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

This study has been completed.
Sponsor:
Collaborator:
CMIC Co, Ltd.
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01192399
First received: August 30, 2010
Last updated: April 4, 2012
Last verified: April 2012
  Purpose

Assess the safety and efficacy of Eculizumab in hemolytic PNH patients.


Condition Intervention Phase
Paroxysmal Nocturnal Hemoglobinuria
Drug: Eculizumab
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change of total scores of FACIT-Fatigue scale [ Time Frame: Baseline to Study Week 12 ] [ Designated as safety issue: No ]
  • Change of PNH RBC count [ Time Frame: Baseline to Study Week 12 ] [ Designated as safety issue: No ]
  • Change of number of units of Packed RBCs transfused [ Time Frame: Baseline through study period ] [ Designated as safety issue: No ]
  • AUC for change of LDH [ Time Frame: From baseline ] [ Designated as safety issue: No ]
  • Change of plasma free hemoglobin [ Time Frame: From baseline to Study Week 12 ] [ Designated as safety issue: No ]
  • Change of EORTC QLQ-C30 score [ Time Frame: From baseline to Study Week 12 ] [ Designated as safety issue: No ]

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
Treatment with Eculizumab for patients with PNH
Drug: Eculizumab
Each vial contains 30 mL of 10 mg/mL eculizumab; initial dose of 600 mg weekly for 4 weeks, followed by 900 mg one week later, then maintenance of 900 mg every 2 weeks for duration of 12 weeks.
Other Name: Soliris

Detailed Description:

The objective of this study is to assess the safety and efficacy of Eculizumab in hemolytic PNH patients. In addition, pharmacokinetic and pharmacodynamic assessments of Eculizumab will be conducted.

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
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
CMIC Co, Ltd.
Investigators
Study Director: Stephen Squinto, PhD Alexion Pharmaceuticals
  More Information

No publications provided

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: April 4, 2012
Health Authority: Japan: Pharmaceuticals and Medical Devices Agency

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 September 18, 2014