A Phase 3 interventional study of Iptacopan in Atypical Hemolytic Uremic Syndrome, sponsored by Novartis Pharmaceuticals. Recruiting at 31 sites in 8 countries. Open to participants aged 18 Years to 100 Years. Per ClinicalTrials.gov, last updated 2026-04-29.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment
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 14 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.
45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.
This study's planned enrollment of 50 is above the median of 34 across 26 interventional studies indexed under Atypical Hemolytic Uremic Syndrome.
Browse Atypical Hemolytic Uremic Syndrome studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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 (as per label) dosage regimen of anti-C5 antibody treatment, for at least 3 months prior to entering the screening period.
Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as:
Stable kidney function as defined by serum creatinine values within ±15% during the Screening period
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
open label arm of iptacopan 200 mg b.i.d.
Drug: Iptacopan
Open Label
Also known as: LNP023
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
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
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Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
No publications or documents are linked to this record.
Started Feb 2024 (actual). Primary completion is estimated for Jul 2028, 52 months after the start.
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Studies of the drug tested here: Iptacopan
Atypical Hemolytic Uremic Syndrome →