Status:
COMPLETED
Eculizumab in Treating Patients With Paroxysmal Nocturnal Hemoglobinuria
Lead Sponsor:
Jonsson Comprehensive Cancer Center
Conditions:
Leukemia
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
RATIONALE: Chemoprevention is the use of certain drugs to keep cancer from forming, growing, or coming back. The use of eculizumab may prevent leukemia and stop the destruction of red blood cells in p...
Detailed Description
OBJECTIVES: Primary * Determine the safety of eculizumab in patients with transfusion-dependent hemolytic paroxysmal nocturnal hemoglobinuria. * Determine the efficacy of this drug, in terms of hemo...
Eligibility Criteria
Inclusion
- DISEASE CHARACTERISTICS:
- Diagnosis of paroxysmal nocturnal hemoglobinuria
- Must have required ≥ 4 episodes of transfusions for anemia or anemia-related symptoms within the past year
- Mean pre-transfusion hemoglobin ≤ 10. 5 g/dL over the past year
- Glycosylphosphatidylinositol (GPI)-deficient red blood cell clone (type III cells) of ≥ 10% by flow cytometry
- Must have received 1 packed red blood cell transfusion during the study observation period (within 48 hours of the hemoglobin level that precipitated the transfusion) and within 1.5 g/dL of the mean pre-transfusion hemoglobin level over the past year
- Pre-transfusion hemoglobin ≤ 9 g/dL with symptoms
- Pre-transfusion hemoglobin ≤ 7 g/dL without symptoms
- Received Neisseria meningitidis vaccination at least 2 weeks before initiation of study therapy
- PATIENT CHARACTERISTICS:
- Age
- 18 and over
- Performance status
- Not specified
- Life expectancy
- Not specified
- Hematopoietic
- See Disease Characteristics
- Absolute neutrophil count \> 500/mm\^3
- Platelet count ≥ 100,000/mm\^3
- Hepatic
- Lactate dehydrogenase ≥ 1.5 times upper limit of normal
- Renal
- Not specified
- Immunologic
- No known or suspected active bacterial infection
- No recurrent bacterial infections
- No history of meningococcal disease
- Other
- No known or suspected hereditary complement deficiency
- No other condition that would increase the patient's risk or confound the outcome of the study
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- PRIOR CONCURRENT THERAPY:
- Biologic therapy
- See Disease Characteristics
- No prior bone marrow transplantation
- Concurrent epoetin alfa allowed\*
- Chemotherapy
- Not specified
- Endocrine therapy
- Concurrent corticosteroids allowed\*\*
- Radiotherapy
- Not specified
- Surgery
- Not specified
- Other
- More than 30 days since prior participation in another investigational drug trial
- More than 30 days since prior investigational agents, devices, or procedures
- Concurrent immunosuppressants allowed\*
- Concurrent warfarin allowed provided INR level is stable for the past 4 weeks and expected to remain stable during observation and study treatment
- Concurrent iron supplements or folic acid allowed\*\*
- Concurrent low-molecular weight heparin allowed\*\* NOTE: \*Provided dose is stable for the past 26 weeks and during study observation and treatment
- NOTE: \*\*Provided dose is stable for the past 4 weeks and expected to remain stable (or decrease for corticosteroids) during study observation and treatment
Exclusion
Key Trial Info
Start Date :
November 1 2004
Trial Type :
INTERVENTIONAL
End Date :
June 1 2005
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT00112983
Start Date
November 1 2004
End Date
June 1 2005
Last Update
May 30 2013
Active Locations (1)
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1
Jonsson Comprehensive Cancer Center at UCLA
Los Angeles, California, United States, 90095-1678