Atypical Hemolytic Uremic SyndromePhase 3NCT05795140
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
A Phase 3 study testing Iptcaopan 200 mg in people with Atypical Hemolytic Uremic Syndrome, run by Novartis Pharmaceuticals at 9 sites in 6 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Atypical Hemolytic Uremic Syndrome
- What is being tested?
- Iptcaopan 200 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 9 sitesin 6 countries
- Study dates
- 8 May 2024to 27 Jul 2032
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent must be obtained prior to participation in the open label extension study
- Willing and able to comply with the study Schedule of Activities
- Participants who have completed the full study treatment period of any prior "Novartis sponsored" iptacopan Phase 3 clinical trial in aHUS (e.g. CLNP023F12301 , CLNP023F12302), are still on iptacopan study treatment and derive benefit from it as per Investigator's judgement
- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e., any boosters required should be administered according to local guidelines)
Who cannot join
- Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
- Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the Investigator could put the participant at risk
- Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
- Pregnant or nursing (lactating) women
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping of investigational drug.
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
9 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
9 sites in 6 countries
Brazil2 sites, 1 recruiting
- Recruiting
Novartis Investigative Site
Rio de Janeiro, 22270-060
- Active, not recruiting
Novartis Investigative Site
São Paulo, São Paulo, 05403 000
India2 sites, 1 recruiting
- Active, not recruiting
Novartis Investigative Site
Nagpur, Maharashtra, 440015
- Recruiting
Novartis Investigative Site
Pune, Maharashtra, 411011
Turkey (Türkiye)2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Ankara, Yenimahalle, 06500
- Recruiting
Novartis Investigative Site
Mersin, Yenisehir, 33110
Czechia1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Ostrava, Poruba, 708 52
Japan1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Iruma-gun, Saitama, 3500495
About this study
Official title: A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS
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.
Read the detailed description
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.
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Being tested
- Iptcaopan 200 mg
- Keywords
- aHUS, iptacopan, atypical hemolytic uremic syndrome, thrombotic microangiopathy, TMA, dialysis, CKD, eGFR, PE/PI, Complement 3 Glomerulopathy (C3G), IgAN, PNH
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome, Thrombotic Microangiopathies, Glomerulonephritis, Membranoproliferative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Iptcaopan 200 mgDrug
Also called LNP023
Open label, participant specific kits, hard gelatin capsules to be taken twice a day
| Study group | Role | What they receive |
|---|---|---|
| Iptacopan 200 mg | Experimental | Open label , single arm Receives: Drug: Iptcaopan 200 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Throughout the study duration, up to 4 years
Secondary outcomes (6)
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)
Measured: 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)
Measured: 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.
Measured: 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
Measured: Throughout study duration, up to 4 years
Number of participants by dialysis requirement status
Dialysis requirement status will be provided
Measured: 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.
Measured: Throughout study duration, up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2023
- Study start8 May 2024
- Primary completion16 Jul 2032 (estimated)
- Study completion27 Jul 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Atypical Hemolytic Uremic SyndromeStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 65 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05795140
- Sponsor's protocol ID
- CLNP023F12001B
- Other IDs
- 2022-502965-34-00
- Status last verified
- May 2026
- First submitted
- 21 Mar 2023
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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
- Expanded access
- Available