Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)
- Conditions
- Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]Atypical Hemolytic Uremic Syndrome (aHUS)MedDRA version: 20.0 Level: LLT Classification code 10019515 Term: Hemolytic uremic syndrome System Organ Class: 100000004851
- Registration Number
- EUCTR2016-002499-29-FR
- Lead Sponsor
- Alexion Pharmaceuticals Incorporated
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 16
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
Are the trial subjects under 18? yes
Number of subjects for this age range: 16
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
1. ADAMTS13 deficiency (Activity < 5%)
2. Shiga toxin-related hemolytic uremic syndrome (STEC-HUS)
3. Positive direct Coombs test
4. Pregnancy 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 to vitamin B12 deficiency
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)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Efficacy of ALXN1210;<br> Secondary Objective: Safety and tolerability of ALXN1210<br> Additional efficacy measures<br> ;Primary end point(s): Complete TMA Response;Timepoint(s) of evaluation of this end point: Week 26
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): - Dialysis requirement status<br> - Time to Complete TMA Response<br> - Complete TMA Response status over time<br> - Observed value and change from baseline in estimated glomerular filtration rate (eGFR) <br> - Change from baseline in chronic kidney disease (CKD) stage<br> - Change from baseline in hematologic parameters (platelets, LDH, hemoglobin)<br> - Increase in hemoglobin of = 20 g/L from baseline<br> - Change from baseline in quality of life as measured by Pediatric FACIT Fatigue questionnaire (patients = 5 years of age)<br> ;Timepoint(s) of evaluation of this end point: Week 26