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Clinical Trials (PDQ®)

  • First Published: 5/6/2009

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Phase II Study of Alemtuzumab and Low-Dose Cyclosporine as Alternative Immunosuppressive Treatment in Patients With Severe Aplastic Anemia or Single Lineage Acquired Marrow Failure. Note: The information about this trial has not been updated by the sponsor/principal investigator/lead organization. Cancer.gov cannot verify the accuracy of the information.

Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Registry Information

Alternate Title

Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure. Note: The information about this trial has not been updated by the sponsor/principal investigator/lead organization. Cancer.gov cannot verify the accuracy of the information.

Basic Trial Information

PhaseTypeStatusAgeSponsorProtocol IDs
Phase IITreatmentUnknown18 and overOtherUNMS-ALESAA
ALESAA, EU-20927, EUDRACT-2008-001151-22, NCT00895739

Objectives

Primary

  1. Determine the safety of alemtuzumab and low-dose cyclosporine, as defined by occurrence of adverse effects, in patients with severe aplastic anemia or single lineage acquired marrow failure.
  2. Determine the efficacy of this regimen, in terms of overall survival, hematological response (partial and complete response, including time to response) and failure-free survival (failure is defined as no response, chronic treatment-maintained response, or relapse), in these patients.

Secondary

  1. Evaluate the incidence of adverse effects after treatment.
  2. Evaluate the long-term safety of alemtuzumab treatment.
  3. Determine the time to achieve a complete hematological response.
  4. Determine the proportion of patients maintaining hematological response free of any treatment.
  5. Determine the incidence of relapse in responding patients.
  6. Determine the incidence of severe infections.
  7. Determine the requirement for IV antibiotics and antifungal therapy.
  8. Determine the requirement for red cell and platelet transfusion.
  9. Determine the incidence of CMV reactivation.
  10. Determine the kinetics of immune reconstitution.
  11. Determine the incidence of paroxysmal nocturnal hemoglobinuria clone (lymphoid or myeloid) development.
  12. Determine the incidence of clonal evolution (i.e., karyotypic abnormalities or secondary myelodysplasia/leukemia).

Entry Criteria

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 109/L (severe) or < 0.2 x 109/L (very severe)
        • Platelet count < 20 x 109/L
        • Reticulocyte count < 20 x 109/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)

Prior/Concurrent Therapy:

  • No prior allogeneic stem cell transplantation
  • At least 2 weeks since prior cyclosporine or filgrastim (G-CSF)

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)

Expected Enrollment

50

Outcomes

Primary Outcome(s)

Safety, as defined by occurrence of adverse effects
Overall survival
Hematologic response (partial and complete response, including time to response)
Failure-free survival (failure is defined as no response, chronic treatment-maintained response, or relapse)

Secondary Outcome(s)

Incidence of adverse effects after treatment
Long-term safety of alemtuzumab treatment
Time to achieve a complete hematological response
Proportion of patients maintaining hematological response free of any treatment
Incidence of relapse in responding patients
Incidence of severe infections
Requirement for IV antibiotics and antifungal therapy
Requirement for red cell and platelet transfusion
Incidence of CMV reactivation
Kinetics of immune reconstitution
Incidence of paroxysmal nocturnal hemoglobinuria (PNH) clone (lymphoid or myeloid) development
Incidence of clonal evolution (i.e., karyotypic abnormalities or secondary myelodysplasia/leukemia)

Outline

Patients receive alemtuzumab subcutaneously on days 1-5*. Patients also receive oral cyclosporine beginning on day 7 and continuing for ≥ 180 days, followed by a taper according to clinical condition.

 [Note: *Patients with single lineage aquired marrow failure receive alemtuzumab on days 1-4.]

After completion of study therapy, patients will be followed up every 3 months for up to 2 years.

Trial Contact Information

Trial Lead Organizations

Federico II University Medical School

Bruno Rotoli, MD, Principal investigator
Ph: 39-081-746-2068
Email: rotoli@unina.it

Registry Information
Official Title Alemtuzumab and Low-Dose Cyclosporine-A as Alternative Immunosuppressive Treatment for Severe Aplastic Anemia (SAA) and Single-Lineage Aplastic Patients
Trial Start Date 2006-06-15
Registered in ClinicalTrials.gov NCT00895739
Date Submitted to PDQ 2009-03-31
Information Last Verified 2009-05-06

Note: The purpose of most clinical trials listed in this database is to test new cancer treatments, or new methods of diagnosing, screening, or preventing cancer. Because all potentially harmful side effects are not known before a trial is conducted, dose and schedule modifications may be required for participants if they develop side effects from the treatment or test. The therapy or test described in this clinical trial is intended for use by clinical oncologists in carefully structured settings, and may not prove to be more effective than standard treatment. A responsible investigator associated with this clinical trial should be consulted before using this protocol.

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