For Healthcare Professionals

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

clipboard-pencil

About the study

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
user-3

Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

Inclusion criteria:

  1. 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.
  2. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
  3. Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months

EXCLUSION CRITERIA

Exclusion Criteria:

  1. 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.
  2. History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
  3. History of hematopoietic stem cell transplantation

Other protocol-defined inclusion/exclusion criteria may apply.

pin location

Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.

Study’s details


Contition

Paroxysmal Nocturnal Hemoglobinuria

Age (in years)

18+

Phase

Phase 3

Participants needed

250

Est. Completion Date

Oct 14, 2027

Treatment type

Interventional


Sponsor

Novartis

ClinicalTrials.gov identifier

NCT04747613

Study number

CLNP023C12001B

Understanding Clinical Trials


Get answers to your questions about clinical trials.What is a clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?
Vector

Interested?

Sign up to save your favorites, 
receive newsletters, resources, and alerts 
about clinical trials related to your conditions of interest.