Clinical trial · Interventional
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia
- 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: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Drugs used in chemotherapy, such as fludarabine and busulfan, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell, 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 cyclosporine together with methotrexate and methylprednisolone may stop this from happening. PURPOSE: This phase II trial is studying how well giving alemtuzumab together with fludarabine and busulfan works when given before donor stem cell transplant in treating young patients with hematologic disorders.
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 |
|---|---|---|---|
| Congenital Amegakaryocytic Thrombocytopenia | — | UNRESOLVED | — |
| Diamond-blackfan Anemia | — | UNRESOLVED | — |
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
| Severe Congenital Neutropenia | — | UNRESOLVED | — |
Interventions
Interventions (10)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| alemtuzumab | Biological | Alemtuzumab | ALIAS |
| allogeneic bone marrow transplantation | Procedure | — | UNRESOLVED |
| allogeneic hematopoietic stem cell transplantation | Procedure | — | UNRESOLVED |
| busulfan | Drug | Busulfan | ALIAS |
| cyclosporine | Drug | — | UNRESOLVED |
| fludarabine phosphate | Drug | Fludarabine | ALIAS |
| methotrexate | Drug | Methotrexate | ALIAS |
| methylprednisolone | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Single arm - conditioning and transplant
- description
- Alemtuzumab 0.5 mg/kg (maximum 15 mg) daily for 3 days; Busulfan i.v. every 6 hours from day -9 to day -6 for 16 total doses; Fludarabine phosphate from day -5 for 4 days at 1.3 mg/kg (if patient was less than 12 kg) or 40 mg/m\*2 per dose; Cyclosporine continuous infusion 3 mg/kg/Day beginning day -1 for GVHD prophylaxis; Methotrexate at 15 mg/m\*2 on day +1, 10 mg/m\*2 on days +3, +6, and (only for MUDs) day +11 also for GVHD prophylaxis; Methylprednisolone only as required for GVHD prophylaxis; allogeneic bone marrow transplantation or allogeneic hematopoietic stem cell transplantation or peripheral blood stem cell transplantation or umbilical cord blood transplantation.
- interventionNames
- Biological: alemtuzumab
- Drug: busulfan
- Drug: cyclosporine
- Drug: fludarabine phosphate
- Drug: methotrexate
- Drug: methylprednisolone
- Procedure: allogeneic bone marrow transplantation
- Procedure: allogeneic hematopoietic stem cell transplantation
- Procedure: peripheral blood stem cell transplantation
- Procedure: umbilical cord blood transplantation
Primary outcomes (1)
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 21 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Diagnosis of 1 of the following hematologic conditions:
* Aplastic anemia with marrow aplasia, meeting all of the following criteria:
* Absolute neutrophil count \< 500/mm\^3
* Platelet and/or red cell transfusion dependent
* Chronic aplastic anemia, meeting all of the following criteria:
* Transfusion dependent
* Unresponsive to immunosuppressive therapy
* Alternative matched unrelated donor has been identified
* Congenital marrow failure syndrome, including any of the following (with closely matched related or unrelated donor):
* Primary red cell aplasia (Diamond-Blackfan syndrome)
* Congenital neutropenia (Kostmann's syndrome)
* Amegakaryocytic thrombocytopenia
* Congenital dyserythropoietic anemias
* Other severe acquired cytopenias in which a transplantation using a combined busulfan/cyclophosphamide conditioning regimen is indicated
* Hemoglobinopathy (with closely matched related or unrelated donor)
* β-thalassemia major
* Sickle cell anemia
* Hemoglobin E/β-thalassemia
* Severe immunodeficiency disease
* Chediak-Higashi disease
* Wiskott-Aldrich syndrome
* Combined immunodeficiency disease (Nezelof's)
* Hyper immunoglobulin M (IgM) syndrome
* Bare lymphocyte syndrome
* Chronic granulomatous disease
* Familial erythrohemophagocytic lymphohistiocytosis
* Other stem cell defects (e.g., osteopetrosis)
* Severe immune dysregulation/autoimmune disorders
* Achieved a transient response to prior immunosuppressive therapy
* Chronic myelogenous leukemia
* Disease in first chronic phase
* Acute myeloid leukemia
* Disease in first remission
* Myelodysplastic syndromes
* Inborn errors of metabolism
* Histiocytosis
* No severe combined immunodeficiency disease
* Matched related or unrelated donor available by high resolution DNA typing
* Related donor, meeting both of the following criteria:
* Matched at both human leukocyte antigen (HLA)-Drβ1 alleles
* No more than 1 mismatch at the 4 HLA-A and -B alleles
* Unrelated donor, meeting 1 of the following criteria:
* Marrow matched at both HLA-Drβ1 alleles AND no more than 1 mismatch at the 4 HLA-A and -B alleles
* Umbilical cord blood matched at 5/6 HLA-A, -B, and -DRβ1 alleles with at least 1 -DRβ1 match AND there are ≥ 3x10\^5 CD34+ (Cluster of differentiation 34-positive) cells per kg body weight of recipient available at the time of cryopreservation
PATIENT CHARACTERISTICS:
* Cardiac ejection fraction ≥ 27%
* Creatinine clearance ≥ 50 mL/min by 24-hour urine collection or glomerular filtration rate
* DLCO (diffusion capacity of lung for carbon monoxide) ≥ 50% of predicted (corrected for anemia/lung volume)
PRIOR CONCURRENT THERAPY:
* No prior transplantation for leukemia from which patient remains engrafted and alemtuzumab is not needed as part of the conditioning regimenReferences
Publications (0)
Data not yet available