Atypical Hemolytic-Uremic Syndrome
Conditions
Keywords
Atypical Hemolytic-Uremic Syndrome
Brief summary
The primary purpose is to assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
Interventions
DRUGEculizumab
Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
Inclusion: 1. Patient's parent/legal guardian must have been willing and able to give written informed consent and the patient must have been willing to give written informed assent (if applicable as determined by the central IRB/IEC). 2. Pediatric patients with aHUS: Patients could have been newly diagnosed, or with previously diagnosed disease, or post-kidney transplant with the disease. 3. Patients one month to 18 years and body weight ≥ 5kg. 4. Platelet count at screening and baseline visit must have been below lower limit of normal (\<LLN). If screening visit and baseline visit are combined into one day, an additional platelet count value obtained at least 24 hours before screening/baseline sample must also be \<LLN. 5. Exhibited signs or symptoms of hemolysis at start of current aHUS event (i.e., lactate dehydrogenase (LDH) ≥1.5 x Upper Limit of Normal \[ULN\] and hemoglobin ≤LLN), fragmented RBC with a negative Coombs test. 6. Serum Creatinine level ≥97 percentile for age at screening (patients requiring dialysis for acute renal failure are also eligible). 7. Patients with aHUS due to complement regulatory protein genetic abnormality or anti-complement factor antibody or those in whom known etiologies of hemolytic uremic syndrome (HUS) have been ruled out as confirmed in the
Exclusion criteria
. 8. Patients must have been vaccinated against N. meningitidis, pneumococcus and haemophilus (per the vaccine label) at least 14 days prior to study drug initiation or otherwise be protected by prophylactic antibiotics. Patients under age two years were to receive antibiotic prophylaxis throughout the treatment period. 9. Female patients of childbearing potential (female patients who have achieved menarche) must have been practicing an effective, reliable and medically approved contraceptive regimen during the entire duration of the study, including the follow-up period. At the time of the last follow-up visit, patients must have agreed to continue to use adequate contraception methods for up to five months following discontinuation of eculizumab treatment. 10. Able and willing to comply with study procedures Exclusion: Any of the following was regarded as a criterion for exclusion from the study: 1. Known familial ADAMTS-13 deficiency (ADAMTS-13 \<5%). 2. Shiga toxin E.coli-related hemolytic uremic syndrome (STEC-HUS \[known Shiga toxin + E.coli\]). 3. History of malignancy within five years of screening. 4. Known human immunodeficiency virus (HIV) infection. 5. Identified drug exposure-related HUS. 6. Infection-related HUS. 7. HUS related to bone marrow transplant (BMT). 8. HUS related to vitamin B12 deficiency. 9. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome. 10. Plasma Therapy for \>5 weeks prior to enrollment. 11. Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage renal disease \[ESRD\]). 12. Patients with a confirmed diagnosis of sepsis defined as positive blood cultures within seven days of the screening visit and not treated with antibiotics to which the organism is sensitive. 13. Presence or suspicion of active and untreated systemic bacterial infection that, in the opinion of the Investigator confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease. 14. Pregnancy or lactation. 15. History of meningococcal/pneumococcal/gonococcal disease. 16. Any medical or psychological condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study. 17. Patients receiving chronic intravenous immunoglobulin (IVIg) within eight weeks unless for unrelated medical condition (e.g., Hypogammaglobinemia), or chronic Rituximab therapy within 12 weeks of the screening visit. 18. Patients receiving other immunosuppressive therapies such as steroids, mTOR inhibitors, calcineurin inhibitors (e.g., cyclosporine or tacrolimus are excluded unless: \[1\] part of an established post-transplant anti-rejection regime, or \[2\] patient has confirmed anti-Complement Factor antibodies antibody requiring immunosuppressive therapy or \[3\] steroids are being used for a condition other than aHUS (example asthma). 19. Participation in any other investigational drug trial or device trial, or procedures beginning four weeks prior to screening and throughout the entire trial 20. Prior use of eculizumab, hypersensitivity to eculizumab, to murine proteins or to one of the excipients.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| Platelet Count Change From Baseline to 26 Weeks | Through 26 weeks | — |
| 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). |
| Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 | Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort | — |
| 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. |
| Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 | Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort | — |
| Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) | Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort | — |
| Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 | Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort | — |
| Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 | Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort | — |
| Platelet Count Change From Baseline to 52 Weeks | Through 52 Weeks | — |
Countries
Australia, Belgium, Canada, France, Germany, Italy, Netherlands, United Kingdom, United States
Participant flow
Recruitment details
A total of 27 patients diagnosed with aHUS signed the informed consent and of these, 22 patients were treated. Five patients were excluded from the study due to failed screening procedure and did not receive eculizumab.
Pre-assignment details
At screening, patients had to have a platelet count \< lower limit of normal range (\<LLN) and serum creatinine level \> 97 percentile for age; and had to exhibit signs or symptoms of hemolysis at the start of the current aHUS event with fragmented RBC and a negative Coombs test.
