Atypical Hemolytic Uremic Syndrome (aHUS) Atypical Hemolytic Uremic Syndrome (aHUS)
Conditions
Interventions
The dosing regimen of ravulizumab based on the participant's body weight consists of a loading dose of 900 to 3000 mg followed by administration of 2100 to 3600 mg 2 weeks after the loading dose, then
Sponsors
Sugita Yuko
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1 Participants must be at least 18 years of age at the time of signing the ICF. 2 Body weight >=20 kg 3 Patients clinically diagnosed as aHUS who have any of diseases/conditions listed below (including patients in whom TMA has not been improved even after treatment for the pathogenesis of diagnosed secondary TMA and therefore, diagnosis of aHUS was made). - Infection (except for pneumococcal infection and Siga toxin-producing Escherichia coli infection) - During pregnancy or postpartum - Post-renal transplantation - Hypertensive crisis/malignant hypertension - Systemic lupus erythematosus and related diseases (e.g. dermatomyositis, mixedconnective tissue disease, etc.)
Exclusion criteria
Exclusion criteria: 1 Participants must be at least 18 years of age at the time of signing the ICF. 2 Body weight >=20 kg 3 Patients clinically diagnosed as aHUS who have any of diseases/conditions listed below (including patients in whom TMA has not been improved even after treatment for the pathogenesis of diagnosed secondary TMA and therefore, diagnosis of aHUS was made). - Infection (except for pneumococcal infection and Siga toxin-producing Escherichia coli infection) - During pregnancy or postpartum - Post-renal transplantation - Hypertensive crisis/malignant hypertension - Systemic lupus erythematosus and related diseases (e.g. dermatomyositis, mixedconnective tissue disease, etc.) 4 Patients with the following three signs: - Thrombocytopenia: Platelet count = 1.5 times ULN) and a marked decrease in serum haptoglobin. Fractured erythrocytes may not be detected. (*) Even if Hb less than 10 g/dL is not fulfilled, the presence of microangiopathic haemolytic anaemia is considered to be a sign of this condition. - Acute kidney injury: one of the followings is fulfilled, 1. delta sCr >= 0.3 mg/dL (within 48 hours), 2. 1.5-fold increase from baseline sCr (within 7 days), 3. urinary output = 6 hours. 5 No prior treatment with complement inhibitors. 6 The investigator plans to provide the participant with 26-week treatment with ravulizumab in accordance with the treatment policy in clinical practice. 7 Ravulizumab treatment is planned to be initiated within 14 days after onset of the latest TMA episode. 8 Patients consenting to meningococcal vaccine administration and appropriate antibiotic prophylaxis (if required). 9 For female participants of childbearing potential or female participants who may be pregnant, the investigator must have judged that the therapeutic benefits of ravulizumab outweigh its potential risks. 10 For lactating female participants, the investigator must have considered continuation or discontinuation of breastfeeding taking into consideration the therapeutic benefits of ravulizumab and the benefits of breastfeeding, and judged that treatment with ravulizumab is acceptable. 11 Participants must be capable of giving signed informed consent as described in Section 10.1.3, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of participants showing improvement in platelet count at any observation timepoint during the 26-week ravulizumab treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| - Proportion of participants showing improvement in renal function at any observation timepoint during the 26-week ravulizumab treatment (key secondary endpoint) - Proportions of participants showing improvement in platelet count, improvement in renal function, complete TMA response, and partial TMA response at each observation timepoint during ravulizumab treatment - Proportion of participants who are able to withdraw from dialysis by Week 26 among those who are on dialysis on Day 1 - Changes from Day 1 to the last observation timepoint in platelet count, hemoglobin, LDH, and eGFR | — |
Contacts
Public ContactYasuo Miyaguchi
Alexion Pharma GK
Outcome results
None listed