Clinical trial · Interventional
Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome
HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING OF BUSULFAN, FLUDARABINE, AND ANTI-THYMOCYTE GLOBULIN FOR PATIENTS WITH BONE MARROW FAILURE SYNDROME - A PHASE 2 STUDY
- 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 low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining abnormal cells (graft-versus-tumor effect). PURPOSE: This phase II trial is studying how well a donor stem cell transplant works after busulfan, fludarabine, methylprednisolone, and antithymocyte globulin in treating patients with bone marrow failure syndrome.
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 |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
| Nonmalignant Neoplasm | — | UNRESOLVED | — |
| Paroxysmal Nocturnal Hemoglobinuria | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| nonmyeloablative allogeneic hematopoietic stem cell transplantation | Procedure | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (1)
- measure
- Donor cell engraftment
- timeFrame
- 10-35 days after transplantation
- description
- neutrophil count over 500/ul
Secondary outcomes (4)
- measure
- Regimen-related toxicities as assessed by NCI's Common Toxicity Criteria
- timeFrame
- 0-60 months after transplantation
- description
- various toxicities of treatment
- measure
- Acute and chronic GVHD
- timeFrame
- 15-100 days; 100 days to 4 years
- description
- ocurrence of acute or chronic GVHD after transplantation
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 75 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Diagnosis of any of the following bone marrow failure syndromes:
* Severe aplastic anemia, meeting 1 of the following criteria:
* Not responsive to immunosuppressive therapy
* With recurrent cytopenia after immunosuppressive therapy or allogeneic hematopoietic cell transplantation
* Low-risk myelodysplastic syndrome, including any of the following:
* Refractory anemia
* Refractory anemia with ringed sideroblasts
* Refractory cytopenia with multi-lineage dysplasia
* Paroxysmal nocturnal hemoglobinuria, meeting 1 of the following criteria:
* With thrombotic episodes
* With severe cytopenia
* No willing, suitable HLA-compatible donor in family or in donor registries
* Related donor with HLA-haploidentical mismatch at three or less of 6 loci
* Patients with very severe neutropenia (\< 200/μL) or febrile episodes, who feel urgent need for allogeneic hematopoietic cell transplantation, are eligible without a search for HLA-matched unrelated donors
PATIENT CHARACTERISTICS:
* Karnofsky performance status 70-100%
* Bilirubin \< 2.0 mg/dL
* AST \< 3 times upper limit of normal
* Creatinine \< 2.0 mg/dL
* Ejection fraction \> 40% by MUGA scan
PRIOR CONCURRENT THERAPY:
* See Disease CharacteristicsReferences
Publications (0)
Data not yet available