World Health Organization site
Skip Navigation Links

Please fill this short user satisfaction survey


Main
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 18 March 2020
Main ID:  EUCTR2016-002499-29-AT
Date of registration: 08/03/2017
Prospective Registration: Yes
Primary sponsor: Alexion Pharmaceuticals Incorporated
Public title: Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)
Scientific title: A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)
Date of first enrolment: 28/04/2017
Target sample size: 23
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-002499-29
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other: Number of treatment arms in the trial: 1  
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Australia Austria Belgium Canada France Germany Italy Japan
Korea, Republic of Russian Federation Spain Sweden Taiwan United Kingdom United States
Contacts
Name: European Clinical Trial Information   
Address:  103-105 rue Anatole France 92300 Levallois-Perret France
Telephone: +33147100615
Email: clinicaltrials.eu@alexion.com
Affiliation:  Alexion Europe SAS
Name: European Clinical Trial Information   
Address:  103-105 rue Anatole France 92300 Levallois-Perret France
Telephone: +33147100615
Email: clinicaltrials.eu@alexion.com
Affiliation:  Alexion Europe SAS
Key inclusion & exclusion criteria
Inclusion criteria:
Cohort 1 inclusion criteria:
1. Patients from birth up to < 18 years of age and weighing = 5 kg at the time of consent.
2. Evidence of thrombotic microangiopathy (TMA), including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
3. Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae
4. Female patients of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ALXN1210

Cohort 2 inclusion criteria:
1. Patients between 12 and <18 years of age who have been treated with eculizumab for aHUS for at least 90 days prior to Screening
2. Patients with a documented diagnosis of aHUS
3. Patients with clinical evidence of response to eculizumab indicated by stable TMA parameters at Screening
4. Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and
Haemophilus influenzae
5. Female patients of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8
months after the last dose of ALXN1210
Are the trial subjects under 18? yes
Number of subjects for this age range: 23
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. ADAMTS13 deficiency (Activity < 5%)
2. Shiga toxin-related hemolytic uremic syndrome (STEC-HUS)
3. Positive direct Coombs test
4. Females who plan to become pregnant during the study or are currently pregnant or breastfeeding
5. Identified drug exposure-related hemolytic uremic syndrome (HUS)
6. Bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT) within last 6 months prior to start of Screening
7. HUS related known genetic defects of cobalamin C metabolism
8. Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
9. Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for ESKD)
10. For Cohort 2 patients, prior use of complement inhibitors other than eculizumab
11. For Cohort 2 patients, any known abnormal TMA parameters within 90 days prior to Screening


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Atypical Hemolytic Uremic Syndrome (aHUS)
MedDRA version: 20.0 Level: LLT Classification code 10019515 Term: Hemolytic uremic syndrome System Organ Class: 100000004851
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Intervention(s)

Product Name: Ravulizumab
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Ravulizumab
Current Sponsor code: ALXN1210
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)
Secondary Objective: To assess in both complement inhibitor naïve pediatric patients (ie, Cohort 1) and complement inhibitor experienced adolescent (i.e. Cohort 2):
- The safety and tolerability of ALXN1210
- Additional efficacy measures
Timepoint(s) of evaluation of this end point: Week 26
Primary end point(s): Complete TMA Response (only for Cohort 1)
Main Objective: Efficacy of ALXN1210 in complement inhibitor treatment naïve pediatric patients (ie, Cohort 1).
Secondary Outcome(s)
Timepoint(s) of evaluation of this end point: Week 26
Secondary end point(s): - Dialysis requirement status
- Time to Complete TMA Response (only for Cohort 1)
- Complete TMA Response status over time (only for Cohort 1)
- Observed value and change from baseline in estimated glomerular filtration rate (eGFR)
- Change from baseline in chronic kidney disease (CKD) stage
- Change from baseline in hematologic parameters (platelets, LDH, hemoglobin)
- Increase in hemoglobin of = 20 g/L from baseline
- Change from baseline in quality of life as measured by Pediatric FACIT Fatigue questionnaire (patients = 5 years of age)
-TMA parameters in patients who discontinue treatment in the Extension Period, but remain in the study
Secondary ID(s)
ALXN1210-aHUS-312
2016-002499-29-GB
Source(s) of Monetary Support
Alexion Pharmaceuticals Inc.
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 28/04/2017
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history Please fill this short user satisfaction survey