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An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan.

Status
Active, not recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-509843-28-00
Acronym
CLNP023C12001B
Enrollment
86
Registered
2024-06-05
Start date
2021-11-10
Completion date
Unknown
Last updated
2026-01-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Brief summary

Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc., through End of Study visit

Detailed description

Response defined as maintaining sustained hemoglobin levels ≥ 12 g/dL in the absence of transfusions evaluated over yearly follow up intervals, Absence of administration of packed-red blood cell transfusions evaluated over yearly follow up intervals, Occurrences of breakthrough hemolysis and of Major Adverse Vascular Events, MAVE occurring evaluated over yearly follow up intervals

Interventions

DRUGIPTACOPAN
DRUGLNP023

Sponsors

Novartis Pharma AG
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
18 Years to No maximum

Design outcomes

Primary

MeasureTime frame
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc., through End of Study visit

Secondary

MeasureTime frame
Response defined as maintaining sustained hemoglobin levels ≥ 12 g/dL in the absence of transfusions evaluated over yearly follow up intervals, Absence of administration of packed-red blood cell transfusions evaluated over yearly follow up intervals, Occurrences of breakthrough hemolysis and of Major Adverse Vascular Events, MAVE occurring evaluated over yearly follow up intervals

Countries

Czechia, France, Germany, Italy, Lithuania, Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026