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

A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS

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.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 65 (estimated)
• Ages: 18 Years to 100 Years
• Sex: All
• Sponsor: Novartis (industry)
• Locations: 8 sites (São Paulo, São Paulo and 7 other locations)
• Trial ID: NCT05795140 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are individuals who have completed a prior Novartis-sponsored Phase 3 clinical trial of iptacopan for aHUS and are still benefiting from the treatment.

Not a fit: Patients currently receiving other complement inhibitors or those with significant comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term management of aHUS, potentially improving patient outcomes.

How similar studies have performed: Other studies involving iptacopan have shown promise, indicating a potential for success in this extension study.

Eligibility criteria

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 this trial is running

São Paulo, São Paulo and 7 other locations

• Novartis Investigative Site — São Paulo, São Paulo, Brazil (ACTIVE_NOT_RECRUITING)
• Novartis Investigative Site — Rio de Janeiro, Brazil (RECRUITING)
• Novartis Investigative Site — Beijing, China (RECRUITING)
• Novartis Investigative Site — Ostrava, Poruba, Czechia (RECRUITING)
• Novartis Investigative Site — Nagpur, Maharashtra, India (ACTIVE_NOT_RECRUITING)
• Novartis Investigative Site — Pune, Maharashtra, India (RECRUITING)
• Novartis Investigative Site — Iruma-gun, Saitama, Japan (RECRUITING)
• Novartis Investigative Site — Mersin, Yenisehir, Turkey (Türkiye) (RECRUITING)

Study contacts

• Study coordinator: Novartis Pharmaceuticals

Conditions: Atypical Hemolytic Uremic Syndrome, aHUS, iptacopan, atypical hemolytic uremic syndrome, thrombotic microangiopathy, TMA, dialysis, CKD