for people ages 18 years and up (full criteria)
at Orange, California and other locations
study started
completion around
Principal Investigator
by Zahra Pakbaz



This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan

Official Title

An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan


The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.


Paroxysmal Nocturnal Hemoglobinuria, Iptacopan, Paroxysmal Nocturnal Hemoglobinuria (PNH), Hemoglobin, Anemia, LNP023, Hemoglobinuria, Paroxysmal Hemoglobinuria


You can join if…

Open to people ages 18 years and up

  • Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension.
  • Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
  • Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months

You CAN'T join if...

  • Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
  • History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
  • History of hematopoietic stem cell transplantation

    Other protocol-defined inclusion/exclusion criteria may apply.


  • Univ of California Irvine (Chao Family Comprehensive Cancer Center) accepting new patients
    Orange California 92868 United States
  • City Of Hope accepting new patients
    Duarte California 91010 United States
  • USC Norris Cancer Center accepting new patients
    Los Angeles California 90033 United States

Lead Scientist at UC Irvine

  • Zahra Pakbaz
    Associate Clinical Professor, Medicine, School of Medicine. Authored (or co-authored) 35 research publications


accepting new patients
Start Date
Completion Date
Novartis Pharmaceuticals
Phase 3 Paroxysmal Nocturnal Hemoglobinuria Research Study
Study Type
Expecting 250 study participants
Last Updated