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C07-001: Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01192399
First received: August 30, 2010
Last updated: February 15, 2017
Last verified: February 2017
  Purpose
The objective of this study was to assess the safety and efficacy of eculizumab in hemolytic PNH patients. In addition, the pharmacokinetics (PK) and pharmacodynamic (PD) response of eculizumab was assessed and compared to overseas studies of eculizumab treatment in hemolytic PNH patients.

Condition Intervention Phase
Paroxysmal Nocturnal Hemoglobinuria
Biological: Eculizumab
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Change of LDH [ Time Frame: Baseline to Study Week 12 ]

Secondary Outcome Measures:
  • Change of total scores of FACIT-Fatigue scale [ Time Frame: Baseline to Study Week 12 ]
  • Change of PNH RBC count [ Time Frame: Baseline to Study Week 12 ]
  • Change of number of units of Packed RBCs transfused [ Time Frame: Baseline through study period ]
  • AUC for change of LDH [ Time Frame: From baseline ]
  • Change of plasma free hemoglobin [ Time Frame: From baseline to Study Week 12 ]
  • Change of EORTC QLQ-C30 score [ Time Frame: From baseline to Study Week 12 ]

Enrollment: 29
Study Start Date: November 2007
Study Completion Date: October 2008
Primary Completion Date: June 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eculizumab
600 mg of eculizumab as intravenous (IV) infusion once a week for 4 doses, followed by 900 mg eculizumab IV infusion 1 week later for 1 dose, then 900 mg eculizumab IV infusion every 2 weeks for 4 doses.
Biological: Eculizumab
Subjects were treated with open label Eculizumab and following the dose described in the arm/group description.
Other Name: Soliris

  Eligibility

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

Inclusion Criteria:

  • Japanese individuals at least 12 years of age
  • Diagnosis of PNH > 6 months
  • One transfusion in the past two years for anemia or anemia-related symptoms
  • LDH level >/= 1.5 x upper limit of normal within 12 weeks

Exclusion Criteria:

  • Platelet count < 30,000/microliters
  • Known or suspected hereditary complement deficiency
  • History of hematopoietic stem cell transplant
  • History of meningococcal disease
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01192399

Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
Study Director: Stephen Squinto, PhD Alexion Pharmaceuticals
  More Information

Publications:
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01192399     History of Changes
Other Study ID Numbers: C07-001 
Study First Received: August 30, 2010
Last Updated: February 15, 2017
Individual Participant Data  
Plan to Share IPD: No

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

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

ClinicalTrials.gov processed this record on February 20, 2017