This trial is active, not recruiting.

Condition atypical hemolytic uremic syndrome
Treatment eculizumab
Phase phase 2
Target C5
Sponsor Alexion Pharmaceuticals
Start date May 2009
End date December 2012
Trial size 15 participants
Trial identifier NCT00844428, BB-IND 11075, C08-003B, EudraCT Number 2008-006955-28


The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adolescent patients with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS).

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Intravenously administered 900mg once per week for 4 weeks, 1200 mg on week 5 and then 1200mg every 2 weeks thereafter.

Primary Outcomes

Assess the effect of eculizumab on thrombotic microangiopathy (TMA).
time frame: Through 26 weeks

Secondary Outcomes

Additional efficacy endpoints related to manifestations of TMA.
time frame: Through 26 weeks
Overall safety and tolerability of eculizumab
time frame: Through 26 weeks
Pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in patients with aHUS.
time frame: Through 26 weeks

Eligibility Criteria

Male or female participants from 12 years up to 18 years old.

Inclusion Criteria: 1. Male or female patients from 12 and up to 18 years of age who have been diagnosed with Atypical Hemolytic-Uremic Syndrome (aHUS). 2. Patients must be receiving PT for aHUS. 3. Platelet Count Pre-PT Baseline Set-Point (collected immediately prior to the Qualifying PT Episode) is within 75% of the average of the pre-PT platelet counts collected at Screening and during the Observation Period. 4. Diagnosis of aHUS 5. Lactate dehydrogenase (LDH) level ≥ ULN. 6. Creatinine level ≥ ULN for age. 7. Sexually active women of childbearing potential must be practicing an effective, reliable and medically acceptable contraceptive regimen during the entire duration of the study, including the follow-up period. 8. Patient's parents/legal guardian must be willing and able to give written informed consent and patient must be willing to give written informed assent. 9. Able and willing to comply with study procedures. Exclusion Criteria: 1. ADAMTS-13 inhibitor or deficiency (i.e., ADAMTS-13 activity <5%) as measured at the screening visit. 2. Malignancy. 3. Typical HUS (Shiga toxin +). 4. Known HIV infection. 5. Identified drug exposure-related HUS. 6. Infection-related HUS. 7. 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. 8. Pregnancy or lactation. 9. Unresolved meningococcal disease. 10. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome. 11. 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. 12. Patients receiving IVIg or Rituximab therapy. 13. Patients receiving other immunosuppressive therapies such as steroids, mTOR inhibitors or FK506 inhibitors are excluded unless: [1] part of a 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. 14. Patients receiving Erythrocyte Stimulating Agents (ESAs) unless already on a stable dose for at least 4 weeks prior to the screening period. 15. 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.

Additional Information

Official title An Open-Label, Multi-Center Controlled Clinical Trial Of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive Atypical Hemolytic Uremic Syndrome (AHUS)
Trial information was received from ClinicalTrials.gov and was last updated in November 2012.
Information provided to ClinicalTrials.gov by Alexion Pharmaceuticals.