Myelodysplastic Syndrome Clinical Trial

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

Summary

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.

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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.

View Eligibility Criteria

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 is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

52

Study ID:

NCT05630001

Recruitment Status:

Active, not recruiting

Sponsor:

Novartis Pharmaceuticals

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There are 23 Locations for this study

See Locations Near You

City Of Hope National Med Center City of Hope Medical Center
Duarte California, 91010, United States
USC Norris Cancer Center
Los Angeles California, 90033, United States
Novartis Investigative Site
Miami Lakes Florida, 33014, United States
Mass Gen Hosp Cancer Center
Boston Massachusetts, 02114, United States
University Of Minnesota
Minneapolis Minnesota, 55455, United States
Montefiore Medical Center .
Bronx New York, 10461, United States
Cleveland Clinic Foundation
Cleveland Ohio, 44195, United States
Prisma Health Upstate
Greenville South Carolina, 29615, United States
Huntsman Cancer Institute Univ of Utah .
Salt Lake City Utah, 84112, United States
Novartis Investigative Site
Nantes Cedex 1 , 44093, France
Novartis Investigative Site
Nice , 06202, France
Novartis Investigative Site
Paris 10 , 75475, France
Novartis Investigative Site
Aachen , 52074, Germany
Novartis Investigative Site
Dresden , 01307, Germany
Novartis Investigative Site
Essen , 45147, Germany
Novartis Investigative Site
Ulm , 89081, Germany
Novartis Investigative Site
Firenze FI, 50134, Italy
Novartis Investigative Site
Bassano Del Grappa VI, 36061, Italy
Novartis Investigative Site
Seoul , 06351, Korea, Republic of
Novartis Investigative Site
Barcelona Catalunya, 08036, Spain
Novartis Investigative Site
Istanbul , 34093, Turkey
Novartis Investigative Site
Leeds , LS9 7, United Kingdom
Novartis Investigative Site
London , SE5 9, United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

52

Study ID:

NCT05630001

Recruitment Status:

Active, not recruiting

Sponsor:


Novartis Pharmaceuticals

How clear is this clinincal trial information?

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