Trial · NCT01193348
An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome
NCT01193348, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2015-04-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome. Trials, record NCT01193348. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01193348
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome
- NCT number
- NCT01193348
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 22 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Atypical Hemolytic-Uremic Syndrome
- Interventions
- Eculizumab
- First posted
- 2010-09-01
- Last updated
- 2015-04-29
- Results first posted
- 2015-04-29
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Patients With Complete TMA Response | Through 26 weeks | Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline which was sustained for at least two consecutive measurements obtained at least four weeks apart). |
| Secondary | Proportion of Patients With Complete Hematologic Response | Through 26 weeks | Proportion of Patients with Complete Hematologic response through 26 weeks of treatment was determined and defined by normalization of platelet count and LDH sustained for at least two consecutive measurements obtained at least four weeks apart. |
| Secondary | Proportion of Patients With Platelet Count Normalization | Through 26 weeks | Proportion of Patients with Platelet Count Normalization through 26 weeks of treatment was determined and defined as the platelet count observed to be ≥ 150 x 109/L on at least two consecutive measurements which span a period of at least four weeks. |
| Secondary | Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement | Through 26 weeks | Proportion of Patients with Estimated Glomerular Filtration Rate (eGFR) Improvement was determined and defined as an increase in eGFR by ≥ 15 mL/min/1.73m2 from baseline, sustained for at least two consecutive measurements obtained at least four weeks apart. |
| Secondary | Platelet Count Change From Baseline to 26 Weeks | Through 26 weeks | |
| Secondary | Proportion of Patients With Complete TMA Response | Through End of Study, Median Exposure 55 Weeks | Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline which was sustained for at least two consecutive measurements obtained at least four weeks apart). |
| Secondary | Proportion of Patients With Complete Hematologic Response | Through End of Study, Median Exposure 55 Weeks | Proportion of Patients with Complete Hematologic response through end of study was determined and defined by normalization of platelet count and LDH sustained for at least two consecutive measurements obtained at least four weeks apart. |
| Secondary | Proportion of Patients With Platelet Count Normalization | Through End of Study, Median Exposure 55 Weeks | Proportion of Patients with Platelet Count Normalization through end of study was determined and defined as the platelet count observed to be ≥ 150 x 109/L on at least two consecutive measurements which span a period of at least four weeks |
| Secondary | Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement | Through End of Study, Median Exposure 55 Weeks | Proportion of Patients with Estimated Glomerular Filtration Rate (eGFR) Improvement was determined and defined as an increase in eGFR by ≥ 15 mL/min/1.73m2 from baseline, sustained for at least two consecutive measurements obtained at least four weeks apart. |
| Secondary | Platelet Count Change From Baseline to 52 Weeks | Through 52 Weeks |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion of Patients With Complete TMA Response | Through 26 weeks | The tabulations of the proportions of patients with Complete TMA Response through 26 weeks were performed for the ITT population. Exact binomial two-sided 95% CI using the Clopper-Pearson method for the responder rate was presented. | Percentage of Participants | NUMBER |
| SECONDARY | Proportion of Patients With Complete Hematologic Response | Through 26 weeks | The tabulations of the proportions of patients with Complete Hematologic Response through 26 weeks were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized. | Percentage of Participants | NUMBER |
| SECONDARY | Proportion of Patients With Platelet Count Normalization | Through 26 weeks | The tabulations of the proportions of patients with Platelet Count Normalization through 26 weeks were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized. | Percentage of Participants | NUMBER |
| SECONDARY | Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement | Through 26 weeks | The tabulations of the proportions of patients with Estimated Glomerular Filtration Rate (eGFR) Improvement through 26 weeks were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized. | Percentage of Participants | NUMBER |
| SECONDARY | Platelet Count Change From Baseline to 26 Weeks | Through 26 weeks | Change from baseline platelet counts were analyzed for the ITT population using a repeated measures ANOVA model. The estimated LS means of change from baseline at each post-baseline visit alongside with 95% CIs and P-values were calculated, as were the parameter estimates for the covariates and their associated P-values. | 10^9 cells/L | LEAST_SQUARES_MEAN |
