Clinical trial · Interventional
Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure
Alemtuzumab and Low-Dose Cyclosporine-A as Alternative Immunosuppressive Treatment for Severe Aplastic Anemia (SAA) and Single-Lineage Aplastic Patients
- 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: Immunosuppressive therapies, such as alemtuzumab and cyclosporine, may improve bone marrow function and increase blood cell counts. Giving alemtuzumab together with cyclosporine may be an effective treatment for severe aplastic anemia or acquired marrow failure. PURPOSE: This phase II trial is studying the side effects of giving alemtuzumab together with cyclosporine and to see how well it works in treating patients with severe aplastic anemia or acquired marrow failure.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Nonmalignant Neoplasm | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| alemtuzumab | Biological | Alemtuzumab | ALIAS |
| cyclosporine | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (4)
- measure
- Safety, as defined by occurrence of adverse effects
- measure
- Overall survival
- measure
- Hematologic response (partial and complete response, including time to response)
- measure
- Failure-free survival (failure is defined as no response, chronic treatment-maintained response, or relapse)
Secondary outcomes (12)
- measure
- Incidence of adverse effects after treatment
- measure
- Long-term safety of alemtuzumab treatment
- measure
- Time to achieve a complete hematological response
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Diagnosis of 1 of the following:
* Severe or very severe aplastic anemia, as defined by the following criteria:
* Meets ≥ 2 of the following criteria:
* Absolute neutrophil count \< 0.5 x 10\^9/L (severe) or \< 0.2 x 10\^9/L (very severe)
* Platelet count \< 20 x 10\^9/L
* Reticulocyte count \< 20 x 10\^9/L
* Hypocellular bone marrow (\< 30% cellularity) without evidence of fibrosis or malignant cells
* Single lineage acquired marrow failure (e.g., pure red cell aplasia, agranulocytosis, amegakaryocytic thrombocytopenia)
* Paroxysmal nocturnal hemoglobinuria clone allowed
* Failed first-line therapy with antithymocyte globulin (ATG) and cyclosporine OR not eligible for ATG-based studies
* Failure is defined as lack of hematological response, requirement for chronic immunosuppressive treatment to sustain response, or relapse
* Not eligible for a low-risk stem cell transplantation
* No evidence of risky myelodysplastic syndromes (i.e., IPSS 3-4), as defined by the presence of marrow blast excess or karyotypic abnormalities, or other primitive marrow disease
* No history of constitutional aplastic anemia (e.g., Fanconi anemia or dyskeratosis congenita)
PATIENT CHARACTERISTICS:
* WHO performance status 0-2
* Not pregnant or nursing
* No active malignant tumor within the past 5 years
* Transaminases ≤ 3 times upper limit of normal (ULN)
* Albumin ≥ 1.5 g/L
* Creatinine ≤ 3 times ULN
* No CMV viremia, as defined by positive PCR or pp65 test
* No cardiac failure (i.e., ejection fraction \< 35%)
* No other concurrent life-threatening disease (including HIV infection)
PRIOR CONCURRENT THERAPY:
* No prior allogeneic stem cell transplantation
* At least 2 weeks since prior cyclosporine or filgrastim (G-CSF)References
Publications (0)
Data not yet available