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Recruiting Phase 3 NCT05795140

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

Trial Parameters

Condition Atypical Hemolytic Uremic Syndrome
Sponsor Novartis Pharmaceuticals
Study Type INTERVENTIONAL
Phase Phase 3
Enrollment 125
Sex ALL
Min Age 18 Years
Max Age 100 Years
Start Date 2024-05-08
Completion 2032-12-23
Interventions
Iptcaopan 200 mg

Brief Summary

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.

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

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