E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

Official title:

Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01194804
• Other study ID #: E07-001
• Status: Completed
• Phase: Phase 2
• Start date: April 2008
• Est. completion date: March 2011

Study information

• Verified date: September 2019
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study. In addition, pharmacokinetic and pharmacodynamic assessments of eculizumab were conducted.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. completion date: March 2011
• Est. primary completion date: September 2010
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Patients must have fully completed C07-001 study 12-week treatment period

Exclusion Criteria:

• Patients who terminated early from the C07-001 study

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• Eculizumab
Each vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days.

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals	CMIC Co, Ltd. Japan

References & Publications (1)

Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Ozawa K, Omine M. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Lactate Dehydrogenase		52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Secondary	Change From Baseline in FACIT-Fatigue Scale Total Score	The FACIT-Fatigue scale, Version 4.0, is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.	52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Secondary	Change From Baseline in PNH Red Blood Cell (RBC) Count		52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Secondary	Change From Baseline in Number of Units of Packed RBCs Transfused	Baseline is defined as the number of units transfused in 3 months prior to baseline	52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Secondary	Change From Baseline in Plasma Free Hemoglobin		52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)