Trial · NCT00844428
Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS
NCT00844428, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2015-01-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS. Trials, record NCT00844428. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00844428
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS
- NCT number
- NCT00844428
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 5 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:2008-006955-28
- Results first posted
- 2015-01-26
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Patients With TMA Event-free Status | Through 26 weeks | TMA Event-free status is defined as the absence for at least 12 weeks of \[1\] decrease in platelet count of \> 25% from the Platelet Count Pre-PT Baseline Set Point; \[2\] PT while the patient is receiving eculizumab, and \[3\] new dialysis. |
| 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. |
| Primary | 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 (≥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 26 Weeks | From Baseline to 26 Weeks | |
| Secondary | Percentage of Patients With Platelet Count Normalization | 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 |
| Secondary | Percentage of Patients With TMA Event-free Status | Through End of Study, Median Exposure 156 Weeks | TMA Event-free status is defined as the absence for at least 12 weeks of \[1\] decrease in platelet count of \> 25% from the Platelet Count Pre-PT Baseline Set Point; \[2\] PT while the patient is receiving eculizumab, and \[3\] new dialysis. |
| Secondary | Percentage of Patients With Hematologic Normalization | Through End of Study, Median Exposure 156 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 156 Weeks | The proportion of patients who achieved a Complete TMA Response from baseline through end of study with eculizumab 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 156 Weeks | TMA Intervention Rate (# PE/PI and # Dialysis Events/Patient/Day) in the eculizumab treatment period (from baseline through end of study) for PE/PI and (from the fifteenth day following the first eculizumab dose through end of study) for new dialysis events was compared with the TMA Intervention Rate during the pre-eculizumab treatment period. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Patients With TMA Event-free Status | Through 26 weeks | The tabulations of the proportions of patients who achieved a TMA Event-Free status through 26 weeks were performed for the ITT population. Exact binomial 95% 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 95% confidence intervals were produced for the analysis. | Percentage of Participants | NUMBER |
| PRIMARY | 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 95% 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 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 |
| SECONDARY | 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 95% confidence intervals were produced for the analysis. | Percentage of Participants | NUMBER |
| SECONDARY | Percentage of Patients With TMA Event-free Status | Through End of Study, Median Exposure 156 Weeks | The tabulations of the proportions of patients who achieved a TMA Event-Free status through 26 weeks were performed for the ITT population. Exact binomial 95% confidence intervals were produced for the analysis. | Percentage of Participants | NUMBER |
| SECONDARY | Percentage of Patients With Hematologic Normalization | Through End of Study, Median Exposure 156 Weeks | Tabulations of the proportion of patients who achieved a hematologic normalization through end of study were performed for the ITT population. Exact 95% 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 156 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 95% confidence intervals were produced. | Percentage of Participants | NUMBER |
| SECONDARY | TMA Intervention Rate | Through End of Study, Median Exposure 156 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 13Source ClinicalTrials.gov
See all 13 outcomesResults by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Patients With TMA Event-free Status | Eculizumab | 80 | 95% Confidence Interval 56 to 94 | Percentage of Participants | |
| Percentage of Patients With Hematologic Normalization | Eculizumab | 90 | 95% Confidence Interval 68 to 99 | Percentage of Participants | |
| Percentage of Patients With Complete TMA Response | Eculizumab | 25 | 95% Confidence Interval 9 to 49 | Percentage of Participants | |
| TMA Intervention Rate | Eculizumab | 0 | Standard Deviation 0 | #events/patient/day | |
| Platelet Count Change From Baseline to 26 Weeks | Eculizumab | 6.75 | 95% Confidence Interval -15.73 to 29.23 | 10^9 cells/L | |
| Percentage of Patients With Platelet Count Normalization | Eculizumab | 90 | 95% Confidence Interval 68 to 99 | Percentage of Participants | |
| Percentage of Patients With TMA Event-free Status | Eculizumab | 95 | 95% Confidence Interval 75 to 100 | Percentage of Participants | |
| Percentage of Patients With Hematologic Normalization | Eculizumab | 90 | 95% Confidence Interval 68 to 99 | Percentage of Participants | |
| Percentage of Patients With Complete TMA Response | Eculizumab | 55 | 95% Confidence Interval 32 to 77 | Percentage of Participants | |
| TMA Intervention Rate | Eculizumab | 0.00 | Standard Deviation 0.02 | #events/patient/day |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow15
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Screening Period | STARTED | Eculizumab | 23 | |
| Screening Period | COMPLETED | Eculizumab | 20 | |
| Screening Period | NOT COMPLETED | Eculizumab | 3 | |
| Observation Period (8 Weeks) | STARTED | Eculizumab | 20 | |
| Observation Period (8 Weeks) | COMPLETED | Eculizumab | 20 | |
| Observation Period (8 Weeks) | NOT COMPLETED | Eculizumab | 0 | |
| Treatment Period (26 Weeks) | STARTED | Eculizumab | 20 | |
| Treatment Period (26 Weeks) | COMPLETED | Eculizumab | 20 | |
| Treatment Period (26 Weeks) | NOT COMPLETED | Eculizumab | 0 | |
| Extension Treatment Period | STARTED | Eculizumab | 19 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 participant flowAdverse events255
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 | Abnormal clotting factor | Blood and lymphatic system disorders | Eculizumab | 1 | 20 | |
| other | Anaemia | Blood and lymphatic system disorders | Eculizumab | 7 | 20 | |
| other | Anaemia folate deficiency | Blood and lymphatic system disorders | Eculizumab | 1 | 20 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Eculizumab | 1 | 20 | |
| other | Leukopenia | Blood and lymphatic system disorders | Eculizumab | 3 | 20 | |
| other | Lymph node pain | Blood and lymphatic system disorders | Eculizumab | 1 | 20 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Eculizumab | 1 | 20 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Eculizumab | 2 | 20 | |
| other | Microcytosis | Blood and lymphatic system disorders | Eculizumab | 1 | 20 | |
| other | Neutropenia | Blood and lymphatic system disorders | Eculizumab | 1 | 20 |
1 to 10 of 255Source ClinicalTrials.gov
See all 255 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 | 20 |
| Total, serious adverse events | Eculizumab | 13 | 20 |
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/NCT00844428
GET https://conductscience.org/api/trials/v1/NCT00844428/events?page=2