Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
- Conditions
- Paroximal nocturnal hemogrobinuria
- Registration Number
- JPRN-jRCT2051210047
- Lead Sponsor
- Suzuki Kazuyuki
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 40
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 <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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 ]
- Secondary Outcome Measures
Name Time Method