* CLNP023C12001B
Research type
Research Study
Full title
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.
IRAS ID
1005096
Contact name
Claus-Peter Danzer
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2020-004385-19
Clinicaltrials.gov Identifier
NCT04747613
Research summary
The study is being performed to evaluate the long term safety, tolerability and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH) and to provide access to the medication for patients who have completed phase 2 and phase 3 trials and derived benefit from iptacopan treatment.
Eligible patients will include those with a diagnosis of PNH and who have participated in the phase 2 and phase 3 studies as listed in the protocol.
The sites involved in the study are hospital Haematology departments. All participants in the study will receive open label oral iptacopan 200mg b.i.d.
Participants will visit the hospital every 4 months during the first year and every 6 months during year 2 and beyond. Participants will receive follow-up phone calls every two months for the duration of the study. It is anticipated that participants will be in the study until 3 years after the last patient is enrolled.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
22/EE/0208
Date of REC Opinion
27 Apr 2022
REC opinion
Further Information Favourable Opinion