Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS

Launched by NOVARTIS PHARMACEUTICALS · Mar 21, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the long-term effects of a medication called iptacopan in people with a condition known as atypical Hemolytic Uremic Syndrome (aHUS). The goal is to see how safe the medication is, how well it works over a longer period, and if patients can tolerate it without serious side effects. The trial is currently looking for participants who have already completed a previous study involving iptacopan and are still benefiting from it, as determined by their doctor.

To be eligible for this study, participants must be between the ages of 65 and 74, be willing to follow the study's requirements, and have received the necessary vaccinations against certain infections. However, individuals who are currently taking other specific medications or have certain health issues, like active infections or pregnancy, cannot participate. Those who join the trial can expect regular check-ins and assessments to monitor their health while using iptacopan. It's important for potential participants to understand what the study involves and to discuss any questions with their healthcare provider.

Gender

ALL

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, 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.