Trial · NCT00122317

Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT00122317, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2017-06-05

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH). Trials, record NCT00122317. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00122317

On this page

Trial

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

Title: Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT number: NCT00122317
Status: Completed
Phases: Phase 3
Enrollment: 187 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Paroxysmal Hemoglobinuria, Nocturnal
Interventions: eculizumab
First posted: 2005-07-22
Last updated: 2018-03-13
Results first posted: 2017-06-05

Source ClinicalTrials.gov

Registered outcomes4

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

List	Outcome	Time frame	Description
Primary	Incidence of Treatment-emergent Adverse Events	From time of consent to a maximum of 2.5 years of study treatment
Secondary	Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve	From time of first infusion through 24 months of study treatment
Secondary	Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline	From time of first infusion through 24 months of study treatment	The FACIT-Fatigue scale 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	Incidence of Thrombosis After Eculizumab Infusion	From time of first ever dose through last dose (up to 24 months of study treatment)	Thrombosis was defined as occurrence of major adverse vascular events

Source ClinicalTrials.gov

Outcomes4

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

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Incidence of Treatment-emergent Adverse Events	From time of consent to a maximum of 2.5 years of study treatment	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve	From time of first infusion through 24 months of study treatment	U/L x Day	MEAN
SECONDARY	Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline	From time of first infusion through 24 months of study treatment	units on a scale	MEAN
SECONDARY	Incidence of Thrombosis After Eculizumab Infusion	From time of first ever dose through last dose (up to 24 months of study treatment)	Number of events per 100 patient-years	NUMBER

Source ClinicalTrials.gov

Results by group5

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

Outcome	Group	Category	Value	Spread	Units
Incidence of Treatment-emergent Adverse Events	Eculizumab		182		Participants
Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve	Eculizumab		-365185.8	Standard Deviation 210407.1	U/L x Day
Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline	Eculizumab		9.24	Standard Deviation 12.42	units on a scale
Incidence of Thrombosis After Eculizumab Infusion	Eculizumab	Prior to first eculizumab dose	7.82		Number of events per 100 patient-years
Incidence of Thrombosis After Eculizumab Infusion	Eculizumab	From first dose of eculizumab through last dose	1.99		Number of events per 100 patient-years

Source ClinicalTrials.gov

Participant flow3

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Eculizumab	187
Overall Study	COMPLETED	Eculizumab	176
Overall Study	NOT COMPLETED	Eculizumab	11

Source ClinicalTrials.gov

Adverse events113

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	Diarrhoea	Gastrointestinal disorders	Eculizumab	44	187
other	Nausea	Gastrointestinal disorders	Eculizumab	39	187
other	Vomiting	Gastrointestinal disorders	Eculizumab	38	187
other	Abdominal Pain	Gastrointestinal disorders	Eculizumab	25	187
other	Abdominal Pain Upper	Gastrointestinal disorders	Eculizumab	18	187
other	Constipation	Gastrointestinal disorders	Eculizumab	16	187
other	Dyspepsia	Gastrointestinal disorders	Eculizumab	14	187
other	Influenza like Illness	General disorders	Eculizumab	29	187
other	Pyrexia	General disorders	Eculizumab	18	187
other	Oedema Peripheral	General disorders	Eculizumab	12	187

1 to 10 of 113Source ClinicalTrials.gov

See all 113 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Eculizumab	3	187
Total, other adverse events	Eculizumab	180	187
Total, serious adverse events	Eculizumab	57	187

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
17702897	DERIVED

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/NCT00122317

GET https://conductscience.org/api/trials/v1/NCT00122317/events?page=2

API reference For AI assistants