Alemtuzumab to Treat Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 25 March 2013
• Main ID: NCT00195624
• Date of registration: 16/09/2005
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: Alemtuzumab to Treat Severe Aplastic Anemia
• Scientific title: A Pilot Study of Alemtuzumab (Campath) in Patients With Relapsed or Refractory Severe Aplastic Anemia
• Date of first enrolment: September 2005
• Target sample size: 62
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT00195624
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Countries of recruitment

United States

Contacts

• Name: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)

• Name: Olga J Rios, R.N.

• Name: Danielle M Townsley, M.D.
• Affiliation: National Heart, Lung, and Blood Institute (NHLBI)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

Relapsed severe aplastic anemia after initial hematologic response to a prior course of
h-ATG or r-ATG based immunosuppression

Or

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 /microL

Age greater than or equal to 2 years old and greater than 12 kg

Prospective subjects 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,
prospective subjects with super severe neutropenia (ANC less than 200 /microL) 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



Age minimum: 2 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Relapsed or Refractory Severe Aplastic Anemia

Severe Aplastic Anemia

Intervention(s)

Drug: Alemtuzumab (Campath(Registered Trademark))

Primary Outcome(s)

Response rate at six months, defined as no longer satisfying blood count criteria for SAA. [Time Frame: 6 months]

Secondary Outcome(s)

Relapse, robustness of the hematopoietic recovery at three and six months, three months responses, survival, and clonal evolution to myelodysplasia and acute leukemia. [Time Frame: 6 months]

Secondary ID(s)

05-H-0242

050242

Source(s) of Monetary Support

Please refer to primary and secondary sponsors