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Completed

NCT Number: NCT01194804

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

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.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Eculizumab

Drug

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

Other names: Soliris

Primary outcomes

  1. 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 outcomes

  1. 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)

    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.

  2. 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)

  3. 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)

    Baseline is defined as the number of units transfused in 3 months prior to baseline

  4. 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)

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Collaborators

  • CMIC Co, Ltd. Japan

Registry information

Official study title

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

Important dates

Study start
2008
Primary completion
2010
Study completion
2011
First posted
Sep 3, 2010
Registry last updated
Sep 17, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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