| SECONDARY | Proportion of Patients With Complete TMA Response | Through End of Study, Median Exposure 55 Weeks | The tabulations of the proportions of patients with Complete TMA Response through end of study were performed for the ITT population. Exact binomial two-sided 95% CI using the Clopper-Pearson method for the responder rate was presented. | Percentage of Participants | NUMBER |
| SECONDARY | Proportion of Patients With Complete Hematologic Response | Through End of Study, Median Exposure 55 Weeks | The tabulations of the proportions of patients with Complete Hematologic Response through end of study were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized. | Percentage of Participants | NUMBER |
| SECONDARY | Proportion of Patients With Platelet Count Normalization | Through End of Study, Median Exposure 55 Weeks | The tabulations of the proportions of patients with Platelet Count Normalization through end of study were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized. | Percentage of Participants | NUMBER |
| SECONDARY | Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement | Through End of Study, Median Exposure 55 Weeks | The tabulations of the proportions of patients with eGFR Improvement through end of study were performed for the ITT population. The number of patients with response and responder rate, along with an exact two-sided 95% CI were summarized. | Percentage of Participants | NUMBER |
| SECONDARY | Platelet Count Change From Baseline to 52 Weeks | Through 52 Weeks | Change from baseline platelet counts were analyzed for the ITT population using a repeated measures ANOVA model. The estimated LS means of change from baseline at each post-baseline visit alongside with 95% CIs and P-values were calculated, as were the parameter estimates for the covariates and their associated P-values. | 10^9 cells/L | LEAST_SQUARES_MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group30
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion of Patients With Complete TMA Response | Eculizumab | 63.6 | 95% Confidence Interval 40.7 to 82.8 | Percentage of Participants | |
| Proportion of Patients With Complete Hematologic Response | Eculizumab | 81.8 | 95% Confidence Interval 59.7 to 94.8 | Percentage of Participants | |
| Proportion of Patients With Platelet Count Normalization | Eculizumab | 95.5 | 95% Confidence Interval 77.2 to 99.9 | Percentage of Participants | |
| Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement | Eculizumab | 86.4 | 95% Confidence Interval 65.1 to 97.1 | Percentage of Participants | |
| Platelet Count Change From Baseline to 26 Weeks | Eculizumab | 204.96 | 95% Confidence Interval 164.44 to 245.49 | 10^9 cells/L | |
| Proportion of Patients With Complete TMA Response | Eculizumab | 68.2 | 95% Confidence Interval 45.1 to 86.1 | Percentage of Participants | |
| Proportion of Patients With Complete Hematologic Response | Eculizumab | 90.9 | 95% Confidence Interval 70.8 to 98.9 | Percentage of Participants | |
| Proportion of Patients With Platelet Count Normalization | Eculizumab | 95.5 | 95% Confidence Interval 77.2 to 99.9 | Percentage of Participants | |
| Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement | Eculizumab | 86.4 | 95% Confidence Interval 65.1 to 97.1 | Percentage of Participants | |
| Platelet Count Change From Baseline to 52 Weeks | Eculizumab | 165.43 | 95% Confidence Interval 98.43 to 232.46 | 10^9 cells/L |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Screening Period | STARTED | Eculizumab | 27 | |
| Screening Period | COMPLETED | Eculizumab | 22 | |
| Screening Period | NOT COMPLETED | Eculizumab | 5 | |
| Treatment Period (26 Weeks) | STARTED | Eculizumab | 22 | |
| Treatment Period (26 Weeks) | COMPLETED | Eculizumab | 19 | |
| Treatment Period (26 Weeks) | NOT COMPLETED | Eculizumab | 3 | |
| Extension Treatment Period | STARTED | Eculizumab | 17 | |
| Extension Treatment Period | COMPLETED | Eculizumab | 16 | |
| Extension Treatment Period | NOT COMPLETED | Eculizumab | 1 | |
| Post Treatment Period (Patients Who Disc | STARTED | Eculizumab | 10 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events149
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 | Events |
|---|---|---|---|---|---|---|
| other | ANAEMIA | Blood and lymphatic system disorders | Eculizumab | 1 | 22 | |
| other | LEUKOPENIA | Blood and lymphatic system disorders | Eculizumab | 1 | 22 | |
| other | LYMPHOPENIA | Blood and lymphatic system disorders | Eculizumab | 1 | 22 | |
| other | NEUTROPENIA | Blood and lymphatic system disorders | Eculizumab | 1 | 22 | |
| other | LYMPHADENOPATHY | Blood and lymphatic system disorders | Eculizumab | 2 | 22 | |
| other | THROMBOCYTOPENIA | Blood and lymphatic system disorders | Eculizumab | 1 | 22 | |
| other | BUNDLE BRANCH BLOCK RIGHT | Cardiac disorders | Eculizumab | 1 | 22 | |
| other | PERICARDIAL EFFUSION | Cardiac disorders | Eculizumab | 1 | 22 | |
| other | EAR PAIN | Ear and labyrinth disorders | Eculizumab | 1 | 22 | |
| other | HYPOTHYROIDISM | Endocrine disorders | Eculizumab | 1 | 22 |
1 to 10 of 149Source ClinicalTrials.gov
See all 149 adverse eventsAdverse 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 | 20 | 22 |
| Total, serious adverse events | Eculizumab | 13 | 22 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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.
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/NCT01193348
GET https://conductscience.org/api/trials/v1/NCT01193348/results?page=2