This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.
The extension study Baseline/Day 1 visit is equivalent to the End of Treatment visit of the parent study. The study will begin on Day 1 followed by on-site visits every 4 months during the study treatment period. A Safety Follow Up tele-visit must be conducted 7 days after last study treatment to collect information on Adverse Events.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
125
Open label, participant specific kits, hard gelatin capsules to be taken twice a day
Novartis Investigative Site
São Paulo, São Paulo, Brazil
RECRUITINGNovartis Investigative Site
Rio de Janeiro, Brazil
RECRUITINGNovartis Investigative Site
Beijing, China
Number of participants with adverse events and serious adverse events
Number of participants with adverse events and serious adverse events will be provided
Time frame: Throughout the study duration, up to 4 years
Number of participants with abnormal safety laboratory parameters, vital signs and ECGs
Number of participants with abnormal safety laboratory parameters , vital signs and ECGs will be provided
Time frame: Throughout the study duration, up to 4 years
Number of participants with absence of aHUS relapse without the use of anti-C5 antibody
Atypical hemolytic uremic syndrome (aHUS) relapse is defined by the coexistence of at least two of the following at the same visit: * thrombocytopenia (platelet count \< 150 x 109 /L), * microangiopathic hemolytic anemia (hemoglobin \< 10 g/dl, LDH \> upper limit of normal, undetectable haptoglobin, presence of schistocyte on blood smear), * worsening kidney function (serum creatinine or urine protein to creatinine ratio (UPCR) \> upper limit of normal and an increase of ≥ 15% compared to baseline levels)
Time frame: Throughout the study duration, up to 4 years
Number of participants with complete TMA response status without the use of anti-C5 antibody therapy
Complete thrombotic microangiopathy (TMA) Response is defined as (1) hematological normalization in platelet count (platelet count ≥150 x 109/L) and LDH (below ULN), and (2) improvement in kidney function (≥ 25% serum creatinine reduction from baseline or ≥ 25% serum creatinine reduction compared to serum creatinine values prior to initiation of anti-C5 antibody therapy)
Time frame: Throughout the study duration, up to 4 years
Estimated glomerular filtration rate (eGFR)
Estimated glomerular filtration rate (eGFR) based on eGFR categories will be collected. Serum creatinine as measured in mg/dL as part of the clinical chemistry panel through the central laboratory will be used to calculate the eGFR applying the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
Novartis Pharmaceuticals
CONTACT
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Novartis Investigative Site
Ostrava, Poruba, Czechia
RECRUITINGNovartis Investigative Site
Nagpur, Maharashtra, India
RECRUITINGNovartis Investigative Site
Pune, Maharashtra, India
RECRUITINGTime frame: Throughout study duration, up to 4 years
Chronic kidney disease (CKD) stage
Chronic kidney disease (CKD) stage (1-5) based on eGFR categories will be provided: * Stage 1 (G1): Kidney damage with normal kidney function * Stage 2 (G2): Mild loss of kidney function * Stage 3 (G3): 3a: Mild to moderate loss of kidney function; 3b: Moderate to severe loss of kidney function * Stage 4 (G4): Severe loss of kidney function * Stage 5 End stage renal disease (kidney failure): Kidney failure and need for transplant or dialysis
Time frame: Throughout study duration, up to 4 years
Number of participants by dialysis requirement status
Dialysis requirement status will be provided
Time frame: Throughout the study duration, up to 4 years
Number of participants with Thrombotic Microangiopathy (TMA) related adverse events
TMA related events during the study defined as any of the following: * Irreversible (\>3 months) reduction in eGFR rate by ≥20%, not attributable to another cause * An episode of acute kidney injury (AKI) attributed to a TMA that requires renal replacement therapy * A non-renal manifestation of a TMA that requires hospitalization, or causes irreversible organ damage or death.
Time frame: Throughout study duration, up to 4 years