Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

Official title:

Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01192399
• Other study ID #: C07-001
• Status: Completed
• Phase: Phase 2
• First received: August 30, 2010
• Last updated: February 11, 2018
• Start date: November 2007
• Est. completion date: June 2008

Study information

• Verified date: February 2018
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.

Description:

The objective of this study was to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH. In addition, the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in Japanese patients were assessed and compared to that of non-Japanese patients who had participated earlier studies of eculizumab treatment in hemolytic PNH.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 29
• Est. completion date: June 2008
• Est. primary completion date: June 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Japanese individuals at least 12 years of age

• Diagnosis of PNH > 6 months

• At least one transfusion in the past 2 years for anemia or anemia-related symptoms

• LDH level = 1.5 x upper limit of normal within 12 weeks

• Presence of a glycosylphosphatidylinositol (GPI)-deficient red blood cell (RBC) clone (type III cells) by flow cytometry of =10%

• Negative serum pregnancy test for women of child-bearing potential

Exclusion Criteria:

• Platelet count < 30,000/µL

• Absolute neutrophil count = 500/µL

• Known or suspected hereditary complement deficiency

• History of hematopoietic stem cell transplant

• History of meningococcal disease

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Biological:
• Eculizumab

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

References & Publications (1)

Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Valentine ME, Khursigara G, Ozawa K, Omine M. Safety and efficacy of the terminal complement inhibitor eculizu — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Lactate Dehydrogenase		Baseline, Week 12
Secondary	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score	The FACIT-Fatigue scale, Version 4.0, is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.	Baseline, Week 12
Secondary	Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count		Baseline, Week 12
Secondary	Number of Units of Packed Red Blood Cells (pRBCs) Transfused	Comparison of number of units of pRBCs transfused in the 12 weeks prior to the first dose of eculizumab, and between baseline and 12 weeks after the first dose of eculizumab	12 weeks pre-treatment, baseline, 12 weeks post-treatment
Secondary	Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)		Baseline to Week 12
Secondary	Change From Baseline in Plasma Free Hemoglobin		Baseline, Week 12
Secondary	Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status)	The European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) incorporates 5 functional scales (physical, role, cognitive, emotional, and social), 3 symptom scales (fatigue, pain, and nausea and vomiting), a global health status scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of the disease. All of the scales and single-item measures range in score from 0 to 100. For global health status, a high score represents a high quality of life.	Baseline, Week 12