Alemtuzumab to Treat Severe Aplastic Anaemia

This study will evaluate the safety and usefulness of a new immunosuppressive drug, alemtuzumab (Campath®), in patients with severe aplastic anemia (SAA). SAA is a rare and serious blood disorder in which the bone marrow stops making red blood cells, white blood cells and platelets. Alemtuzumab is a monoclonal antibody that attaches to and kills white blood cells called lymphocytes. In certain types of aplastic anemia, lymphocytes are responsible for the destruction of stem cells in the bone marrow, leading to a decrease in blood counts. Because alemtuzumab destroys lymphocytes, it may be effective in treating aplastic anemia. Alemtuzumab is currently approved to treat chronic lymphocytic leukemia and is also helpful in other conditions that require immunosuppression, such as rheumatoid arthritis and immune cytopenias.

Official Title

A Pilot Study of Alemtuzumab (Campath) in Patients With Relapsed or Refractory Severe Aplastic Anemia.

Conditions

• Relapsed or Refractory Severe Aplastic Anemia
• Severe Aplastic Anemia

Study Type

Interventional

Study Design

 

  Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study.

Further Details

Primary Outcome Measures:

• Response rate at six months, defined as no longer satisfying blood count criteria for SAA.

Secondary Outcome Measures:

• Relapse, robustness of the hematopoietic recovery at three and six months, three months responses, survival, and clonal evolution to myelodysplasia and acute leukemia.

Study Start

September 2005

Eligibility & Criteria

Official Title

A Pilot Study of Alemtuzumab (Campath) in Patients With Relapsed or Refractory Severe Aplastic Anemia.

Conditions

• Relapsed or Refractory Severe Aplastic Anemia
• Severe Aplastic Anemia

Study Type

Interventional

Study Design

 

  Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study.

Further Details

Primary Outcome Measures:

• Response rate at six months, defined as no longer satisfying blood count criteria for SAA.

Secondary Outcome Measures:

• Relapse, robustness of the hematopoietic recovery at three and six months, three months responses, survival, and clonal evolution to myelodysplasia and acute leukemia.

Study Start

September 2005

Eligibility & Criteria

• Ages Eligible for Study:  

     2 Years and older

•  

   Genders Eligible for Study:

   

     Both

Inclusion Criteria:

• Relapsed severe aplastic anemia after initial hematologic response to a prior course of h-ATG or r-ATG based immunosuppression
• Refractory severe aplastic anemia not responding to both horse-ATG and rabbit ATG-based immunosuppression
• The criteria for severe aplastic anemia are two of the three criteria:
  • Absolute neutrophil count less than or equal to 500 /mm(3)
  • Platelets to less than or equal to 20,000/mm(3)
  • Absolute reticulocyte count less than 60,000 /uL
• Age greater than or equal to 2 years old
• Patients or their parent(s)/responsible guardian(s) must be able to comprehend and be willing to sign an informed consent.

Exclusion Criteria:

• Diagnosis of Fanconi’s anemia
• Evidence of a clonal disorder on cytogenetics. In the refractory disease setting, patients with super severe neutropenia (ANC less than 200 /uL) will not be excluded if results of cytogenetics are not available or pending.
• Infection not adequately responding to appropriate therapy
• HIV positivity
• Failure to discontinue the herbal supplements Echinacea purpurea or Usnea barbata (Old Man’s Beard) within 2 weeks of enrollment
• Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient’s ability to tolerate protocol therapy, or that death within 7-10 days is likely
• Previous hypersensitivity to alemtuzumab or its components
• Potential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
• Current pregnancy, or unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
• Not able to understand the investigational nature of the study or give informed consent.

 

 

Total Enrolment

62

Contact Details

POC: Patient Recruitment and Public Liaison Office
Phone No.: (800) 411-1222, 1-866-411-1010
Email: prpl@mail.cc.nih.gov
Website: www.abcdef.com

Location:

United States, Maryland
National Institutes of Health Clinical Center,
9000 Rockville Pike
Bethesda, Maryland
USA, 20892

 

   

Total Enrolment

62

Contact Details

POC: Patient Recruitment and Public Liaison Office
Phone No.: (800) 411-1222, 1-866-411-1010
Email: prpl@mail.cc.nih.gov
Website: www.abcdef.com

Location:

United States, Maryland
National Institutes of Health Clinical Center,
9000 Rockville Pike
Bethesda, Maryland
USA, 20892