Phase 3
N=58
Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome (aHUS)
Bottom Line
View on ClinicalTrials.gov: NCT02949128 ↗Enrolled (actual)
58
Serious AEs
65.5%
Results posted
Feb 2020
Primary outcome: Primary: Percentage Of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 — 53.6; 83.9; 76.8; 58.9 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Ravulizumab (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Jan 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 |
53.6; 83.9; 76.8; 58.9 | — |
| SECONDARY Time To Complete TMA Response |
86.0 | — |
| SECONDARY Participants Who Do Not Require Dialysis at Weeks 26 and 52 |
16; 16 | — |
| SECONDARY Proportion Of Participants With Complete TMA Response At Week 52 |
0.500; 0.909; 0.750; 0.659 | — |
| SECONDARY Change From Baseline In Estimated Glomerular Filtration Rate (eGFR) At Weeks 26 and 52 |
10.00; 29.00; 23.00 | — |
| SECONDARY Participants With Change From Baseline In CKD Stage At Weeks 26 and 52 |
32; 2; 13; 30; 2; 11 | — |
| SECONDARY Change From Baseline In Platelet Count At Weeks 26 and 52 |
95.25; 125.00; 126.25 | — |
| SECONDARY Change From Baseline In LDH At Weeks 26 and 52 |
508.00; -310.75; -293.75 | — |
| SECONDARY Change From Baseline In Hemoglobin At Weeks 26 and 52 |
85.00; 35.00; 41.75 | — |
| SECONDARY Percentage Of Complement Inhibitor Treatment-naïve Participants With An Increase From Baseline In Hemoglobin ≥20 g/L Through Week 26 and Week 52 |
75.5; 86.4 | — |
| SECONDARY Change From Baseline In Quality Of Life As Measured By The EuroQol 5-Dimension 3-Level (EQ-5D-3L) At Weeks 26 and 52 |
0.59; 0.15; 0.26 | — |
| SECONDARY Change From Baseline In Quality Of Life As Measured By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Version 4 Questionnaire At Weeks 26 and 52 |
24.00; 20.00; 16.50 | — |
Summary
The purpose of the study is to assess the safety and efficacy of ravulizumab to control disease activity in adolescent and adult participants with aHUS who had not previously used a complement inhibitor.
Eligibility Criteria
Inclusion Criteria
- Male or female ≥ 12 years of age and weighing ≥ 40 kg at the time of consent.
- Evidence of thrombotic microangiopathy, 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. Participants who received a meningococcal vaccine less than 2 weeks before initiating ravulizumab treatment must have received treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Participants who had not been vaccinated prior to initiating ravulizumab treatment should have received prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination. Participants < 18 years of age must have been vaccinated against haemophilus influenzae type b and streptococcus pneumoniae according to national and local vaccination schedule guidelines.
- Female participants of childbearing potential and male participants with female partners of childbearing potential had to use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
Exclusion Criteria
- A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 deficiency (activity < 5%).
- Shiga toxin-related hemolytic uremic syndrome.
- Positive direct Coombs test.
- Pregnancy or breastfeeding.
- Identified drug exposure-related hemolytic uremic syndrome (HUS).
- Bone marrow transplant/hematopoietic stem cell transplant 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, or antiphospholipid antibody positivity or syndrome.
- Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage kidney disease).
Data sourced from ClinicalTrials.gov (NCT02949128). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.