Clinical trial NCT01192399: Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Official title: Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: Hemolytic Paroxysmal Nocturnal Hemoglobinuria
Eculizumab
PNH
Japan
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2010-09-01
Enrollment: 29
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Change From Baseline in Lactate Dehydrogenase
timeFrame: Baseline, Week 12
Secondary outcomes:
measure: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score
timeFrame: Baseline, Week 12
measure: Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count
timeFrame: Baseline, Week 12
measure: Number of Units of Packed Red Blood Cells (pRBCs) Transfused
timeFrame: 12 weeks pre-treatment, baseline, 12 weeks post-treatment
measure: Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)
timeFrame: Baseline to Week 12
measure: Change From Baseline in Plasma Free Hemoglobin
timeFrame: Baseline, Week 12
measure: Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status)
timeFrame: Baseline, Week 12
Other outcomes:
Locations:
Study officials:
affiliation: Alexion Pharmaceuticals, Inc.
name: Stephen Squinto, PhD
role: STUDY_DIRECTOR
References:
citation: 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 eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial. Int J Hematol. 2011 Jan;93(1):36-46. doi: 10.1007/s12185-010-0748-9. Epub 2011 Jan 12.