Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

NCT ID: NCT01192399

Last Updated: 2018-03-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 29 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-11-30
• Study Completion Date: 2008-06-30

Brief Summary

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

Detailed 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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Eculizumab

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

Group Type: EXPERIMENTAL

Eculizumab

Intervention Type: BIOLOGICAL

Interventions

Eculizumab

Intervention Type: BIOLOGICAL

Other Intervention Names

Soliris

Eligibility Criteria

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

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Stephen Squinto, PhD

Role: STUDY_DIRECTOR

Alexion Pharmaceuticals, Inc.

References

Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Valentine ME, Khursigara G, Ozawa K, Omine M. Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial. Int J Hematol. 2011 Jan;93(1):36-46. doi: 10.1007/s12185-010-0748-9. Epub 2011 Jan 12.

Reference Type: RESULT

PMID: 21222185 (View on PubMed)

Other Identifiers

C07-001

Identifier Type: -

Identifier Source: org_study_id