Trial · NCT00844545

Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS

NCT00844545, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2014-12-01

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS. Trials, record NCT00844545. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00844545

On this page

Trial

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

Title: Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS
NCT number: NCT00844545
Status: Completed
Phases: Phase 2
Enrollment: 16 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Atypical Hemolytic Uremic Syndrome
Interventions: Eculizumab
First posted: 2009-02-16
Last updated: 2015-07-23
Other registries' IDs: eudract:2008-006952-23
Results first posted: 2014-12-01

Source ClinicalTrials.gov

Registered outcomes11

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

List	Outcome	Time frame	Description
Primary	Platelet Count Change From Baseline to 26 Weeks	From Baseline to 26 weeks
Primary	Percentage of Patients With Platelet Count Normalization	Through 26 weeks	The primary objective of the study (per protocol) was to assess the effect of eculizumab to reduce TMA as measured by platelet count change from baseline (BL) during the Treatment Period (26 weeks) in patients with plasma therapy (PT)-resistant aHUS (protocol defined), including assessment of the proportion of patients who achieved Platelet Count Normalization from baseline through 26 weeks. Platelet Count Normalization was defined as the platelet count observed to be ≥150 x 10\^9/L on at least two consecutive measurements which span a period of at least four weeks.
Primary	Percentage of Patients With Hematologic Normalization	Through 26 weeks	Hematologic Normalization was defined as normalization of both platelet count and lactic dehydrogenase (LDH) sustained for at least two consecutive measurements which spanned a period of at least four weeks.
Secondary	Percentage of Patients With Complete TMA Response	Through 26 weeks	The proportion of patients who achieved a Complete TMA Response from baseline through 26 weeks of treatment with eculizumab was determined. Complete TMA Response was defined as Hematologic Normalization plus improvement in renal function (defined as as ≥25% reduction from baseline in serum creatinine), which was sustained for two consecutive measurements over a period of at least four weeks.
Secondary	TMA Intervention Rate	Through 26 weeks	TMA Intervention Rate (# PE/PI and # Dialysis Events/Patient/Day) in the eculizumab treatment period (from baseline through 26 weeks) for PE/PI and (from the fifteenth day following the first eculizumab dose through 26 weeks) for new dialysis events was compared with the TMA Intervention Rate during the pre-eculizumab treatment period.
Secondary	Platelet Count Change From Baseline to 156 Weeks	From Baseline to 156 Weeks
Secondary	Percentage of Patients With Platelet Count Normalization	Through End of Study, Median Exposure 100.29 Weeks	Platelet Count Normalization was defined as the platelet count observed to be ≥150 x 10\^9/L on at least two consecutive measurements which span a period of at least four weeks.
Secondary	Percentage of Patients With Hematologic Normalization	Through End of Study, Median Exposure 100.29 Weeks	Hematologic Normalization was defined as normalization of both platelet count and lactic dehydrogenase (LDH) sustained for at least two consecutive measurements which spanned a period of at least four weeks.
Secondary	Percentage of Patients With Complete TMA Response	Through End of Study, Median Exposure 100.29 Weeks	The proportion of patients who achieved a Complete TMA Response from baseline through end of the study was determined. Complete TMA Response was defined as Hematologic Normalization plus improvement in renal function (defined as ≥25% reduction from baseline in serum creatinine), which was sustained for two consecutive measurements over a period of at least four weeks.
Secondary	TMA Intervention Rate	Through End of Study, Median Exposure 100.29 Weeks	TMA Intervention Rate (# PE/PI and # Dialysis Events/Patient/Day) in the eculizumab treatment period (from baseline through end of the study) for PE/PI and (from the fifteenth day following the first eculizumab dose through end of the study) for new dialysis events was compared with the TMA Intervention Rate during the pre-eculizumab treatment period.

