Status:
COMPLETED
Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Atypical Hemolytic Uremic Syndrome
Eligibility:
All Genders
12-17 years
Phase:
PHASE2
Brief Summary
The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adolescent patients with plasma therapy-resistant Atypical Hemolytic-Uremic Syndrome (aHUS).
Eligibility Criteria
Inclusion
- Male or female patients from 12 and up to 18 years weighing ≥ 40 kg who have been diagnosed with Atypical Hemolytic-Uremic Syndrome (aHUS).
- Decrease in platelet count despite at least 4 plasma therapy (PT) treatments in the 1 week immediately prior to screening.
- Screening platelet count , \< 150 x10\^9/L and at least 25% lower than the average remission platelet count or
- If remission counts not available, platelet count at onset of the current aHUS episode must be ≤75x10\^9/L and platelet count at screening must be ≤ 100 x 10\^9/L despite PT treatment administration of at least 4 PT treatments in the 1 week immediately prior to screening
- Known complement regulatory protein genetic abnormality
- Lactate dehydrogenase (LDH) level ≥ ULN unless the patient has been receiving plasma exchange and LDH at the onset of the current aHUS episode was at least the ULN. If LDH is normal at screening, other markers indicative of ongoing hemolysis such as haptoglobin, schistocytes should be evaluated and discussed with Sponsor
- Creatinine level ≥ ULN for age
- Female patients of childbearing potential must be practicing an effective, reliable and medically approved contraceptive regimen during the entire duration of the study, including the follow-up period and for up to 5 months following of eculizumab treatment discontinuation.
- 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.
- Able and willing to comply with study procedures.
Exclusion
- 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
- Patients between ages from 12 and up to 18 years weighing \< 40 kg
Key Trial Info
Start Date :
May 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2013
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT00844844
Start Date
May 1 2009
End Date
July 1 2013
Last Update
July 23 2015
Active Locations (17)
Enter a location and click search to find clinical trials sorted by distance.
1
Atlanta, Georgia, United States, 30322
2
Fort Wayne, Indiana, United States, 46804
3
New York, New York, United States, 10032
4
New York, New York, United States, 10065