Atypical Hemolytic Uremic Syndrome Clinical Trial
Official title:
A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With aHUS
The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.
Status | Recruiting |
Enrollment | 50 |
Est. completion date | July 21, 2029 |
Est. primary completion date | January 25, 2029 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Male and female adult participants = 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded. - Currently on the recommended weight-based dosage regimen of anti-C5 antibody treatment for at least 3 months prior to the screening visit. - Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) for at least 3 months prior to entering the screening period as defined by: 1. Hematological normalization in platelet count =150 x 109/L and LDH below upper limit of normal [ULN], and 2. Stable or improving kidney function as defined by =15% increase in serum creatinine. - Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan. - If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations. Exclusion Criteria: - History of aHUS disease relapse while on anti-C5 antibody treatment. - eGFR < 30 ml/min/1.73m^2 - Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae. - Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration. - Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation - Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study - Any medical condition deemed likely to interfere with the patient's participation in the study |
Country | Name | City | State |
---|---|---|---|
Japan | Novartis Investigative Site | Bunkyo ku | Tokyo |
Japan | Novartis Investigative Site | Iruma-gun | Saitama |
Turkey | Novartis Investigative Site | Ankara | |
Turkey | Novartis Investigative Site | Mersin |
Lead Sponsor | Collaborator |
---|---|
Novartis Pharmaceuticals |
Japan, Turkey,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percentage of participants free of TMA manifestation | Absence of thrombotic microangiopathy (TMA) manifestation, without use of anti-C5 antibody, during the 12 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment. | 12 months | |
Secondary | Percentage of participants free of TMA manifestation in study participants with functionally significant mutations in complement genes or positive anti FH antibodies | Absence of thrombotic microangiopathy (TMA) manifestation in study participants with functionally significant mutations in complement genes or positive anti FH antibodies, without the use of anti-C5 antibody during iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment. | 12 months, 24 months | |
Secondary | Percentage of participants free of TMA manifestation | Absence of thrombotic microangiopathy (TMA) manifestation, without use of anti-C5 antibody, during the 24 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment. | 24 months | |
Secondary | Time to TMA manifestation | Time to thrombotic microangiopathy (TMA) manifestation | 12 months, 24 months | |
Secondary | Change from baseline in platelets | Change from baseline in platelets at month 12 and month 24. | Baseline, month 12, month 24 | |
Secondary | Change from baseline in LDH | Change from baseline in lactate dehydrogenase (LDH) at month 12 and month 24. | Baseline, month 12, month 24 | |
Secondary | Change from baseline in hemoglobin | Change from baseline in hemoglobin at month 12 and month 24. | Baseline, month 12, month 24 | |
Secondary | Change from baseline in serum creatinine | Change from baseline in serum creatinine at month 12 and month 24. | Baseline, month 12, month 24 | |
Secondary | Change from baseline in UPCR | Change from baseline in urine protein to creatinine ratio (UPCR) at month 12 and month 24. | Baseline, month 12, month 24 | |
Secondary | Change from baseline in eGFR | Change from baseline in estimated glomerular filtration rate (eGFR) at month 12 and month 24. | Baseline, month 12, month 24 | |
Secondary | Change from baseline in CKD stage | Change from baseline in chronic kidney disease (CKD) stage at month 12 and month 24. | Baseline, month 12, month 24 | |
Secondary | Number of participants who require dialysis | Dialysis requirement status (Yes/ No) | month 12 and month 24 | |
Secondary | Percentage of participants with TMA related events. | Percentage of participants with thrombotic microangiopathy (TMA) related events. | month 12 and month 24 |
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