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.
Worldwide Contacts
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