Switching from anti-C5 antibody treatment to iptacopan for aHUS patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3 study evaluates the efficacy and safety of iptacopan in patients with atypical hemolytic uremic syndrome (aHUS) who are switching from anti-C5 antibody treatment. It is a multicenter, single-arm, open-label study that includes a screening period of up to 8 weeks, followed by a 12-month core treatment period and an additional 12-month extension treatment period. The study will assess various efficacy outcomes relevant to aHUS to determine the benefits of iptacopan compared to the previous treatment.

Why it matters

Who can join: the full rules

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

Where it's running

• Novartis Investigative Site
  Bordeaux, France Recruiting
• Novartis Investigative Site
  Paris, France Recruiting
• Novartis Investigative Site
  Paris, France Recruiting
• Novartis Investigative Site
  Rouen, France Recruiting
• Novartis Investigative Site
  Toulouse, France Recruiting
• Novartis Investigative Site
  Tours, France Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Ravulizumab Outcomes in Polish Patients with aHUS

  This observational study in Poland follows patients with atypical hemolytic uremic syndrome who are treated with ravulizumab to see how they do over time in real-world clinical care.

  Recruiting at 5 sites AstraZeneca · Gdansk and 9 other locations · NCT07399730

• Ravulizumab for people with clinical aHUS

  This study will test whether ravulizumab raises platelet counts in people diagnosed with clinical atypical hemolytic uremic syndrome (aHUS), including those with certain infections (excluding pneumococcal and STEC), pregnancy/postpartum, recent kidney transplant, malignant hypertension, or autoimmune conditions.

  Recruiting at 3 sites Phase 4 Alexion Pharmaceuticals, Inc. · Bunkyō City and 11 other locations · NCT07308574

• National Registry for Rare Kidney Diseases

  This study is collecting health information from people with rare kidney diseases to help improve treatments and create better care guidelines.

  Recruiting UK Kidney Association · Bristol, South West · NCT06065852

• Using RNA sequencing to diagnose rare genetic diseases

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  Recruiting Mario Negri Institute for Pharmacological Research · Ranica, BG · NCT05996731

• Understanding rare gene mutations in atypical hemolytic uremic syndrome for personalized therapy

  This study is testing how rare gene mutations in people with atypical hemolytic uremic syndrome can help doctors create personalized treatments to improve their health.

  Recruiting Mario Negri Institute for Pharmacological Research · Ranica, BG · NCT05805202

• Long-term evaluation of iptacopan for patients with atypical hemolytic uremic syndrome

  This study is testing how well the medication iptacopan works and how safe it is for people with atypical hemolytic uremic syndrome over a longer period of time.

  Recruiting at 7 sites Phase 3 Novartis · São Paulo, São Paulo and 8 other locations · NCT05795140

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.