Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

JRCT ID: jRCT2021210059

Registered date:17/12/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Paroxysmal Nocturnal Hemoglobinuria
• Date of first enrollment: 09/12/2021
• Target sample size: 19
• Countries of recruitment: Italy,Japan,Republic of Korea,Japan,Taiwan,Japan
• Study type: Interventional
• Intervention(s): Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral

Outcome(s)

• Primary Outcome: Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include 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

Exclude 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

Related Information

• Primary Sponsor: Suzuki Kazuyuki
• Secondary ID(s): NCT04747613