Trial · NCT01192399

Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

NCT01192399, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2018-03-13

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients. Trials, record NCT01192399. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01192399

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
NCT number: NCT01192399
Status: Completed
Phases: Phase 2
Enrollment: 29 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Interventions: Eculizumab
First posted: 2010-09-01
Last updated: 2018-03-13
Results first posted: 2018-03-13

Source ClinicalTrials.gov

Registered outcomes7

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
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	Baseline, Week 12	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.
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	12 weeks pre-treatment, baseline, 12 weeks post-treatment	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
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)	Baseline, Week 12	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.

Source ClinicalTrials.gov

Outcomes7

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Change From Baseline in Lactate Dehydrogenase	Baseline, Week 12	Units/Liter	MEAN
SECONDARY	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score	Baseline, Week 12	units on a scale	MEAN
SECONDARY	Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count	Baseline, Week 12	cells x 10^12/L	MEAN
SECONDARY	Number of Units of Packed Red Blood Cells (pRBCs) Transfused	12 weeks pre-treatment, baseline, 12 weeks post-treatment	Units	MEAN
SECONDARY	Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)	Baseline to Week 12	U/L x Day	MEAN
SECONDARY	Change From Baseline in Plasma Free Hemoglobin	Baseline, Week 12	mg/dL	MEAN
SECONDARY	Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status)	Baseline, Week 12	units on a scale	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group15

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in Lactate Dehydrogenase	Eculizumab	Baseline	1845.1	Standard Error 115.34	Units/Liter
Change From Baseline in Lactate Dehydrogenase	Eculizumab	Week 12	398.7	Standard Error 99.15	Units/Liter
Change From Baseline in Lactate Dehydrogenase	Eculizumab	Change from Baseline at Week 12	-1446.4	Standard Error 138.68	Units/Liter
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score	Eculizumab	Baseline	38.5	Standard Error 1.88	units on a scale
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score	Eculizumab	Change from Baseline at Week 12	4.1	Standard Error 2.25	units on a scale
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count	Eculizumab	Baseline	1.3	Standard Error 0.14	cells x 10^12/L
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count	Eculizumab	Week 12	1.8	Standard Error 0.19	cells x 10^12/L
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count	Eculizumab	Change from Baseline at Week 12	0.5	Standard Error 0.10	cells x 10^12/L
Number of Units of Packed Red Blood Cells (pRBCs) Transfused	Eculizumab	12 Weeks Pre-Dose	5.2	Standard Error 1.04	Units
Number of Units of Packed Red Blood Cells (pRBCs) Transfused	Eculizumab	12 Weeks Post-Dose	1.5	Standard Error 0.67	Units

1 to 10 of 15Source ClinicalTrials.gov

See all 15 results by group

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Eculizumab	29
Overall Study	COMPLETED	Eculizumab	27
Overall Study	NOT COMPLETED	Eculizumab	2

Source ClinicalTrials.gov

Adverse events18

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	Eculizumab	2	29
other	Leukopenia	Blood and lymphatic system disorders	Eculizumab	2	29
other	Tinnitis	Ear and labyrinth disorders	Eculizumab	2	29
other	Conjunctival haemorrhage	Eye disorders	Eculizumab	2	29
other	Nausea	Gastrointestinal disorders	Eculizumab	6	29
other	Diarrhoea	Gastrointestinal disorders	Eculizumab	4	29
other	Vomiting	Gastrointestinal disorders	Eculizumab	3	29
other	Chest discomfort	General disorders	Eculizumab	2	29
other	Fatigue	General disorders	Eculizumab	2	29
other	Pyrexia	General disorders	Eculizumab	2	29

1 to 10 of 18Source ClinicalTrials.gov

See all 18 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Eculizumab	0	29
Total, other adverse events	Eculizumab	28	29
Total, serious adverse events	Eculizumab	1	29

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
21222185	RESULT

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01192399

GET https://conductscience.org/api/trials/v1/NCT01192399/registered

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