Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

• Status: Recruiting
• Trial end date: 2026-06-30
• Target enrollment: 0
• Participant gender: All

Summary

This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

Criteria

Inclusion criteria:

• Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed Phase 2 or Phase 3 clinical studies

• Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections

• Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months

Exclusion Criteria:

• Participants who in the parent PNH studies either screen or baseline failed, or prematurely withdrew from the study for any reason

• 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 subject at increased risk or potentially confound study data.

• History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae

• History of hematopoietic stem cell transplantation

Other protocol-defined inclusion/exclusion criteria may apply.