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

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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01194804

Enrollment

Registered

2010-09-03

Start date

2008-04-30

Completion date

2011-03-31

Last updated

2019-09-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

Hemolytic Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, PNH

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.

Interventions

DRUGEculizumab

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

12 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame	Description
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)	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.
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)	Baseline is defined as the number of units transfused in 3 months prior to baseline
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)	—

Participant flow

Participants by arm

Arm	Count
Eculizumab Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol 900 mg intravenous every 14 days.	27
Total	27

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Withdrawal by Subject	1

Baseline characteristics

Characteristic	Eculizumab
Age, Continuous	48.3 years STANDARD_DEVIATION 12.35
Race/Ethnicity, Customized Japanese	27 Participants
Region of Enrollment Japan	27 Participants
Sex: Female, Male Female	14 Participants
Sex: Female, Male Male	13 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 27
other Total, other adverse events	26 / 27
serious Total, serious adverse events	12 / 27