- AuthorisedTherapeutic confirmatory (Phase III)
An open-label, single-arm, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients who have completed a clinical trial (parent study) in the iptacopan pediatric program.
This study looks at the long-term safety of a medicine called iptacopan. It is designed for children and young people who have already completed an earlier trial for iptacopan to treat either paroxysmal nocturnal haemoglobinuria (a rare blood condition) or IgA nephropathy (a kidney disease).
For: Paroxysmal Nocturnal Hemoglobinuria (PNH) and IgA nephropathy (IgAN)Italy - RecruitingTherapeutic confirmatory (Phase III)
A multicenter, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult aHUS patients who are naive to complement inhibitor therapy
This study is looking at a new medicine called IPTACOPAN for adults who have a rare kidney disease called atypical hemolytic uremic syndrome (aHUS). It wants to see if the medicine helps improve blood and kidney problems without needing other common treatments.
For: Atypical hemolytic uremic syndromeSlovenia · Slovakia · Czechia - RecruitingTherapeutic confirmatory (Phase III)
A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb≥10 g/dL in response to anti-C5 antibody and switch to iptacopan
This study looks at a new medicine called iptacopan for adults with a rare blood condition called PNH. It checks if switching to iptacopan is safe and works well for patients who are already feeling better on other treatments.
For: Paroxysmal Nocturnal Hemoglobinuria (PNH)Spain · Germany · France - Active not recruitingPHASE2
Iptacopan in Patients With ANCA Associated Vasculitis
This study is looking at a new medicine called iptacopan for people with a rare condition called ANCA-associated vasculitis. It aims to see if iptacopan, when given with standard treatment, can help people get better and stay better, and if it's safe to use.
For: Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated VasculitisUnited States · Argentina · Australia - RecruitingPHASE3
Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.
This study is testing a new medicine called iptacopan for people aged 12-60 with a kidney disease called C3 Glomerulopathy (C3G). It aims to see if the medicine is safe and helps improve kidney function compared to a dummy pill.
For: C3GUnited States · Argentina · Belgium - RecruitingPHASE3
Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN
This study is testing a new medicine, iptacopan, for a kidney disease called IC-MPGN. It wants to see if the medicine can help improve kidney function and reduce protein in the urine, compared to a dummy medicine, in people aged 12 to 60.
For: IC-MPGNUnited States · Argentina · Brazil - Active not recruitingPHASE3
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
This study looks at the long-term safety and effects of a medicine called iptacopan for people with a rare blood condition called PNH. It's for patients who have already been taking iptacopan in other studies and found it helpful, to make sure they can continue treatment safely.
For: Paroxysmal Nocturnal HemoglobinuriaUnited States · Brazil · China - Active not recruitingPHASE3
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.
This study is testing a new medicine called iptacopan for people aged 18-85 with a type of muscle weakness called generalized Myasthenia Gravis (gMG). It's compared to a dummy medicine to see if it's safe and helps improve symptoms while continuing usual care.
For: Generalized Myasthenia GravisUnited States · Argentina · Australia - RecruitingPHASE3
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
This study looks at switching from current aHUS treatment (anti-C5 antibodies) to a new treatment called iptacopan. Researchers want to see if iptacopan is safe and effective for adults with aHUS who are already doing well on their current medication.
For: Atypical Hemolytic Uremic SyndromeChina · France · Germany