Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)

A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-002499-29-GB

Enrollment

Registered

2017-02-09

Start date

2017-05-18

Completion date

Unknown

Last updated

2018-10-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Atypical Hemolytic Uremic Syndrome (aHUS) MedDRA version: 20.0 Level: LLT Classification code 10019515 Term: Hemolytic uremic syndrome System Organ Class: 100000004851

Interventions

Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Current Sponsor code: ALXN1210 Othe

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Cohort 1 inclusion criteria: 1. Patients from birth up to =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Main Objective: Efficacy of ALXN1210 in complement inhibitor treatment naïve pediatric patients (ie, Cohort 1).;Secondary Objective: To assess in both complement inhibitor naïve pediatric patients (ie, Cohort 1) and complement inhibitor experienced adolescent (i.e. Cohort 2): - Safety and tolerability of ALXN1210 - Additional efficacy measures;Primary end point(s): Complete TMA Response (only for Cohort 1);Timepoint(s) of evaluation of this end point: Week 26	—

Measure

Time frame

Main Objective: Efficacy of ALXN1210 in complement inhibitor treatment naïve pediatric patients (ie, Cohort 1).;Secondary Objective: To assess in both complement inhibitor naïve pediatric patients (ie, Cohort 1) and complement inhibitor experienced adolescent (i.e. Cohort 2): - Safety and tolerability of ALXN1210 - Additional efficacy measures;Primary end point(s): Complete TMA Response (only for Cohort 1);Timepoint(s) of evaluation of this end point: Week 26

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Secondary

Measure	Time frame
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) ;Timepoint(s) of evaluation of this end point: Week 26	—

Measure

Time frame

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) ;Timepoint(s) of evaluation of this end point: Week 26

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Countries

Australia, Austria, Belgium, Canada, France, Germany, Italy, Japan, Korea, Republic of, Russian Federation, Spain, Sweden, Taiwan, United Kingdom, United States

Contacts

Public ContactEuropean Clinical Trial Information

Alexion Europe SAS

clinicaltrials.eu@alexion.com+33147100606

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026