NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

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

NCT05935215

RecruitingPhase 3

Sponsor: Novartis Pharmaceuticals

Conditions: Atypical Hemolytic Uremic Syndrome

Interventions: Iptacopan

Countries: China, France, Germany, Italy, Japan, Spain, Turkey (Türkiye), United Kingdom

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.

Eligibility overview

Sex: ALL

Age: 18 Years to 100 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Inclusion Criteria:

* 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:

1. Hematological normalization in platelet count ≥150 x 10\^9/L and LDH below upper limit of normal \[ULN\], and
2. 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.

Exclusion Criteria:

* 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
Locations (31)
  • Nanjing, Jiangsu, China
  • Beijing, China
  • Beijing, China
  • Shanghai, China
  • Bordeaux, France
  • Paris, France
  • Paris, France
  • Rouen, France
  • Toulouse, France
  • Tours, France
  • Essen, Germany
  • Kiel, Germany
  • Ranica, BG, Italy
  • Milan, MI, Italy
  • Roma, RM, Italy
  • Matsumoto-shi, Nagano, Japan
  • Iruma-gun, Saitama, Japan
  • Bunkyo Ku, Tokyo, Japan
  • Santiago Compostela, A Coruna, Spain
  • Barcelona, Spain
  • Córdoba, Spain
  • Málaga, Spain
  • Seville, Spain
  • Valencia, Spain
  • Izmir, Balcova, Turkey (Türkiye)
  • Köseköy, Kocaeli, Turkey (Türkiye)
  • Ankara, Yenimahalle, Turkey (Türkiye)
  • Mersin, Yenisehir, Turkey (Türkiye)
  • Glasgow, Scotland, United Kingdom
  • Newcastle upon Tyne, Tyne and Wear, United Kingdom
  • + 1 more on CT.gov