Phase 2
N=17
Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS
Atypical Hemolytic Uremic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00844545 ↗Enrolled (actual)
17
Serious AEs
100.0%
Results posted
Dec 2014
Primary outcome: Primary: Platelet Count Change From Baseline to 26 Weeks — 65.18 10^9 cells/L — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Eculizumab (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Sep 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Platelet Count Change From Baseline to 26 Weeks |
65.18 | <0.0001 sig |
| PRIMARY Percentage of Patients With Platelet Count Normalization |
88 | — |
| PRIMARY Percentage of Patients With Hematologic Normalization |
88 | — |
| SECONDARY Percentage of Patients With Complete TMA Response |
76 | — |
| SECONDARY TMA Intervention Rate |
0.04 | <0.0001 sig |
| SECONDARY Platelet Count Change From Baseline to 156 Weeks |
111.62 | <0.0001 sig |
| SECONDARY Percentage of Patients With Platelet Count Normalization |
88 | — |
| SECONDARY Percentage of Patients With Hematologic Normalization |
88 | — |
| SECONDARY Percentage of Patients With Complete TMA Response |
76 | — |
| SECONDARY TMA Intervention Rate |
0.04 | <0.0001 sig |
| SECONDARY Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration |
145.16; 93.66; 345.14; 151.80 | — |
Summary
The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adult patients with plasma therapy-resistant Atypical Hemolytic-Uremic Syndrome (aHUS).
Eligibility Criteria
Exclusion Criteria
- TTP, (defined as ADAMTS-13 activity <5%) from an historical observation (prior to initiation of plasma therapy) or as tested at the screening visit by the central laboratory
- Malignancy within 5 years of screening
- Typical HUS (Shiga toxin +)
- Known HIV infection
- Identified drug exposure-related HUS.
- Infection-related HUS
- HUS related to bone marrow transplant
- HUS related to vitamin B12 deficiency
- Renal function status requiring chronic dialysis
- Patients with a confirmed diagnosis of sepsis
- 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
- Pregnancy or lactation
- Unresolved meningococcal disease
- Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome
- 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
- Patients who have received previous treatment with eculizumab
- Patients receiving IVIG within 8 weeks or Rituximab therapy within 12 weeks of screening.
- Patients receiving other immunosuppressive therapies such as steroids, mTOR inhibitors or tacrolimus are excluded unless: [1] part of an established post-transplant anti-rejection regime, [2] patient has confirmed anti-CFH antibody requiring immunosuppressive therapy, and [3] dose of such medications have been unchanged for at least 4 weeks prior to the screening period or [4] patient is experiencing an acute aHUS relapse immediately after transplant
- Patients receiving Erythrocyte Stimulating Agents (ESAs) unless already on a stable dose for at least 4 weeks prior to the screening period, or a washout period of at least 2 weeks from the last dose of ESA therapy.
- Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures beginning 4 weeks prior to screening and throughout the entire trial.
- Hypersensitivity to eculizumab, to murine proteins or to one of the excipients
Data sourced from ClinicalTrials.gov (NCT00844545). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.