A Phase 3 interventional study of Iptacopan in Paroxysmal Nocturnal Hemoglobinuria, sponsored by Novartis Pharmaceuticals. Active, not recruiting at 60 sites in 17 countries. Open to participants aged 18 Years to 100 Years. Per ClinicalTrials.gov, last updated 2026-08-07.
Sponsored by Novartis Pharmaceuticals · Phase 3, Interventional, and Treatment
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
The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.
188 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 48 are open to participants now.
This study's enrollment of 208 is above the median of 34 across 147 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply.
Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy
Drug: Iptacopan
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
Also known as: LNP023
Proportion of participants with adverse events
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
Time frame: Up to 74 months
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
Time frame: Up to 74 months
Proportion of participants who remain free from transfusions
Proportion of participants who remain free from transfusions evaluated over yearly intervals
Time frame: Up to 74 months
Rate of breakthrough hemolysis (BTH)
Rate of breakthrough hemolysis (BTH)
Time frame: Up to 74 months
Proportion of participants with Major Adverse Vascular Events MAVEs
MAVEs (incl. thrombosis) evaluated over yearly intervals
Time frame: Up to 74 months
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
This study is active, not recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Novartis Pharmaceuticals