A Phase 3 interventional study of Iptcaopan 200 mg in Atypical Hemolytic Uremic Syndrome, sponsored by Novartis Pharmaceuticals. Recruiting at 9 sites in 6 countries. Open to participants aged 18 Years to 100 Years. Per ClinicalTrials.gov, last updated 2026-06-04.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment
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.
45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.
This study's planned enrollment of 65 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.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Open label , single arm
Drug: Iptcaopan 200 mg
Open label, participant specific kits, hard gelatin capsules to be taken twice a day
Also known as: LNP023
Number of participants with adverse events and serious adverse events
Number of participants with adverse events and serious adverse events, including abnormal safety laboratory parameters, vital signs and ECGs
Time frame: Throughout the study duration, up to 4 years
Number of participants with absence of TMA manifestation without the use of anti-C5 antibody
Thrombotic microangiopathy (TMA) manifestation is defined by the coexistence of at least two of the following three criteria at the same visit attributable to aHUS: * thrombocytopenia (platelet count decrease of ≥ 25% compared to baseline and \< lower limit of normal (LLN)), * microangiopathic hemolytic anemia (hemoglobin ≤ LLN for age and gender and lactate dehydrogenase (LDH) ≥ 1.5 x upper limit of normal (ULN), * worsening kidney function (serum creatinine increase of ≥ 25% 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.
Time 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 is defined as any one 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
Not enrolling at this site.
Not enrolling at this site.
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.
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Studies of the drug tested here: Iptacopan
Atypical Hemolytic Uremic Syndrome →