E07-001: Safety and Efficacy Extension 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:

NCT01194804

First received: August 30, 2010

Last updated: December 5, 2011

Last verified: December 2011

History of Changes

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 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: Extension Study to C07-001 Protocol

Resource links provided by NLM:

Genetics Home Reference related topics: paroxysmal nocturnal hemoglobinuria

Drug Information available for: Eculizumab

U.S. FDA Resources

Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:

Safety and Efficacy of Eculizumab treatment [ Time Frame: Baseline through Week 26 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change of LDH [ Time Frame: From baseline ] [ Designated as safety issue: No ]
Change of total scores of FACIT-Fatigue scale [ Time Frame: From baseline ] [ Designated as safety issue: No ]
Change of PNH RBC [ Time Frame: From baseline ] [ Designated as safety issue: No ]
Number of units of Packed RBCs transfused [ Time Frame: From baseline ] [ Designated as safety issue: No ]
Lactate dehydrogenase (LDH) area under the curve (AUC) [ Time Frame: From baseline ] [ Designated as safety issue: No ]
Change of plasma free Hemoglobin [ Time Frame: From baseline ] [ Designated as safety issue: No ]
Change of total EORTC QLQ-C30 score [ Time Frame: From baseline ] [ Designated as safety issue: No ]

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

Ages Eligible for Study:	12 Years and older
Genders Eligible for Study:	Both
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

No publications provided

Responsible Party:	Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT01194804 History of Changes
Other Study ID Numbers:	E07-001
Study First Received:	August 30, 2010
Last Updated:	December 5, 2011
Health Authority:	Japan: Pharmaceuticals and Medical Devices Agency

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

ClinicalTrials.gov processed this record on June 18, 2013

To Top