Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00838513
First received: February 3, 2009
Last updated: November 5, 2012
Last verified: November 2012
  Purpose

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).


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 Adult Patients With Plasma Therapy-sensitive 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 on 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 ]
  • Overall safety and tolerability of eculizumab [ Time Frame: Through 26 weeks ] [ Designated as safety issue: Yes ]
  • Pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in patients with aHUS. [ Time Frame: Through 26 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 15
Study Start Date: May 2009
Estimated Study Completion Date: December 2012
Estimated Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female patients ≥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. Able to give written informed consent.
  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.
  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: NCT00838513

Locations
France
CHU Pellegrin
Bordeaux, France
Hôpital Edouard Herriot
Lyon, France
CHU de Nantez-Hotel Dieu
Nantes, France
Hopital Necker
Paris, France
Hopital Bretonneau
Tours, France
Germany
Universitats Acchen
Aachen, Germany
Italy
Istituto di Ricerche Farmacologiche Mario Negri
Ranica (BG) Villa Camozzi, Italy
Netherlands
Radboud University Medical Center
Nijmegen, Netherlands
Sweden
Karolinska Universitetssjukhuset Huddinge
Stockholm, Sweden
United Kingdom
Office of Kenneth Douglas
Glasgow, United Kingdom
Freeman Hospital
Newcastle, United Kingdom
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: NCT00838513     History of Changes
Other Study ID Numbers: C08-003A, BB-IND 11075, EudraCT Number 2008-006954-17
Study First Received: February 3, 2009
Last Updated: November 5, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Alexion Pharmaceuticals:
aHUS

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

ClinicalTrials.gov processed this record on August 21, 2014