Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00844844
First received: February 13, 2009
Last updated: June 11, 2014
Last verified: June 2014
  Purpose

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).


Condition Intervention Phase
Atypical Hemolytic Uremic Syndrome
Drug: Eculizumab
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Multi-Center Controlled Clinical Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant Atypical Hemolytic Uremic Syndrome (AHUS)

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Assess the effect of eculizumab to reduce thrombotic microangiopathy (TMA). [ Time Frame: Through 26 Weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Additional efficacy endpoints related to manifestations of TMA. [ Time Frame: Through 26 Weeks ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in patients with aHUS. [ Time Frame: Through 26 Weeks ] [ Designated as safety issue: Yes ]
  • Overall safety and tolerability of eculizumab. [ Time Frame: Through 26 Weeks ] [ Designated as safety issue: Yes ]

Enrollment: 1
Study Start Date: May 2009
Study Completion Date: July 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eculizumab Drug: Eculizumab
Drug: Eculizumab Intravenously administered 900mg once per week for 4 weeks, 1200 mg on week 5 then 1200mg every 2 weeks thereafter.

  Eligibility

Ages Eligible for Study:   12 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female patients up to 18 years of age who have been diagnosed with Atypical Hemolytic-Uremic Syndrome (aHUS).
  2. Decrease in platelet count despite at least 4 plasma therapy (PT) treatments in the 1 week immediately prior to screening.

    1. Screening platelet count , 150 x109/L and at least 25% lower than remission platelet count or
    2. If remission counts not available, screening platelet count <75x109/L
  3. Diagnosis of aHUS
  4. Lactate dehydrogenase (LDH) level ≥ ULN.
  5. Creatinine level ≥ ULN for age.
  6. Female patients 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.
  7. 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.
  8. 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. Renal function status requiring chronic dialysis
  8. 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.
  9. Pregnancy or lactation.
  10. Unresolved meningococcal disease.
  11. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome.
  12. 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.
  13. Patients receiving IVIg or Rituximab therapy.
  14. 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.
  15. Patients receiving Erythrocyte Stimulating Agents (ESAs) unless already on a stable dose for at least 4 weeks prior to the screening period.
  16. 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00844844

Locations
United States, Georgia
Emory University, Children's Center
Atlanta, Georgia, United States
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

No publications provided by Alexion Pharmaceuticals

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00844844     History of Changes
Other Study ID Numbers: C08-002B, BB-IND-11075, EudraCT Number 2008-006953-41
Study First Received: February 13, 2009
Last Updated: June 11, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Alexion Pharmaceuticals:
aHUS

Additional relevant MeSH terms:
Hemolysis
Syndrome
Azotemia
Hemolytic-Uremic Syndrome
Pathologic Processes
Disease
Uremia
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders

ClinicalTrials.gov processed this record on September 16, 2014