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

Novartis

Status and phase

Completed

Phase 3

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Treatments

Drug: Iptacopan

Study type

Interventional

Funder types

Industry

Identifiers

NCT05630001

2022-502148-10-00 (Other Identifier)

CLNP023C12303

Details and patient eligibility

About

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.

Full description

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.

Enrollment

52 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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