Last Update: Jan 16, 2024
A Multicenter, Single Arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Have Hb≥10 g/dL in Response to Anti-C5 Antibody and Switch to Iptacopan
ClinicalTrials.gov Identifier:
Novartis Reference Number:CLNP023C12303
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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.

This is a multicenter, single-arm, open label trial, with iptacopan treatment for 24 weeks in adult PNH patients.

This study is comprised of two periods:

A Screening period lasting up to 8 weeks.
A 24-week open-label, iptacopan Treatment period.

After completion of the treatment period, participants who continue to benefit from the iptacopan treatment based on the study doctor's evaluation will be able to join the Roll-over extension study.

Paroxysmal Nocturnal Hemoglobinuria
Phase 3
Recruiting
50
Apr 24, 2023
Jan 09, 2025
All
18 Years - (Adult, Older Adult)

Interventions

Drug

Iptacopan

Treatment with iptacopan at a dose of 200 mg b.i.d. will start on the first day (Day 1) and continue for 24 weeks.

Eligibility 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:

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 Location

Novartis Investigative Site

Recruiting

Nantes Cedex 1,44093,France

Novartis Investigative Site

Recruiting

Nice,06202,France

Novartis Investigative Site

Recruiting

Paris 10,75475,France

Novartis Investigative Site

Recruiting

Ulm,89081,Germany

Novartis Investigative Site

Recruiting

Dresden,01307,Germany

Novartis Investigative Site

Recruiting

Aachen,52074,Germany

Novartis Investigative Site

Recruiting

Avellino,AV,83100,Italy

Novartis Investigative Site

Recruiting

Bassano Del Grappa,VI,36061,Italy

Novartis Investigative Site

Recruiting

San Giovanni Rotondo,FG,71013,Italy

Novartis Investigative Site

Recruiting

Firenze,FI,50134,Italy

Novartis Investigative Site

Recruiting

Seoul,06351,Korea, Republic of

Novartis Investigative Site

Recruiting

Barcelona,Catalunya,08036,Spain

Novartis Investigative Site

Recruiting

Istanbul,34093,Turkey

Novartis Investigative Site

Recruiting

Aydin,09100,Turkey

Novartis Investigative Site

Recruiting

Leeds,LS9 7TF,United Kingdom

Novartis Investigative Site

Recruiting

London,SE5 9RS,United Kingdom

Novartis Investigative Site

Recruiting

Bronx,New York,10461,United States

Novartis Investigative Site

Recruiting

Minneapolis,Minnesota,55455,United States

Novartis Investigative Site

Recruiting

Augusta,Georgia,30912,United States

Novartis Investigative Site

Recruiting

Greenville,South Carolina,29615,United States

Novartis Investigative Site

Recruiting

Torrance,California,90509-2910,United States

Novartis Investigative Site

Recruiting

Cerritos,California,90703,United States

Novartis Investigative Site

Recruiting

Cleveland,Ohio,44195,United States

Novartis Investigative Site

Recruiting

Worcester,Massachusetts,01665,United States

Novartis Investigative Site

Recruiting

Duarte,California,91010,United States

Novartis Investigative Site

Recruiting

Saint Louis,Missouri,63110,United States

Novartis Investigative Site

Recruiting

Los Angeles,California,90033,United States

Novartis Investigative Site

Recruiting

Durham,North Carolina,27710,United States

Novartis Investigative Site

Recruiting

Boston,Massachusetts,02114,United States

Novartis Investigative Site

Recruiting

Miami Lakes,Florida,33014,United States

Worldwide Contacts

If the location of your choosing does not feature any contact detail, please reach out using the information below.

Novartis Pharmaceuticals

Novartis Pharmaceuticals