Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00122330
• Date of registration: 18/07/2005
• Prospective Registration: No
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
• Scientific title: Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
• Date of first enrolment: October 2004
• Target sample size: 75
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00122330
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Educational/Counseling/Training
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Sweden, Switzerland, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Must have required at least 4 transfusions in the past 12 months

- PNH type III red blood cell (RBC) clone by flow cytometry of >10%

- Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal

- Platelet count > 100,000/mm3

- Patient taking erythropoietin must be on a stable dose for at least 26 weeks

- Patient taking immunosuppressants must be on a stable dose for at least 26 weeks

- Patient taking corticosteroids must be on a stable dose for at least 4 weeks

- Patient taking coumadin must be at a stable INR for at least 4 weeks

- Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks

- Willing and able to give written informed consent

- Must avoid conception

Exclusion Criteria:

- Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl

- Absolute neutrophil count <500/ul

- Active bacterial infection

- Hereditary complement deficiency

- Participation in any other investigational drug trial or exposure to other
investigational agent, device, or procedures within 30 days

- Pregnant, breast-feeding, or intending to conceive

- History of meningococcal disease

- History of bone marrow transplantation



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Hemoglobinuria, Paroxysmal

Intervention(s)

Drug: eculizumab

Secondary ID(s)

TRIUMPH

Source(s) of Monetary Support

Please refer to primary and secondary sponsors