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: |
|
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.
|
Ethics review
|
Status: Approved
Approval date: 28/04/2017
Contact:
|
Results
|
Results available:
|
|
Date Posted:
|
|
Date Completed:
|
|
URL:
|
|
|
|