Evaluation of the safety and efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) using alemtuzumab for patients with aplastic anemia

UMIN ID: C000000356

Registered date:20/03/2006

Basic Information

• Recruitment status: Complete: follow-up complete
• Health condition(s) or Problem(s) studied: Aplastic anemia
• Date of first enrollment: 2004/12/01
• Target sample size: 38
• Countries of recruitment: Japan
• Study type: Interventional
• Intervention(s): Alemtuzumab is added to the fludarabine-based regimen at 0.16 – 0.25 mg/kg per day for 6 days (days -10 to -5).

Outcome(s)

• Primary Outcome: Survival on day 60 after transplantation Engraftment within 60 days Incidence of grade II to IV acute GVHD within 60 days
• Secondary Outcome: • Regimen-related toxicity • Incidence of infectious disease • Overall survival at day 365 • Therapeutic efficacy for aplastic anemia

Key inclusion & exclusion criteria

• Age minimum: 20years-old
• Age maximum: 65years-old
• Gender: Male and Female
• Exclude criteria: (1) Diabetes uncontrollable even with regular insulin use. (2) Uncontrollable hypertension. (3) Current active infection. (4) 5% or more malignant cell involvement in bone marrow. (5) Abnormality in chromosome 7 detected by bone marrow G-banding or FISH. (6) Current active cancer. (7) Pregnant or nursing women or women who may be pregnant. (8) Uncontrollable mental illness. (9) Hepatitis B virus antigen-positive (HBsAg or HBeAg). (10) HIV-positive. (11) A history of hypersensitivity to transplant conditioning agents (fludarabine phosphate, cyclophosphamide) or agents for GVHD prophylaxis (ciclosporin, methotrexate). (12) A history of hematopoietic stem cell transplantation.

Related Information

• Primary Sponsor: GCP-ISS HE0403 group
• Source(s) of Monetary Support: 2004 Health and Labor Sciences Research Grant (Research on Measures for Intractable Diseases) of the Ministry of Health, Labor and Welfare.