ERKNet

The European Rare Kidney Disease Reference Network

  

Research Project

Project Title:

Single arm study of ALXN1210 in complement inhibitor treatment-naive adult and adolescent patients with athypical Hemolytic Uremic Syndrome (ALXN1210-aHUS-311 trial)

Project Type:

Clinical Trial, Enrolment ongoing

Disease group(s):

Thrombotic microangiopathies

Project Summary:

The purpose of the study is to assess the efficacy of ALXN1210 to control disease activity in adolescent and adult patients with aHUS who have not previously used a complement inhibitor.

Primary Outcome Measures:
- Complete TMA response

Secondary Outcome Measures:
- 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)

Inclusion Criteria
- Male or female ≥ 12 years of age and weighing ≥ 40 kg at the time of consent.
- Evidence of thrombotic microangiopathy (TMA), including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
- Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study drug. Patients who receive a meningococcal vaccine less than 2 weeks before initiating ALXN1210 treatment must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Patients who have not been vaccinated prior to initiating ALXN1210 treatment should receive prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination. Patients < 18 years of age must have been vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to national and local vaccination schedule guidelines.
- 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.

Exclusion criteria
- ADAMTS13 deficiency (Activity < 5%).
- Shiga toxin-related hemolytic uremic syndrome (STEC-HUS).
- Positive direct Coombs test.
- Pregnancy or breastfeeding.
- Identified drug exposure- related hemolytic uremic syndrome (HUS).
- Bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT) within last 6 months prior to start of Screening.
- HUS related to known genetic defects of cobalamin C metabolism.
- Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome.
- Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for ESKD).

Lead principal investigator(s):

Giuseppe Remuzzi, Bergamo

Project Period:

11/2016   -   12/2019

Sponsors:

Industry

ClinicalTrials.gov:

NCT02949128

EudraCT Nr.:

2016‐002027‐29

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