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

NCT01194804 | Completed Phase 2 Results

• 1 other identifier: E07-001
• Study Type: interventional
• Target: 27
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

Hemolytic Paroxysmal Nocturnal Hemoglobinuria; Eculizumab; PNH

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Change From Baseline in FACIT-Fatigue Scale Total Score

  52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)

• 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)

• Change From Baseline in Number of Units of Packed RBCs Transfused

  52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)

• 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)

Study Arms (1)

Eculizumab

EXPERIMENTAL

Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol

Drug: Eculizumab

Interventions

Eculizumab DRUG

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

Also known as: Soliris

Eculizumab

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Patients who terminated early from the C07-001 study

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• CMIC Co, Ltd. Japan: collaborator

Related 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 J Hematol. 2013 Oct;98(4):406-16. doi: 10.1007/s12185-013-1404-y. Epub 2013 Aug 11.

  PMID: 23934275 RESULT

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2010
• First Posted: September 3, 2010
• Study Start: April 1, 2008
• Primary Completion: September 1, 2010
• Study Completion: March 1, 2011
• Last Updated: September 17, 2019
• Results First Posted: July 12, 2019

Record last verified: 2019-09

Data Sharing

• IPD Sharing: Will not share