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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
Last Update Posted : February 17, 2017
Sponsor:
Collaborator:
CMIC Co, Ltd. Japan
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
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
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. Safety and Efficacy of Eculizumab treatment [ Time Frame: Baseline through Week 26 ]

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


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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

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
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