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

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 multicenter, single-arm, open-label extension study aims to assess the long-term safety, tolerability, and efficacy of iptacopan in participants diagnosed with atypical hemolytic uremic syndrome (aHUS). Participants who have previously completed a Phase 3 clinical trial of iptacopan will continue treatment and be monitored through on-site visits every four months. The study includes a follow-up tele-visit to collect data on any adverse events after the last treatment. The goal is to gather comprehensive data on the long-term effects of iptacopan in this patient population.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Signed informed consent must be obtained prior to participation in the open label extension study
2. Willing and able to comply with the study Schedule of Activities
3. 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
4. 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)

Exclusion Criteria:

1. Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
2. 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
3. Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
4. History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
5. Pregnant or nursing (lactating) women
6. 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.

Where it's running

• Novartis Investigative Site
  Rio de Janeiro, Brazil Recruiting
• Novartis Investigative Site
  Beijing, China Recruiting
• Novartis Investigative Site
  Ostrava, Poruba, Czechia Recruiting
• Novartis Investigative Site
  Pune, Maharashtra, India Recruiting
• Novartis Investigative Site
  Iruma-gun, Saitama, Japan Recruiting
• Novartis Investigative Site
  Ankara, Yenimahalle, Turkey (Türkiye) Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: +41613241111

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

  This study is testing if a new RNA sequencing method can help find the genetic causes of rare diseases in people who haven't been diagnosed after other tests.

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

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

  This study is testing if switching to a new treatment called iptacopan can help people with atypical hemolytic uremic syndrome (aHUS) feel better after using a different medication.

  Recruiting at 26 sites Phase 3 Novartis · Nanjing, Jiangsu and 30 other locations · NCT05935215

• 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

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.