Study Description
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.
Interventions
Iptacopan
Eligibility Criteria
Inclusion criteria:
* Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension.
* 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:
* 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.
Novartis Investigative Site
Recruiting
Paris 10,75475,France
Novartis Investigative Site
Recruiting
Toulouse,31059,France
Novartis Investigative Site
Recruiting
Lille,59037,France
Novartis Investigative Site
Recruiting
Nantes Cedex 1,44093,France
Novartis Investigative Site
Recruiting
Nice,06202,France
Novartis Investigative Site
Recruiting
Aachen,52074,Germany
Novartis Investigative Site
Recruiting
Dresden,01307,Germany
Novartis Investigative Site
Recruiting
Ulm,89081,Germany
Novartis Investigative Site
Recruiting
Firenze,FI,50134,Italy
Novartis Investigative Site
Recruiting
Milano,MI,20122,Italy
Novartis Investigative Site
Recruiting
Roma,RM,00161,Italy
Novartis Investigative Site
Recruiting
Bassano Del Grappa,Vicenza,36061,Italy
Novartis Investigative Site
Recruiting
Ascoli Piceno,AP,63100,Italy
Novartis Investigative Site
Recruiting
Avellino,AV,83100,Italy
Novartis Investigative Site
Recruiting
Barcelona,Catalunya,08036,Spain
Novartis Investigative Site
Recruiting
London,Se5 9rs,United Kingdom
Novartis Investigative Site
Recruiting
Leeds,West Yorkshire,Ls9 7tf,United Kingdom
Cleveland Clinic Foundation
Recruiting
Cleveland,Ohio,44195,United States
Jaroslaw Maciejewski
Univ of California Irvine (Chao Family Comprehensive Cancer Center)
Recruiting
Orange,California,92868,United States
Zahra Pakbaz
The University of Utah
Recruiting
Salt Lake City,Utah,84132,United States
June Lee
Srinivas Tantravahi
Augusta University
Recruiting
Augusta,Georgia,30912,United States
Abdullah Kutlar
Worldwide Contacts
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