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

Last updated: November 22, 2024
Sponsor: Novartis Pharmaceuticals
Overall Status: Completed

Phase

3

Condition

Aplastic Anemia

Anemia

Bone Marrow Disorder

Treatment

Iptacopan

Clinical Study ID

NCT05630001
CLNP023C12303
2022-502148-10-00
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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 andwith a diagnosis of PNH confirmed by treating physician.

  • Stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumabor ravulizumab) for at least 6 months prior to screening

  • Mean hemoglobin level ≥10 g/dL

  • Vaccination against Neisseria meningitidis and S. pneumoniae infection are requiredprior to the start of iptacopan treatment.

  • If not received previously, vaccination against Haemophilus influenzae infections isrecommended, if available and according to local regulations.

  • Ability to communicate well with the investigator, to understand and comply with therequirements of the study

  • Other protocol -defined inclusion criteria may apply at the end.

Exclusion

Exclusion Criteria:

  • Participation in any other investigational drug trial or use of otherinvestigational drugs at the time of enrollment

  • Patients requiring red blood cell transfusion in the 6 months prior to screening orduring screening

  • History of stem cell transplantation or any solid organ transplantation

  • Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 daysprior to study drug administration

  • Presence of fever ≥ 38.0 °C (100.4 °F) within 7 days prior to study drugadministration

  • Human immunodeficiency virus (HIV) infection (known history of HIV or test positivefor 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, orunstable thrombotic event not amenable to active treatment as judged by theinvestigator 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 inthe trial.

  • Any medical condition deemed likely to interfere with the patient's participation inthe study

  • Female patients who are pregnant or breastfeeding, or intending to conceive duringthe course of the study

Study Design

Total Participants: 52
Treatment Group(s): 1
Primary Treatment: Iptacopan
Phase: 3
Study Start date:
April 24, 2023
Estimated Completion Date:
October 17, 2024

Study 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.

Connect with a study center

  • Novartis Investigative Site

    Nantes Cedex 1, 44093
    France

    Site Not Available

  • Novartis Investigative Site

    Nice, 06202
    France

    Site Not Available

  • Novartis Investigative Site

    Paris 10, 75475
    France

    Site Not Available

  • Novartis Investigative Site

    Aachen, 52074
    Germany

    Site Not Available

  • Novartis Investigative Site

    Dresden, 01307
    Germany

    Site Not Available

  • Novartis Investigative Site

    Essen, 45147
    Germany

    Site Not Available

  • Novartis Investigative Site

    Ulm, 89081
    Germany

    Site Not Available

  • Novartis Investigative Site

    Avellino, AV 83100
    Italy

    Site Not Available

  • Novartis Investigative Site

    San Giovanni Rotondo, FG 71013
    Italy

    Site Not Available

  • Novartis Investigative Site

    Firenze, FI 50134
    Italy

    Site Not Available

  • Novartis Investigative Site

    Bassano Del Grappa, Vicenza 36061
    Italy

    Site Not Available

  • Novartis Investigative Site

    Seoul, 06351
    Korea, Republic of

    Site Not Available

  • Novartis Investigative Site

    Barcelona, Catalunya 08036
    Spain

    Site Not Available

  • Novartis Investigative Site

    Aydin, 09100
    Turkey

    Site Not Available

  • Novartis Investigative Site

    Istanbul, 34093
    Turkey

    Site Not Available

  • Novartis Investigative Site

    Leeds, West Yorkshire LS9 7TF
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    London, SE5 9RS
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    Cerritos, California 90703
    United States

    Site Not Available

  • City Of Hope National Med Center

    Duarte, California 91010
    United States

    Site Not Available

  • City Of Hope National Med Center City of Hope Medical Center

    Duarte, California 91010
    United States

    Site Not Available

  • Novartis Investigative Site

    Duarte, California 91010
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Los Angeles, California 90033
    United States

    Active - Recruiting

  • USC Norris Cancer Center

    Los Angeles, California 90033
    United States

    Site Not Available

  • Novartis Investigative Site

    Torrance, California 90509-2910
    United States

    Site Not Available

  • Lakes Research

    Miami Lakes, Florida 33014
    United States

    Site Not Available

  • Novartis Investigative Site

    Miami Lakes, Florida 33014
    United States

    Site Not Available

  • Novartis Investigative Site

    Augusta, Georgia 30912
    United States

    Site Not Available

  • Mass Gen Hosp Cancer Center

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Novartis Investigative Site

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Worcester, Massachusetts 01665
    United States

    Site Not Available

  • Novartis Investigative Site

    Minneapolis, Minnesota 55455
    United States

    Active - Recruiting

  • University Of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • Novartis Investigative Site

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Montefiore Medical Center

    Bronx, New York 10461
    United States

    Site Not Available

  • Montefiore Medical Center .

    Bronx, New York 10461
    United States

    Site Not Available

  • Novartis Investigative Site

    Bronx, New York 10461
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Cleveland Clinic Foundation

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • Novartis Investigative Site

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Greenville, South Carolina 29615
    United States

    Active - Recruiting

  • Prisma Health Upstate

    Greenville, South Carolina 29615
    United States

    Site Not Available

  • Huntsman Cancer Institute Univ of Utah

    Salt Lake City, Utah 84112 0550
    United States

    Site Not Available

  • Huntsman Cancer Institute Univ of Utah .

    Salt Lake City, Utah 84112 0550
    United States

    Site Not Available

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