1 to 10 of 11Source ClinicalTrials.gov

See all 11 registered outcomes

Outcomes11

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Platelet Count Change From Baseline to 26 Weeks	From Baseline to 26 weeks	Change from baseline platelet counts were analyzed for the ITT population using a repeated measurement ANOVA model. A least squares (LS) mean for the change from baseline was produced for each study day for which a measurement of platelet count was scheduled. Significance of change was assessed at the 5% level at each time point.	10^9 cells/L	LEAST_SQUARES_MEAN
PRIMARY	Percentage of Patients With Platelet Count Normalization	Through 26 weeks	The Tabulations of the proportion of patients who achieved platelet count normalization through 26 weeks were performed for the ITT population. Exact binomial confidence intervals were produced for the analysis.	Percentage of Participants	NUMBER
PRIMARY	Percentage of Patients With Hematologic Normalization	Through 26 weeks	Tabulations of the proportion of patients who achieved a hematologic normalization through 26 weeks were performed for the ITT population. Exact binomial confidence intervals were produced for the analysis.	Percentage of Participants	NUMBER
SECONDARY	Percentage of Patients With Complete TMA Response	Through 26 weeks	Tabulations of the proportion of patients who achieved a complete TMA response from baseline through 26 weeks were performed. For this endpoint, for any relevant proportions, exact binomial confidence intervals were produced.	Percentage of Participants	NUMBER
SECONDARY	TMA Intervention Rate	Through 26 weeks	A signed rank test assessed differences in magnitudes of change in TMA intervention rate between the pre-eculizumab treatment period and during eculizumab treatment period for ITT population.	# events/patient/day	MEAN
SECONDARY	Platelet Count Change From Baseline to 156 Weeks	From Baseline to 156 Weeks	Change from baseline platelet counts were analyzed for the ITT population using a repeated measurement ANOVA model. A least squares (LS) mean for the change from baseline was produced for each study day for which a measurement of platelet count was scheduled. Significance of change was assessed at the 5% level at each time point.	10^9 cells/L	LEAST_SQUARES_MEAN
SECONDARY	Percentage of Patients With Platelet Count Normalization	Through End of Study, Median Exposure 100.29 Weeks	The Tabulations of the proportion of patients who achieved platelet count normalization through end of the study were performed for the ITT population. Exact binomial confidence intervals were produced for the analysis.	Percentage of Participants	NUMBER
SECONDARY	Percentage of Patients With Hematologic Normalization	Through End of Study, Median Exposure 100.29 Weeks	Tabulations of the proportion of patients who achieved a hematologic normalization through end of study were performed for the ITT population. Exact binomial confidence intervals were produced for the analysis.	Percentage of Participants	NUMBER
SECONDARY	Percentage of Patients With Complete TMA Response	Through End of Study, Median Exposure 100.29 Weeks	Tabulations of the proportion of patients who achieved a complete TMA response from baseline through end of study were performed. For this endpoint, for any relevant proportions, exact binomial confidence intervals were produced.	Percentage of Participants	NUMBER
SECONDARY	TMA Intervention Rate	Through End of Study, Median Exposure 100.29 Weeks	A signed rank test assessed differences in magnitudes of change in TMA intervention rate between the pre-eculizumab treatment period and during eculizumab treatment period for ITT population.	# events/patient/day	MEAN

1 to 10 of 11Source ClinicalTrials.gov

See all 11 outcomes

Results by group14

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

Outcome	Group	Value	Spread	Units
Platelet Count Change From Baseline to 26 Weeks	Eculizumab	65.18	95% Confidence Interval 37.01 to 93.36	10^9 cells/L
Percentage of Patients With Platelet Count Normalization	Eculizumab	82	95% Confidence Interval 57 to 96	Percentage of Participants
Percentage of Patients With Hematologic Normalization	Eculizumab	76	95% Confidence Interval 50 to 93	Percentage of Participants
Percentage of Patients With Complete TMA Response	Eculizumab	65	95% Confidence Interval 38 to 86	Percentage of Participants
TMA Intervention Rate	Eculizumab	0.04	Standard Deviation 0.10	# events/patient/day
Platelet Count Change From Baseline to 156 Weeks	Eculizumab	111.62	95% Confidence Interval 98.12 to 125.13	10^9 cells/L
Percentage of Patients With Platelet Count Normalization	Eculizumab	88	95% Confidence Interval 64 to 99	Percentage of Participants
Percentage of Patients With Hematologic Normalization	Eculizumab	88	95% Confidence Interval 64 to 99	Percentage of Participants
Percentage of Patients With Complete TMA Response	Eculizumab	76	95% Confidence Interval 50 to 93	Percentage of Participants
TMA Intervention Rate	Eculizumab	0.04	Standard Deviation 0.11	# events/patient/day

1 to 10 of 14Source ClinicalTrials.gov

See all 14 results by group

Participant flow12

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

Period	Milestone	Group	Participants
Screening Period	STARTED	Eculizumab	17
Screening Period	COMPLETED	Eculizumab	17
Screening Period	NOT COMPLETED	Eculizumab	0
Treatment Period (26 Weeks)	STARTED	Eculizumab	17
Treatment Period (26 Weeks)	COMPLETED	Eculizumab	15
Treatment Period (26 Weeks)	NOT COMPLETED	Eculizumab	2
Extension Treatment Period	STARTED	Eculizumab	13
Extension Treatment Period	COMPLETED	Eculizumab	11
Extension Treatment Period	NOT COMPLETED	Eculizumab	2
Post Treatment Period	STARTED	Eculizumab	6

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events233

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	6	17
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Eculizumab	1	17
other	Leukopenia	Blood and lymphatic system disorders	Eculizumab	3	17
other	Lymphopenia	Blood and lymphatic system disorders	Eculizumab	1	17
other	Neutropenia	Blood and lymphatic system disorders	Eculizumab	1	17
other	Thrombocytopenia	Blood and lymphatic system disorders	Eculizumab	1	17
other	Bradycardia	Cardiac disorders	Eculizumab	2	17
other	Pericardial effusion	Cardiac disorders	Eculizumab	1	17
other	Cerumen impaction	Ear and labyrinth disorders	Eculizumab	1	17
other	Vertigo	Ear and labyrinth disorders	Eculizumab	1	17

1 to 10 of 233Source ClinicalTrials.gov

See all 233 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Eculizumab
Total, other adverse events	Eculizumab	17	17
Total, serious adverse events	Eculizumab	17	17

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
23738544	DERIVED	PubMed
33783815	DERIVED	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/NCT00844545

GET https://conductscience.org/api/trials/v1/NCT00844545/events?page=2

API reference For AI assistants