An open-label, single-arm, multicenter, phase 3 study to assess pharmacokinetics, safety and tolerability of iptacopan in pediatric PNH patients 2 to <18 years of age
Primary:
To evaluate the safety and tolerability of iptacopan during the 26-week treatment period.
To characterize the PK of iptacopan.
Secondary:
To evaluate the proportion of participants who achieve an increase in hemoglobin levels from baseline of ≥ 1 g/dL and the proportion of patients who achieve an increase in hemoglobin levels from baseline of ≥ 2 g/dL at 26 weeks and at 52 weeks (in the absence of RBC transfusions from Day 14 until the end of the 26-week and 52-week treatment periods).
Chemotherapy multiple agents systemic
- Rutgers University
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.
Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site ClinicalTrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.
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