A Phase 2 interventional study of eculizumab in Atypical Hemolytic Uremic Syndrome, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 13 sites in 7 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2015-07-23.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 2, Interventional, and Treatment
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Among 10 studies testing eculizumab in Atypical Hemolytic Uremic Syndrome — 1 open to participants now, 1 under way, 1 of them phase 3. eculizumab · Atypical Hemolytic Uremic Syndrome.
Among the 10 studies testing eculizumab in Atypical Hemolytic Uremic Syndrome, this one is the 7th largest by enrollment, is one of 4 that started in 2009, is one of 8 in phase 2, and was the 1st registered.
45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.
Browse Atypical Hemolytic Uremic Syndrome studies →1 of the 10 studies testing eculizumab in Atypical Hemolytic Uremic Syndrome is open now, in 1 country. Recruiting first, the newest first:
Assistance Publique - Hôpitaux de Paris · Sites in France
Ages 18+
By country, each count listing its studies:
eculizumab: first FDA approval 2007 (SOLIRIS), first EMA approval 2007. What it is approved for, and how studies give it, is on its page →
Of the 12 Atypical Hemolytic Uremic Syndrome studies recruiting now, 10 (83%) set no upper age limit and none (0%) stop at 75 or younger; 6 take children under 18; 2 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
10 studies; the count open now beside each. This study's row is marked.
First in 2009; this study’s year is marked. 4 started in 2009.
This study's enrollment of 15 is below the median of 34 across 26 interventional studies indexed under Atypical Hemolytic Uremic Syndrome.
Median enrollment 19 across the 10 interventional studies. Of the 9 that completed or were terminated, 7 (78%) have results posted.
This tab as a report to print or save as PDF → With an account.
7 of 10 have an industry lead sponsor. The 4 most frequent:
This study is registered under Alexion Pharmaceuticals, Inc., one of the names counted as AstraZeneca. Under that name alone:
Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca: 4 open now, 5 under way, none of them phase 3. Of 14 finished, 9 (64%) have results posted.
Among the 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca, this one is the 7th largest by enrollment of the 10 interventional ones that give a count, is one of 5 that started in 2009, is one of 7 in phase 2, and was the 1st registered.
AstraZeneca’s Atypical Hemolytic Uremic Syndrome studies by the year they started, since 2009.
None of the studies under way reaches primary completion within a year.
Among the 10 studies testing eculizumab in Atypical Hemolytic Uremic Syndrome, from their registry updates:
Nothing recorded among them since 2026-09-25.
Among AstraZeneca’s 19 in Atypical Hemolytic Uremic Syndrome:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adult patients with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: eculizumab
All patients received open-label eculizumab administered intravenously on the following dose schedule: Induction dose - 900 mg per week for four weeks and a dose of 1200 mg one week later; Maintenance dose - 1200 mg every two weeks. Patients who received plasma exchange or infusion during the eculizumab treatment period received a supplemental dose of 600 mg within one hour before plasma infusion or within one hour after the completion of each plasma exchange.
Percentage of Patients With TMA Event-free Status
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.
Time frame: Through 26 weeks
Percentage of Patients With Hematologic Normalization
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.
Time frame: Through 26 weeks
Percentage of Patients With Complete TMA Response
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.
Time frame: Through 26 weeks
TMA Intervention Rate
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.
Time frame: Through 26 weeks
Platelet Count Change From Baseline to 26 Weeks
Time frame: From Baseline to 26 Weeks
Percentage of Patients With Platelet Count Normalization
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
Time frame: Through 26 Weeks
Percentage of Patients With TMA Event-free Status
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.
Time frame: Through End of Study, Median Exposure 156 Weeks
Percentage of Patients With Hematologic Normalization
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.
Time frame: Through End of Study, Median Exposure 156 Weeks
Percentage of Patients With Complete TMA Response
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.
Time frame: Through End of Study, Median Exposure 156 Weeks
TMA Intervention Rate
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.
Time frame: Through End of Study, Median Exposure 156 Weeks
Platelet Count Change From Baseline to 156 Weeks
Time frame: From Baseline to 156 Weeks
Percentage of Patients With Platelet Count Normalization
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 Not specified.
Time frame: Through End of Study, Median Exposure 156 Weeks
Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration
Time frame: Induction Phase for 4 weeks followed by Maintenance Phase starting on Week 5 through 26 weeks or longer.
