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Clinical Trials/NCT01193348
NCT01193348CompletedPhase 2

An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome

Alexion Pharmaceuticals, Inc.0 sites22 target enrollmentStarted: September 2010Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 2
Status
Completed
Enrollment
22
Primary Endpoint
Proportion of Patients With Complete TMA Response
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

The primary purpose is to assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.

Study Design

Study Type
Interventional
Allocation
Non Randomized
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
1 Month to 18 Years (Child, Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Not provided

Exclusion Criteria

  • Not provided

Arms & Interventions

Eculizumab

Experimental

Intervention: Eculizumab (Drug)

Outcomes

Primary Outcomes

Proportion of Patients With Complete TMA Response

Time Frame: Through 26 weeks

Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline which was sustained for at least two consecutive measurements obtained at least four weeks apart).

Secondary Outcomes

  • Proportion of Patients With Complete Hematologic Response(Through End of Study, Median Exposure 55 Weeks)
  • Proportion of Patients With Platelet Count Normalization(Through End of Study, Median Exposure 55 Weeks)
  • Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement(Through End of Study, Median Exposure 55 Weeks)
  • Platelet Count Change From Baseline to 26 Weeks(Through 26 weeks)
  • Proportion of Patients With Complete TMA Response(Through End of Study, Median Exposure 55 Weeks)
  • Platelet Count Change From Baseline to 52 Weeks(Through 52 Weeks)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg)(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

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