Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.
About the study
The purpose of the study is to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switch from their current standard of care treatment (eculizumab or ravulizumab) to study treatment, iptacopan/LNP023.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- 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.
EXCLUSION CRITERIA
Exclusion Criteria:
- 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
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study Details
Contition
Paroxysmal Nocturnal Hemoglobinuria
Age (in years)
18+
Phase
PHASE3
Participants Needed
52
Est. Completion Date
Oct 17, 2024
Treatment Type
INTERVENTIONAL
Sponsor
Novartis
ClinicalTrials.gov NCT Identifier
NCT05630001
Study Number
CLNP023C12303
Understanding Clinical Trials
Get answers to your questions about clinical trials.What is clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?