C08-003A/B combined 2 studies: one for adults (C08-003A, N=15) and one for adolescents (C08-003B, N=5) Please refer to NCT00844428 for combined studies with enrollment number corresponding to each individual study
| Milestone | Eculizumab |
|---|---|
| Started | 23 |
| Completed | 20 |
| Not completed | 3 |
| Withdrew: Screen failure | 3 |
| Milestone | Eculizumab |
|---|---|
| Started | 20 |
| Completed | 20 |
| Not completed | 0 |
| Milestone | Eculizumab |
|---|---|
| Started | 20 |
| Completed | 20 |
| Not completed | 0 |
| Milestone | Eculizumab |
|---|---|
| Started | 19 |
| Completed | 18 |
| Not completed | 1 |
| Withdrew: Death | 1 |
| Milestone | Eculizumab |
|---|---|
| Started | 5 |
| Completed | 4 |
| Not completed | 1 |
| Withdrew: Death | 1 |
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.
| Percentage of Participants | Eculizumab |
|---|---|
| Percentage of Patients With TMA Event-free Status | 80 (56 to 94) |
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.
| Percentage of Participants | Eculizumab |
|---|---|
| Percentage of Patients With Hematologic Normalization | 90 (68 to 99) |
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.
| Percentage of Participants | Eculizumab |
|---|---|
| Percentage of Patients With Complete TMA Response | 25 (9 to 49) |
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.
| #events/patient/day | Eculizumab |
|---|---|
| TMA Intervention Rate | 0 ± 0 |
| 10^9 cells/L | Eculizumab |
|---|---|
| Platelet Count Change From Baseline to 26 Weeks | 6.75 (-15.73 to 29.23) |
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
| Percentage of Participants | Eculizumab |
|---|---|
| Percentage of Patients With Platelet Count Normalization | 90 (68 to 99) |
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.
| Percentage of Participants | Eculizumab |
|---|---|
| Percentage of Patients With TMA Event-free Status | 95 (75 to 100) |
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.
| Percentage of Participants | Eculizumab |
|---|---|
| Percentage of Patients With Hematologic Normalization | 90 (68 to 99) |
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.
| Percentage of Participants | Eculizumab |
|---|---|
| Percentage of Patients With Complete TMA Response | 55 (32 to 77) |
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.
| #events/patient/day | Eculizumab |
|---|---|
| TMA Intervention Rate | 0.00 ± 0.02 |
| 10^9 cells/L | Eculizumab |
|---|---|
| Platelet Count Change From Baseline to 156 Weeks | -3.68 (-25.15 to 17.79) |
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 Not specified.
| Percentage of Participants | Eculizumab |
|---|---|
| Percentage of Patients With Platelet Count Normalization | 90 (68 to 99) |
| micrograms/mil | Eculizumab |
|---|---|
| max concentration during induction period | 161.47 ± 27.29 |
| min concentration during induction period | 112.43 ± 16.98 |
| max concentration during maintenance | 427.48 ± 67.54 |
| min concentration during maintenance | 212.45 ± 53.75 |
Collected over Through end of study; Exposure to eculizumab in this study extended for a median of 156 weeks and ranged from 26 weeks to 182 weeks.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Eculizumab | — | 13/20 (65%) | 20/20 (100%) |
| Event | Eculizumab |
|---|---|
| PneumoniaInfections and infestations | 2/20 |
| Renal failure chronicRenal and urinary disorders | 2/20 |
| Pericardial effusionCardiac disorders | 1/20 |
| Gastrointestinal haemorrhageGastrointestinal disorders | 1/20 |
| Impaired gastric emptyingGastrointestinal disorders | 1/20 |
| Medical device complicationGeneral disorders | 1/20 |
| Transplant rejectionImmune system disorders | 1/20 |
| AmyloidosisImmune system disorders | 1/20 |
| BacteraemiaInfections and infestations | 1/20 |
| Clostridium difficile colitisInfections and infestations | 1/20 |
| Event | Eculizumab |
|---|---|
| NasopharyngitisInfections and infestations | 12/20 |
| Bone painMusculoskeletal and connective tissue disorders | 11/20 |
| HeadacheNervous system disorders | 11/20 |
| DiarrhoeaGastrointestinal disorders | 10/20 |
| NauseaGastrointestinal disorders | 10/20 |
| VomitingGastrointestinal disorders | 10/20 |
| HypertensionVascular disorders | 9/20 |
| Upper respiratory tract infectionInfections and infestations | 8/20 |
| AnaemiaBlood and lymphatic system disorders | 7/20 |
| PyrexiaGeneral disorders | 7/20 |
The intent-to-treat (ITT) population was defined as all patients who received any amount of eculizumab, and were considered evaluable for safety and efficacy analyses. For both the efficacy and safety analyses, all 20 patients who were treated with study drug were included in the ITT population.
| Age, Continuous(Years) | Eculizumab |
|---|---|
| Mean | 32.3 ± 14.92 |
| Sex: Female, Male(Participants) | Eculizumab |
|---|---|
| Female | 12 |
| Male | 8 |
| Race (NIH/OMB)(Participants) | Eculizumab |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 18 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
The record gives a start of Jul 2009, a primary completion of Oct 2010 and a completion of Dec 2013. It does not say whether these dates are actual or estimated.
Results were first posted in Jan 2015, 4 years 3 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jun 2015. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Eculizumab
Atypical Hemolytic Uremic Syndrome →
Alexion Pharmaceuticals, Inc. →