
| Fanconi Anemia | — | UNRESOLVED | — |
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Severe Congenital Neutropenia | — | UNRESOLVED | — |
| Thrombocytopenia | — | UNRESOLVED | — |
Interventions
Interventions (7)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| allogeneic bone marrow transplantation | Procedure | — | UNRESOLVED |
| anti-thymocyte globulin | Biological | — | UNRESOLVED |
| busulfan | Drug | Busulfan | ALIAS |
| fludarabine phosphate | Drug | Fludarabine | ALIAS |
| peripheral blood stem cell transplantation | Procedure | — | UNRESOLVED |
| radiation therapy | Radiation | — | UNRESOLVED |
| umbilical cord blood transplantation | Procedure | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (1)
- measure
- Graft rejection measured by ANC < 500 with no evidence of donor cells in blood or marrow from transplantation to week 4 post transplantation
Secondary outcomes (5)
- measure
- Toxicity grades 3 or 4 assessed from conditioning through 1 year post transplantation
- measure
- Engraftment at 1, 3, 6, 9, and 12 months post transplantation
- measure
- Mixed chimerism at 1, 3, 6, 9, and 12 months post transplantation
- measure
- Survival measured from the day of first dose of conditioning
- measure
- Disease-free survival measured from the day of first dose of conditioning
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 17 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Diagnosis of one of the following hematopoietic disorders:
* Severe aplastic anemia with marrow aplasia (i.e., absolute neutrophil count \< 500/mm\^3, platelet and/or red blood cell transfusion dependent), meeting 1 of the following criteria:
* Closely matched related donor
* Unresponsive to immunosuppressive therapy within 3 months after follow-up AND alternative matched unrelated donor available
* Congenital marrow failure syndrome, including any of the following:
* Primary red blood cell aplasia (Diamond-Blackfan syndrome)
* Congenital neutropenia (Kostmann's syndrome)
* Amegakaryocytic thrombocytopenia
* Hemoglobinopathy including any of the following:
* β-thalassemia major
* Sickle cell anemia
* Severe immunodeficiency disease including any of the following:
* Chediak-Higashi disease
* Wiskott-Aldrich syndrome
* Combined immunodeficiency disease (Nezelof's)
* Hyperimmunoglobulin M syndrome
* Bare lymphocyte syndrome
* Other stem cell defects (e.g., osteopetrosis)
* Chronic myelogenous leukemia in first chronic phase
* Not eligible for other ongoing phase II/III studies
* Acute myeloid leukemia in first remission
* Not eligible for other ongoing phase II/III studies
* Inborn errors of metabolism
* No severe combined immunodeficiency disorder
* Available donor, meeting 1 of the following criteria:
* Related donor matched by high resolution DNA typing at both HLA Drβ1 alleles and ≤ 1 mismatch at the 4 HLA-A and -B alleles
* Unrelated donor, meeting one of the following criteria:
* Bone marrow matched by high resolution DNA typing at both HLA Drβ1 alleles and ≤ 1 mismatch by high resolution DNA typing at the 4 HLA-A and -B alleles
* Umbilical cord blood matched at 4/6 HLA-A, -B, and Drβ1 alleles by high resolution typing with ≥ 1 Drβ1 match and ≥ 3 X 10\^7 cells/kg body weight of recipient
PATIENT CHARACTERISTICS:
* See Disease Characteristics
* No active bacterial, viral, or fungal infection
* Cardiac shortening fraction ≥ 27%
* Creatinine clearance ≥ 60 mL/min
* DLCO ≥ 60% of predicted (corrected for anemia/lung volume)
PRIOR CONCURRENT THERAPY:
* See Disease CharacteristicsReferences
Publications (0)
Data not yet available