Atypical Hemolytic Uremic Syndrome (aHUS) MedDRA version: 20.0 Level: LLT Classification code 10019515 Term: Hemolytic uremic syndrome System Organ Class: 100000004851
Conditions
Interventions
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ALXN1210
Other descriptive name: ALXN1210
Sponsors
Alexion Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female = 12 years of age and weighing = 40 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 kidney injury. 3. Documented meningococcal vaccination not more than 3 years prior to dosing, under 18 also requiring 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: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 40 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: 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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Efficacy of ALXN1210; Secondary Objective: Safety and tolerability of ALXN1210 Additional efficacy measures ;Primary end point(s): Complete TMA Response;Timepoint(s) of evaluation of this end point: Week 26 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Dialysis requirement status - Time to Complete TMA Response - Complete TMA Response status over time - 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 = 20g/L from baseline - Change from baseline in quality of life, as measured by the EQ-5D-3L (all patients) - Change from baseline in quality of life, as measured by the FACIT Fatigue Version 4 questionnaire (patients = 18 years of age) - Change from baseline in quality of life, as measured by the Pediatric FACIT Fatigue questionnaire (patients 12 to < 18 years of age) -TMA parameters in patients who discontinue treatment but remain in the study ;Timepoint(s) of evaluation of this end point: Week 26 | — |
Countries
Australia, Austria, Belgium, Czech Republic, France, Germany, Japan, Korea, Republic of, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed