Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Alexion Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Treatments: Biological: Eculizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT01192399

C07-001

Details and patient eligibility

About

A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.

Full description

The objective of this study was to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH. In addition, the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in Japanese patients were assessed and compared to that of non-Japanese patients who had participated earlier studies of eculizumab treatment in hemolytic PNH.

Enrollment: 29 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Japanese individuals at least 12 years of age
• Diagnosis of PNH > 6 months
• At least one transfusion in the past 2 years for anemia or anemia-related symptoms
• LDH level ≥ 1.5 x upper limit of normal within 12 weeks
• Presence of a glycosylphosphatidylinositol (GPI)-deficient red blood cell (RBC) clone (type III cells) by flow cytometry of ≥10%
• Negative serum pregnancy test for women of child-bearing potential

Exclusion criteria

• Platelet count < 30,000/µL
• Absolute neutrophil count ≤ 500/µL
• Known or suspected hereditary complement deficiency
• History of hematopoietic stem cell transplant
• History of meningococcal disease

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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29 participants in 1 patient group

Eculizumab

Experimental group

Description:

Eculizumab intravenous infusions every week x 4 doses, then 900 mg 1 week later for 1 dose, then 900 mg every 2 weeks for 4 doses

Treatment:

Biological: Eculizumab