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

Recruiting now · Phase 3

Conditions studied: Atypical Hemolytic Uremic Syndrome

In brief

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.

Key facts

Study ID

NCT05935215

Run by

Novartis Pharmaceuticals

People needed

50

Starts

2024-02-28

Expected to finish

2029-07-19

Last updated by the study team

2026-04-29

Who can join

Age: 18 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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

Where it is running

• Novartis Investigative Site — London, United Kingdom (enrolling)
• Novartis Investigative Site — Köseköy, Kocaeli, Turkey (Türkiye) (enrolling)
• Novartis Investigative Site — Ankara, Yenimahalle, Turkey (Türkiye) (enrolling)
• Novartis Investigative Site — Mersin, Yenisehir, Turkey (Türkiye) (enrolling)
• Novartis Investigative Site — Glasgow, Scotland, United Kingdom (enrolling)
• Novartis Investigative Site — Newcastle upon Tyne, Tyne and Wear, United Kingdom (enrolling)
• Novartis Investigative Site — Bordeaux, France (enrolling)
• Novartis Investigative Site — Paris, France (enrolling)
• Novartis Investigative Site — Paris, France (enrolling)
• Novartis Investigative Site — Rouen, France (enrolling)
• Novartis Investigative Site — Toulouse, France (enrolling)
• Novartis Investigative Site — Tours, France (enrolling)
• Novartis Investigative Site — Essen, Germany (enrolling)
• Novartis Investigative Site — Kiel, Germany (enrolling)
• Novartis Investigative Site — Ranica, BG, Italy (enrolling)
• Novartis Investigative Site — Milan, MI, Italy (enrolling)
• Novartis Investigative Site — Roma, RM, Italy (enrolling)
• Novartis Investigative Site — Matsumoto-shi, Nagano, Japan (enrolling)
• Novartis Investigative Site — Iruma-gun, Saitama, Japan (enrolling)
• Novartis Investigative Site — Bunkyo Ku, Tokyo, Japan (enrolling)
• Novartis Investigative Site — Santiago Compostela, A Coruna, Spain (enrolling)
• Novartis Investigative Site — Barcelona, Spain (enrolling)
• Novartis Investigative Site — Córdoba, Spain (enrolling)
• Novartis Investigative Site — Málaga, Spain (enrolling)
• Novartis Investigative Site — Seville, Spain (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.