Paroxysmal Nocturnal Hemoglobinuria
Conditions
Interventions
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
Sponsors
Yamauchi Kyosuke
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male and female participants >=18 years of age with a diagnosis of PNH who have completed Phase 2 or Phase 3 clinical studies - 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: - Participants who in the parent PNH studies either screen or baseline failed, or prematurely withdrew from the study for any reason - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit | — |
Countries
Inited Kingdom, Italy, Japan, Republic of Korea, Taiwan, United State
Contacts
Public ContactKyosuke Yamauchi
Novartis Pharma. K.K.
Outcome results
None listed