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.
The study is designed as a multicenter, single-arm, open label study to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in participants with aHUS. It consists of a screening period of up to 8 weeks followed by a 12-Month Core Treatment period and 12-Month Extension Treatment period. The study will assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Open Label
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.
Time frame: 12 months
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.
Time frame: 12 months, 24 months
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.
Time frame: 24 months
Time to TMA manifestation
Time to thrombotic microangiopathy (TMA) manifestation
Time frame: 12 months, 24 months
Change from baseline in platelets
Change from baseline in platelets at month 12 and month 24.
Time frame: Baseline, month 12, month 24
Change from baseline in LDH
Change from baseline in lactate dehydrogenase (LDH) at month 12 and month 24.
Time frame: Baseline, month 12, month 24
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Novartis Investigative Site
Nanjing, Jiangsu, China
RECRUITINGNovartis Investigative Site
Beijing, China
RECRUITINGNovartis Investigative Site
Beijing, China
RECRUITINGNovartis Investigative Site
Shanghai, China
RECRUITINGNovartis Investigative Site
Bordeaux, France
RECRUITINGNovartis Investigative Site
Paris, France
RECRUITINGNovartis Investigative Site
Paris, France
RECRUITINGNovartis Investigative Site
Rouen, France
RECRUITINGNovartis Investigative Site
Toulouse, France
RECRUITINGNovartis Investigative Site
Tours, France
RECRUITING...and 19 more locations
Change from baseline in hemoglobin
Change from baseline in hemoglobin at month 12 and month 24.
Time frame: Baseline, month 12, month 24
Change from baseline in serum creatinine
Change from baseline in serum creatinine at month 12 and month 24.
Time frame: Baseline, month 12, month 24
Change from baseline in UPCR
Change from baseline in urine protein to creatinine ratio (UPCR) at month 12 and month 24.
Time frame: Baseline, month 12, month 24
Change from baseline in eGFR
Change from baseline in estimated glomerular filtration rate (eGFR) at month 12 and month 24.
Time frame: Baseline, month 12, month 24
Change from baseline in CKD stage
Change from baseline in chronic kidney disease (CKD) stage at month 12 and month 24.
Time frame: Baseline, month 12, month 24
Number of participants who require dialysis
Dialysis requirement status (Yes/ No)
Time frame: month 12 and month 24
Percentage of participants with TMA related events.
Percentage of participants with thrombotic microangiopathy (TMA) related events.
Time frame: month 12 and month 24