Phase 2 N=29 Treatment

Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

Paroxysmal Nocturnal Hemoglobinuria

Bottom Line

View on ClinicalTrials.gov: NCT01192399 ↗

Enrolled (actual)

Serious AEs

3.5%

Results posted

Mar 2018

Primary outcome: Primary: Change From Baseline in Lactate Dehydrogenase — 1845.1; 398.7; -1446.4 Units/Liter — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Eculizumab (Biological)
Age: Pediatric, Adult, Older Adult · 12+ yrs
Sex: All
Sponsor: Alexion Pharmaceuticals, Inc.
Primary completion: Jun 2008

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline in Lactate Dehydrogenase	1845.1; 398.7; -1446.4	<0.0001 sig
SECONDARY Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score	38.5; 4.1	—
SECONDARY Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count	1.3; 1.8; 0.5	—
SECONDARY Number of Units of Packed Red Blood Cells (pRBCs) Transfused	5.2; 1.5; -3.7	—
SECONDARY Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)	-113540.5	—
SECONDARY Change From Baseline in Plasma Free Hemoglobin	22.6; -19.8	—
SECONDARY Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status)	8.9	—

Summary

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01192399). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.