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Atypical Hemolytic Uremic SyndromePhase 3NCT05935215

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

A Phase 3 study testing Iptacopan in people with Atypical Hemolytic Uremic Syndrome, run by Novartis Pharmaceuticals at 31 sites in 8 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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. 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?
Iptacopan
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
31 sitesin 8 countries
Study dates
28 Feb 2024to 19 Jul 2029
Phase
Phase 3
Are there results?
Not yet: the study is under way

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

  • 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.
  • In the opinion of the investigator the participant has responded to anti-C5 antibodytreatment prior to screening and has clinical evidence of response (in absence of PE/PI) during 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:
  • Hematological normalization in platelet count ≥150 x 10^9/L and LDH below upper limit of normal [ULN], and
  • Stable kidney function as defined by serum creatinine values within ±15% during the Screening period
  • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan.
  • If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations.

Who cannot join

  • History of aHUS disease relapse while on anti-C5 antibody treatment.
  • eGFR < 30 ml/min/1.73m^2
  • Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae.
  • Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration.
  • Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation
  • Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
  • Any medical condition deemed likely to interfere with the patient's participation in the study

Trial locations and contacts

31 sites in 8 countries. Contact the study team or a site near you to ask about taking part.

Novartis Pharmaceuticals contact

1-888-669-6682

novartis.email@novartis.com

Novartis Pharmaceuticals contact

+41613241111

31 sites in 8 countries

France6 sites, 6 recruiting

  • Novartis Investigative Site

    Bordeaux, 33076

    Recruiting

  • Novartis Investigative Site

    Paris, 75015

    Recruiting

  • Novartis Investigative Site

    Paris, 75970

    Recruiting

  • Novartis Investigative Site

    Rouen, 76031

    Recruiting

  • Novartis Investigative Site

    Toulouse, 31054

    Recruiting

  • Novartis Investigative Site

    Tours, 37044

    Recruiting

Other trials in France

Spain6 sites, 6 recruiting

  • Novartis Investigative Site

    Barcelona, 08036

    Recruiting

  • Novartis Investigative Site

    Córdoba, 14004

    Recruiting

  • Novartis Investigative Site

    Málaga, 29010

    Recruiting

  • Novartis Investigative Site

    Santiago Compostela, A Coruna, 15706

    Recruiting

  • Novartis Investigative Site

    Seville, 41013

    Recruiting

  • Novartis Investigative Site

    Valencia, 46026

    Recruiting

Other trials in Spain

China4 sites

  • Novartis Investigative Site

    Beijing, 100034

    Active, not recruiting

  • Novartis Investigative Site

    Beijing, 100730

    Active, not recruiting

  • Novartis Investigative Site

    Nanjing, Jiangsu, 210009

    Withdrawn

  • Novartis Investigative Site

    Shanghai, 200025

    Withdrawn

Other trials in China

Turkey (Türkiye)4 sites, 3 recruiting

  • Novartis Investigative Site

    Ankara, Yenimahalle, 06500

    Recruiting

  • Novartis Investigative Site

    Izmir, Balcova, 35340

    Withdrawn

  • Novartis Investigative Site

    Köseköy, Kocaeli, 41380

    Recruiting

  • Novartis Investigative Site

    Mersin, Yenisehir, 33110

    Recruiting

Other trials in Turkey (Türkiye)

Italy3 sites, 3 recruiting

  • Novartis Investigative Site

    Milan, MI, 20122

    Recruiting

  • Novartis Investigative Site

    Ranica, BG, 24020

    Recruiting

  • Novartis Investigative Site

    Roma, RM, 00168

    Recruiting

Other trials in Italy

Japan3 sites, 3 recruiting

  • Novartis Investigative Site

    Bunkyo Ku, Tokyo, 113-8655

    Recruiting

  • Novartis Investigative Site

    Iruma-gun, Saitama, 3500495

    Recruiting

  • Novartis Investigative Site

    Matsumoto-shi, Nagano, 3908510

    Recruiting

Other trials in Japan

United Kingdom3 sites, 3 recruiting

  • Novartis Investigative Site

    Glasgow, Scotland, G51 4TF

    Recruiting

  • Novartis Investigative Site

    London, NW1 2BU

    Recruiting

  • Novartis Investigative Site

    Newcastle upon Tyne, Tyne and Wear, NE7 7DN

    Recruiting

Other trials in United Kingdom

Germany2 sites, 2 recruiting

  • Novartis Investigative Site

    Essen, 45147

    Recruiting

  • Novartis Investigative Site

    Kiel, 24105

    Recruiting

Other trials in Germany

About this study

Official title: A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With aHUS

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.

Read the detailed description

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.

Being tested
Iptacopan
Keywords
Atypical Hemolytic Uremic Syndrome, aHUS,, thrombotic microangiopathy, TMA
Medical subject headings
Atypical Hemolytic Uremic Syndrome, Thrombotic Microangiopathies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • IptacopanDrug

    Also called LNP023

    Open Label

Study groups in NCT05935215
Study groupRoleWhat they receive
iptacopan 200 mg b.i.d.Experimentalopen label arm of iptacopan 200 mg b.i.d.
Receives: Drug: Iptacopan

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: 12 months

Secondary outcomes (12)

  1. 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.

    Measured: 12 months, 24 months

  2. 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.

    Measured: 24 months

  3. Time to TMA manifestation

    Time to thrombotic microangiopathy (TMA) manifestation

    Measured: 12 months, 24 months

  4. Change from baseline in platelets

    Change from baseline in platelets at month 12 and month 24.

    Measured: Baseline, month 12, month 24

  5. Change from baseline in LDH

    Change from baseline in lactate dehydrogenase (LDH) at month 12 and month 24.

    Measured: Baseline, month 12, month 24

  6. Change from baseline in hemoglobin

    Change from baseline in hemoglobin at month 12 and month 24.

    Measured: Baseline, month 12, month 24

  7. Change from baseline in serum creatinine

    Change from baseline in serum creatinine at month 12 and month 24.

    Measured: Baseline, month 12, month 24

  8. Change from baseline in UPCR

    Change from baseline in urine protein to creatinine ratio (UPCR) at month 12 and month 24.

    Measured: Baseline, month 12, month 24

  9. Change from baseline in eGFR

    Change from baseline in estimated glomerular filtration rate (eGFR) at month 12 and month 24.

    Measured: Baseline, month 12, month 24

  10. Change from baseline in CKD stage

    Change from baseline in chronic kidney disease (CKD) stage at month 12 and month 24.

    Measured: Baseline, month 12, month 24

  11. Number of participants who require dialysis

    Dialysis requirement status (Yes/ No)

    Measured: month 12 and month 24

  12. Percentage of participants with TMA related events.

    Percentage of participants with thrombotic microangiopathy (TMA) related events.

    Measured: month 12 and month 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Jul 2023
  2. Study start28 Feb 2024
  3. Primary completion21 Jul 2028 (estimated)
  4. Study completion19 Jul 2029 (estimated)

Resources

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
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05935215
Sponsor's protocol ID
CLNP023F12302
Status last verified
Apr 2026
First submitted
29 Jun 2023
Last updated
29 Apr 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

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Iptacopan in people with Atypical Hemolytic Uremic Syndrome, run by Novartis Pharmaceuticals at 31 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 29 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 31 sites in France, Spain, China, Turkey (Türkiye), Italy and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of participants free of TMA manifestation (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05935215.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Apr 2026.

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