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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01194804
• Date of registration: 30/08/2010
• Prospective Registration: No
• Primary sponsor: Alexion Pharmaceuticals
• Public title: E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
• Scientific title: Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol
• Date of first enrolment: April 2008
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01194804
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Key inclusion & exclusion criteria

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



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Intervention(s)

Drug: Eculizumab

Primary Outcome(s)

Change From Baseline in Lactate Dehydrogenase [Time Frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)]

Secondary Outcome(s)

Change From Baseline in Number of Units of Packed RBCs Transfused [Time Frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)]

Change From Baseline in FACIT-Fatigue Scale Total Score [Time Frame: 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 [Time Frame: 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 [Time Frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)]

Secondary ID(s)

E07-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

CMIC Co, Ltd. Japan

Results

• Results available: Yes
• Date Posted: 12/07/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01194804