Status:
COMPLETED
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
The purpose of this Phase 3 study was to determine whether iptacopan is efficacious and safe for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH) patients who were naïve to complement inhibi...
Detailed Description
This study was a multicenter, single-arm, open-label trial which was comprised of 8 weeks screening period, 24-week core treatment period and 24-week extension treatment period. Eligible PNH patients...
Eligibility Criteria
Inclusion
- Male and female participants ≥ 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with RBCs and WBCs 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
Exclusion
- 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.
Key Trial Info
Start Date :
July 19 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 18 2023
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT04820530
Start Date
July 19 2021
End Date
April 18 2023
Last Update
October 9 2024
Active Locations (12)
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1
Novartis Investigative Site
Beijing, China, 100730
2
Novartis Investigative Site
Tianjin, China, 300020
3
Novartis Investigative Site
Tianjin, China, 300052
4
Novartis Investigative Site
Paris, France, 75475