Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

JRCT ID: jRCT2051210047

Registered date:09/07/2021

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Paroximal nocturnal hemogrobinuria
• Date of first enrollment: 27/04/2022
• Target sample size: 40
• Countries of recruitment: China,Japan,Czech Republic,Japan,France,Japan,Germany,Japan,Italy,Japan,Korea,Japan,Malaysia,Japan,Singapore,Japan,UK,Japan,US,Japan
• Study type: Interventional
• Intervention(s): LNP023 monotherapy

Outcome(s)

• Primary Outcome: Proportion of participants achieving a sustained increase in hemoglobin levels of >= 2 g/dL in the absence of red blood cell transfusion [ Time Frame: Day 168 ]

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 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

Exclude criteria

- 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 <100E9/L; platelets <30E9/L; neutrophils <0.5E9/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.

Related Information

• Primary Sponsor: Suzuki Kazuyuki
• Secondary ID(s): NCT04820530