All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.
The purpose of this Phase 3 study is to determine whether iptacopan is efficacious and safe for the treatment of PNH patients who are naive to complement inhibitor therapy
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Jul 19, 2021
Jul 10, 2023
18 Years and older (Adult, Older Adult)
Taken orally b.i.d. Dosage supplied: 200mg Dosage form: Hard gelatin capsule Route of Administration: oral
Male and female participants ≥ 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%
Mean hemoglobin level <10 g/dL
LDH > 1.5 x Upper Limit of Normal (ULN)
Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment
If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given
Prior treatment with a complement inhibitor, including anti-C5 antibody
Known or suspected hereditary complement deficiency
History of hematopoietic stem cell transplantation
Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L).
Active systemic bacterial, viral (incl. COVID-19)or fungal infection within 14 days prior to study drug administration.
History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
Major concurrent comorbidities including but not limited to severe kidney disease (e.g., dialysis), advanced cardiac disease (e.g., NYHA class IV heart failure), severe pulmonary disease (e.g., severe pulmonary hypertension (WHO class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.
Other protocol defined inclusion/exclusion criteria may apply.