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

• Register: EUCTR
• Last refreshed on: 30 November 2020
• Main ID: EUCTR2016-002499-29-IT
• Date of registration: 30/01/2018
• Prospective Registration: No
• 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: 16/08/2017
• Target sample size: 16
• 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

Austria, Belgium, France, Germany, Italy, Spain, Sweden, United Kingdom, United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

Key inclusion & exclusion criteria

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
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

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)


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 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
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)

Main Objective: Efficacy of ALXN1210

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

Secondary Objective: Safety and tolerability of ALXN1210
Additional efficacy measures

Primary end point(s): Complete TMA Response

Secondary Outcome(s)

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 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

Secondary ID(s)

ALXN1210-aHUS-312

2016-002499-29-GB

Source(s) of Monetary Support

Alexion Pharmaceuticals Inc.

Ethics review

Status: Approved
Approval date: 06/06/2017
Contact: