Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure

NCT00895739 | Unknown Phase 2

• 4 other identifiers: UNMS-ALESAACDR0000639649EU-20927EUDRACT-2008-001151-22
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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

Nonmalignant Neoplasm

Keywords

aplastic anemia

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (12)

• 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

Interventions

alemtuzumab BIOLOGICAL

cyclosporine DRUG

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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)

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Federico II University: lead

Study Sites (1)

Federico II University Medical School

Naples, 80131, Italy

RECRUITING

MeSH Terms

Conditions

Neoplasms; Anemia, Aplastic

Interventions

Alemtuzumab; Cyclosporine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides

Study Officials

• Bruno Rotoli, MD

  Federico II University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: May 7, 2009
• First Posted: May 8, 2009
• Study Start: June 1, 2006
• Last Updated: August 12, 2013

Record last verified: 2009-05

Locations

IT (1)