Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.

Completed · Phase 3

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria

In brief

The purpose of the study was to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switched from their current standard of care treatment (eculizumab or ravulizumab) to study treatment, iptacopan/LNP023.

Key facts

Study ID

NCT05630001

Run by

Novartis Pharmaceuticals

People needed

52

Starts

2023-04-24

Expected to finish

2024-10-17

Last updated by the study team

2026-01-13

Who can join

Age: 18 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Signed informed consent must be obtained prior to participation in the study.
• Male and female participants ≥ 18 years of age, at the time of ICF signatures and with a diagnosis of PNH confirmed by treating physician.
• Stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumab or ravulizumab) for at least 6 months prior to screening
• Mean hemoglobin level ≥10 g/dL
• Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment.
• If not received previously, vaccination against Haemophilus influenzae infections is recommended, if available and according to local regulations.
• Ability to communicate well with the investigator, to understand and comply with the requirements of the study
• Other protocol -defined inclusion criteria may apply at the end.

You may not qualify if…

• Participation in any other investigational drug trial or use of other investigational drugs at the time of enrollment
• Patients requiring red blood cell transfusion in the 6 months prior to screening or during screening
• History of stem cell transplantation or any solid organ transplantation
• Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to study drug administration
• Presence of fever ≥ 38.0 °C (100.4 °F) within 7 days prior to study drug administration
• Human immunodeficiency virus (HIV) infection (known history of HIV or test positive for HIV antibody at Screening)
• A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus
• Unstable medical condition including, but not limited to, myocardial ischemia, active gastrointestinal bleeding, coexisting chronic anemia unrelated to PNH, or unstable thrombotic event not amenable to active treatment as judged by the investigator at Screening.
• History of cancer of any part of the body within the past 5 years,
• Ongoing drug or alcohol abuse that could interfere with patient's participation in the trial.
• Any medical condition deemed likely to interfere with the patient's participation in the study
• Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study

Where it is running

• City Of Hope National Med Center — Duarte, California, United States
• USC Norris Cancer Center — Los Angeles, California, United States
• Lakes Research — Miami Lakes, Florida, United States
• Mass Gen Hosp Cancer Center — Boston, Massachusetts, United States
• University Of Minnesota — Minneapolis, Minnesota, United States
• Montefiore Medical Center — The Bronx, New York, United States
• Cleveland Clinic Foundation — Cleveland, Ohio, United States
• Prisma Health Upstate — Greenville, South Carolina, United States
• Huntsman Cancer Institute Univ of Utah — Salt Lake City, Utah, United States
• Novartis Investigative Site — Nantes, France
• Novartis Investigative Site — Nice, France
• Novartis Investigative Site — Paris, France
• Novartis Investigative Site — Dresden, Saxony, Germany
• Novartis Investigative Site — Aachen, Germany
• Novartis Investigative Site — Essen, Germany
• Novartis Investigative Site — Ulm, Germany
• Novartis Investigative Site — Florence, FI, Italy
• Novartis Investigative Site — Bassano del Grappa, VI, Italy
• Novartis Investigative Site — Seoul, Seoul, South Korea
• Novartis Investigative Site — Barcelona, Spain
• Novartis Investigative Site — Istanbul, Fatih, Turkey (Türkiye)
• Novartis Investigative Site — Leeds, West Yorkshire, United Kingdom
• Novartis Investigative Site — London, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.