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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01194804
First Posted: September 3, 2010
Last Update Posted: February 17, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
CMIC Co, Ltd. Japan
Information provided by (Responsible Party):
Alexion Pharmaceuticals
  Purpose
The objective of this study is to assess the long term safety of Eculizumab in hemolytic PNH patients who completed the 4 weeks screening and 12 weeks treatment period of C07-001 study. In addition, pharmacokinetic and pharmacodynamic assessments of Eculizumab will be conducted.

Condition Intervention Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: Eculizumab Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
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

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Safety and Efficacy of Eculizumab treatment [ Time Frame: Baseline through Week 26 ]

Secondary Outcome Measures:
  • Change of LDH [ Time Frame: From baseline ]
  • Change of total scores of FACIT-Fatigue scale [ Time Frame: From baseline ]
  • Change of PNH RBC [ Time Frame: From baseline ]
  • Number of units of Packed RBCs transfused [ Time Frame: From baseline ]
  • Lactate dehydrogenase (LDH) area under the curve (AUC) [ Time Frame: From baseline ]
  • Change of plasma free Hemoglobin [ Time Frame: From baseline ]
  • Change of total EORTC QLQ-C30 score [ Time Frame: From baseline ]

Enrollment: 27
Study Start Date: April 2008
Study Completion Date: March 2011
Primary Completion Date: September 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
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
  Contacts and Locations
No Contacts or Locations Provided
  More Information

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01194804     History of Changes
Other Study ID Numbers: E07-001
First Submitted: August 30, 2010
First Posted: September 3, 2010
Last Update Posted: February 17, 2017
Last Verified: February 2017

Keywords provided by Alexion Pharmaceuticals:
Hemolytic Paroxysmal Nocturnal Hemoglobinuria
Eculizumab
PNH

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases