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

August 6, 2026 updated by: Novartis Pharmaceuticals

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

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
208

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

  • Name: Novartis Pharmaceuticals
  • Phone Number: +41613241111

Study Locations

  • Brazil
    • São Paulo
      • Santo André, São Paulo, Brazil, 09090-790
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brazil, 01323-900
        • Novartis Investigative Site
  • China
      • Beijing, China, 100730
        • Novartis Investigative Site
      • Tianjin, China, 300052
        • Novartis Investigative Site
      • Tianjin, China, 300020
        • Novartis Investigative Site
  • Czechia
      • Brno, Czechia, 625 00
        • Novartis Investigative Site
    • Poruba
      • Ostrava, Poruba, Czechia, 708 52
        • Novartis Investigative Site
  • France
      • Lille, France, 59037
        • Novartis Investigative Site
      • Nantes, France, 44093
        • Novartis Investigative Site
      • Nice, France, 06202
        • Novartis Investigative Site
      • Paris, France, 75475
        • Novartis Investigative Site
      • Toulouse, France, 31059
        • Novartis Investigative Site
  • Germany
      • Aachen, Germany, 52074
        • Novartis Investigative Site
      • Essen, Germany, 45147
        • Novartis Investigative Site
      • Hamburg, Germany, 20246
        • Novartis Investigative Site
      • Riesa, Germany, 01589
        • Novartis Investigative Site
      • Ulm, Germany, 89081
        • Novartis Investigative Site
    • Saxony
      • Dresden, Saxony, Germany, 01307
        • Novartis Investigative Site
  • Italy
    • AP
      • Ascoli Piceno, AP, Italy, 63100
        • Novartis Investigative Site
    • AV
      • Avellino, AV, Italy, 83100
        • Novartis Investigative Site
    • FI
      • Florence, FI, Italy, 50134
        • Novartis Investigative Site
    • MI
      • Milan, MI, Italy, 20122
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italy, 00161
        • Novartis Investigative Site
    • TO
      • Torino, TO, Italy, 10126
        • Novartis Investigative Site
    • VI
      • Bassano del Grappa, VI, Italy, 36061
        • Novartis Investigative Site
  • Japan
      • Fukushima, Japan, 9601295
        • Novartis Investigative Site
      • Ishikawa, Japan, 9208641
        • Novartis Investigative Site
      • Kyoto, Japan, 6068507
        • Novartis Investigative Site
      • Niigata, Japan, 9518520
        • Novartis Investigative Site
    • Aichi-ken
      • Nagoya, Aichi-ken, Japan, 453-8511
        • Novartis Investigative Site
    • Kanagawa
      • Isehara, Kanagawa, Japan, 259-1193
        • Novartis Investigative Site
    • Nagano
      • Suwa, Nagano, Japan, 392-8510
        • Novartis Investigative Site
    • Osaka
      • Suita, Osaka, Japan, 5650871
        • Novartis Investigative Site
    • Tokyo
      • Shinjuku Ku, Tokyo, Japan, 160-0023
        • Novartis Investigative Site
  • Lithuania
      • Vilnius, Lithuania, LT-08406
        • Novartis Investigative Site
  • Malaysia
    • Sabah
      • Kota Kinabalu, Sabah, Malaysia, 88586
        • Novartis Investigative Site
    • Sarawak
      • Kuching, Sarawak, Malaysia, 93586
        • Novartis Investigative Site
  • Netherlands
      • Nijmegen, Netherlands, 6525 GA
        • Novartis Investigative Site
  • Singapore
      • Singapore, Singapore, 119074
        • Novartis Investigative Site
  • South Korea
    • Seoul
      • Seoul, Seoul, South Korea, 06351
        • Novartis Investigative Site
      • Seoul, Seoul, South Korea, 03080
        • Novartis Investigative Site
  • Spain
      • Barcelona, Spain, 08036
        • Novartis Investigative Site
    • A Coruna
      • Santiago Compostela, A Coruna, Spain, 15706
        • Novartis Investigative Site
    • Gipuzkoa
      • Donostia / San Sebastian, Gipuzkoa, Spain, 20014
        • Novartis Investigative Site
  • Taiwan
      • Hualien City, Taiwan, 970
        • Novartis Investigative Site
      • Taipei, Taiwan, 10002
        • Novartis Investigative Site
  • Turkey (Türkiye)
    • Fatih
      • Istanbul, Fatih, Turkey (Türkiye), 34093
        • Novartis Investigative Site
  • United Kingdom
      • London, United Kingdom, SE5 9RS
        • Novartis Investigative Site
    • West Yorkshire
      • Leeds, West Yorkshire, United Kingdom, LS9 7TF
        • Novartis Investigative Site
  • United States
    • California
      • Duarte, California, United States, 91010
        • City Of Hope
      • Duarte, California, United States, 91010
        • City Of Hope National Med Center
      • Los Angeles, California, United States, 90033
        • USC Norris Cancer Center
      • Orange, California, United States, 92868
        • Univ Cali Irvine ALS Neuromuscular
      • Orange, California, United States, 92868
        • Univ of California Irvine (Chao Family Comprehensive Cancer Center)
    • Florida
      • Miami Lakes, Florida, United States, 33014
        • Lakes Research
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Augusta University
      • Augusta, Georgia, United States, 30912
        • Augusta University Georgia
    • New York
      • The Bronx, New York, United States, 10461
        • Montefiore Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic Foundation
    • South Carolina
      • Greenville, South Carolina, United States, 29615
        • Prisma Health Upstate

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion criteria:

  • 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

Exclusion Criteria:

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Iptacopan
Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy
Drug: Iptacopan
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
Other Names:
  • LNP023

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Proportion of participants with adverse events
Time Frame: Up to 74 months
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
Up to 74 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Time Frame: Up to 74 months
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
Up to 74 months
Proportion of participants who remain free from transfusions
Time Frame: Up to 74 months
Proportion of participants who remain free from transfusions evaluated over yearly intervals
Up to 74 months
Rate of breakthrough hemolysis (BTH)
Time Frame: Up to 74 months
Rate of breakthrough hemolysis (BTH)
Up to 74 months
Proportion of participants with Major Adverse Vascular Events MAVEs
Time Frame: Up to 74 months
MAVEs (incl. thrombosis) evaluated over yearly intervals
Up to 74 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 27, 2021

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 18, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 18, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 5, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 9, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 10, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 7, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 6, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CLNP023C12001B
  • 2023-509843-28-00 (Other Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings