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• Register: EUCTR
• Last refreshed on: 8 October 2021
• Main ID: EUCTR2010-020310-28-ES
• Date of registration: 08/09/2011
• Prospective Registration: Yes
• Primary sponsor: ALEXION PHARMACEUTICALS, INC.
• Public title: -
• Scientific title: AN OPEN-LABEL, MULTI-CENTER CLINICAL TRIAL OF ECULIZUMAB IN PEDIATRIC PATIENTS WITH ATYPICAL HEMOLYTIC-UREMIC SYNDROME (aHUS) - -
• Date of first enrolment: 07/11/2011
• Target sample size: 15
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-020310-28
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: ALEXION EUROPE SAS

• Name: European Clinical Trial Information
• Affiliation: ALEXION EUROPE SAS

Key inclusion & exclusion criteria

Inclusion criteria:
1. Patient?s parent/legal guardian must be willing and able to give written informed consent and the patient must be willing to give written informed assent [if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)].
2. Pediatric patients with aHUS. Patients may be newly diagnosed, or with previously diagnosed disease, or post-kidney transplant with the disease.
3. Patients from 1 month up to 18 years of age and body weight ? 5 kg.
4. Patient exhibit Thrombocytopenia, hemolysis and elevated Serum Creatinine
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Plasma Therapy for > 5 weeks prior to enrollment.
2. Chronic dialysis
3. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Pediatric patients with Atypical Hemolytic-Uremic Syndrome (aHUS)
MedDRA version: 14.0 Level: LLT Classification code 10019515 Term: Hemolytic uremic syndrome System Organ Class: 10005329 - Blood and lymphatic system disorders

Intervention(s)

Trade Name: SOLIRIS
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: eculizumab
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: -

Primary end point(s): Primary Efficacy Endpoint:
Proportion of patients with complete TMA response.

Primary Safety Endpoint:
Safety assessments.

Main Objective: Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.

Secondary Objective: Efficacy

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: -

Secondary end point(s): -

Secondary ID(s)

2010-020310-28-NL

C10-003

NCT01193348

Source(s) of Monetary Support

ALEXION PHARMACEUTICALS INC

Ethics review

Status: Approved
Approval date: 13/10/2011
Contact:

Results

• Results available: Yes
• Date Posted: 29/07/2016
• Date Completed: 08/01/2014
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-020310-28/results