Participants by arm
| Arm | Count |
|---|---|
| Eculizumab Eculizumab: Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible. | 22 |
| Total | 22 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Extension Treatment Period | Physician Decision | 1 |
| Post Treatment Period (Patients Who Disc | Patient did not return for visit | 2 |
| Screening Period | Screen Failure | 5 |
| Treatment Period (26 Weeks) | Patient Requested to Withdraw | 1 |
| Treatment Period (26 Weeks) | Positive Shiga-Toxin Result vial Local | 1 |
| Treatment Period (26 Weeks) | Serious Adverse Event | 1 |
Baseline characteristics
| Characteristic | Eculizumab |
|---|---|
| Age, Continuous | 6.6 Years STANDARD_DEVIATION 6.06 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants |
| Race (NIH/OMB) White | 18 Participants |
| Sex: Female, Male Female | 10 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 20 / 22 |
| serious Total, serious adverse events | 13 / 22 |
Outcome results
Primary
Proportion of Patients With Complete TMA Response
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).
Time frame: Through 26 weeks
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eculizumab | Proportion of Patients With Complete TMA Response | 63.6 Percentage of Participants |
95% CI: [40.7, 82.8]
Secondary
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7
Time frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort
Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 | Min concentration during maintenance | 241.8 micrograms/mL | Standard Deviation 106 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 | Max concentration during maintenance | 459.5 micrograms/mL | Standard Deviation 105.5 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 | Min Concentration during induction period | 297.2 micrograms/mL | Standard Deviation 74.7 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7 | Max concentration during induction period | 436.3 micrograms/mL | Standard Deviation 128.3 |
Secondary
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg)
Time frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort
Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) | Min Concentration during induction period | 185.1 micrograms/mL | Standard Deviation 19.5 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) | Max concentration during induction period | 242.5 micrograms/mL | Standard Deviation 24.5 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) | Min concentration during maintenance | 337.6 micrograms/mL | Standard Deviation 43.4 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg) | Max concentration during maintenance | 579.5 micrograms/mL | Standard Deviation 66.5 |
Secondary
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1
Time frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort
Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data. N=0 in the maintenance phase because the patient changed to ≥40kg body weight category
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 | Min Concentration during induction period | 196.8 micrograms/mL |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 | Max concentration during induction period | 282.2 micrograms/mL |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 | Min concentration during Maintenance | NA micrograms/mL |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1 | Max concentration during Maintenance | NA micrograms/mL |
Secondary
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5
Time frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort
Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 | Min Concentration during induction period | 125.5 micrograms/mL | Standard Deviation 26.3 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 | Max concentration during induction period | 180.9 micrograms/mL | Standard Deviation 42.6 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 | Min concentration during maintenance | 278.4 micrograms/mL | Standard Deviation 130.3 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5 | Max concentration during maintenance | 572.8 micrograms/mL | Standard Deviation 267.7 |
Secondary
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3
Time frame: Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort
Population: PK parameters Cmin and Cmax were estimated using a population PK model developed from the observed PK concentration data
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 | Min Concentration during induction period | 223.2 micrograms/mL | Standard Deviation 19 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 | Max concentration during induction period | 312.8 micrograms/mL | Standard Deviation 51.2 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 | Min concentration during maintenance | 187.2 micrograms/mL | Standard Deviation 42 |
| Eculizumab | Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3 | Max concentration during maintenance | 499.7 micrograms/mL | Standard Deviation 27.8 |
Secondary
Platelet Count Change From Baseline to 26 Weeks
Time frame: Through 26 weeks
Population: 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.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Eculizumab | Platelet Count Change From Baseline to 26 Weeks | 204.96 10^9 cells/L |
p-value: <0.000195% CI: [164.44, 245.49]ANOVA
Secondary
Platelet Count Change From Baseline to 52 Weeks
Time frame: Through 52 Weeks
Population: 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.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Eculizumab | Platelet Count Change From Baseline to 52 Weeks | 165.43 10^9 cells/L |
p-value: <0.000195% CI: [98.43, 232.43]ANOVA
Secondary
Proportion of Patients With Complete Hematologic Response
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.
Time frame: Through 26 weeks
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eculizumab | Proportion of Patients With Complete Hematologic Response | 81.8 Percentage of Participants |
95% CI: [59.7, 94.8]
Secondary
Proportion of Patients With Complete Hematologic Response
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.
Time frame: Through End of Study, Median Exposure 55 Weeks
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eculizumab | Proportion of Patients With Complete Hematologic Response | 90.9 Percentage of Participants |
95% CI: [70.8, 98.9]
Secondary
Proportion of Patients With Complete TMA Response
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).
Time frame: Through End of Study, Median Exposure 55 Weeks
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eculizumab | Proportion of Patients With Complete TMA Response | 68.2 Percentage of Participants |
95% CI: [45.1, 86.1]
Secondary
Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement
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.
Time frame: Through End of Study, Median Exposure 55 Weeks
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eculizumab | Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement | 86.4 Percentage of Participants |
95% CI: [65.1, 97.1]
Secondary
Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement
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.
Time frame: Through 26 weeks
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eculizumab | Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement | 86.4 Percentage of Participants |
95% CI: [65.1, 97.1]
Secondary
Proportion of Patients With Platelet Count Normalization
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
Time frame: Through End of Study, Median Exposure 55 Weeks
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eculizumab | Proportion of Patients With Platelet Count Normalization | 95.5 Percentage of Participants |
95% CI: [77.2, 99.9]
Secondary
Proportion of Patients With Platelet Count Normalization
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.
Time frame: Through 26 weeks
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eculizumab | Proportion of Patients With Platelet Count Normalization | 95.5 Percentage of Participants |
95% CI: [77.2, 99.9]