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

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ClinicalTrials.gov Identifier: NCT01194804
Recruitment Status : Completed
First Posted : September 3, 2010
Results First Posted : July 12, 2019
Last Update Posted : September 17, 2019
Sponsor:
Collaborator:
CMIC Co, Ltd. Japan
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: Eculizumab Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol
Study Start Date : April 2008
Actual Primary Completion Date : September 2010
Actual Study Completion Date : March 2011


Arm Intervention/treatment
Experimental: Eculizumab
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
Drug: Eculizumab
Each vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days.
Other Name: Soliris




Primary Outcome Measures :
  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 Outcome Measures :
  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) ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Publications of Results:
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01194804     History of Changes
Other Study ID Numbers: E07-001
First Posted: September 3, 2010    Key Record Dates
Results First Posted: July 12, 2019
Last Update Posted: September 17, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion Pharmaceuticals:
Hemolytic Paroxysmal Nocturnal Hemoglobinuria
Eculizumab
PNH
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases