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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
208
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
Proportion of participants with adverse events
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
Time frame: Up to 74 months
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
Time frame: Up to 74 months
Proportion of participants who remain free from transfusions
Proportion of participants who remain free from transfusions evaluated over yearly intervals
Time frame: Up to 74 months
Rate of breakthrough hemolysis (BTH)
Rate of breakthrough hemolysis (BTH)
Time frame: Up to 74 months
Proportion of participants with Major Adverse Vascular Events MAVEs
MAVEs (incl. thrombosis) evaluated over yearly intervals
Time frame: Up to 74 months
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