Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
About the study
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
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION 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
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.
Study Locations
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How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Paroxysmal Nocturnal Hemoglobinuria
Age (in years)
18+
Phase
Phase 3
Participants needed
250
Est. Completion Date
Oct 14, 2027
Treatment type
Interventional
Sponsor
Novartis
ClinicalTrials.gov identifier
NCT04747613
Study number
CLNP023C12001B
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