Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
About the study
This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- Signed informed consent must be obtained prior to participation in the open label extension study
- Willing and able to comply with the study Schedule of Activities
- 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
- 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
Exclusion Criteria:
- Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
- 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
- Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
- Pregnant or nursing (lactating) women
- 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.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Atypical Hemolytic Uremic Syndrome
Age (in years)
18+
Phase
Phase 3
Participants needed
150
Est. Completion Date
Aug 23, 2029
Treatment type
Interventional
Sponsor
Novartis
ClinicalTrials.gov identifier
NCT05795140
Study number
CLNP023F12001B
Understanding Clinical Trials
Get answers to your questions about clinical trials.What is a clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?