We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04747613
Recruitment Status : Recruiting
First Posted : February 10, 2021
Last Update Posted : August 1, 2022
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
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

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: Iptacopan Phase 3

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 167 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : July 27, 2021
Estimated Primary Completion Date : May 29, 2026
Estimated Study Completion Date : June 26, 2026

Arm Intervention/treatment
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 Name: LNP023

Primary Outcome Measures :
  1. Proportion of participants with adverse events [ Time Frame: 60 months ]
    Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit

Secondary Outcome Measures :
  1. Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions [ Time Frame: 60 months ]
    Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals

  2. Proportion of participants who remain free from transfusions [ Time Frame: 60 months ]
    Proportion of participants who remain free from transfusions evaluated over yearly intervals

  3. Rate of breakthrough hemolysis (BTH) [ Time Frame: 60 months ]
    Rate of breakthrough hemolysis (BTH)

  4. Proportion of participants with Major Adverse Vascular Events MAVEs [ Time Frame: 60 months ]
    MAVEs (incl. thrombosis) evaluated over yearly intervals

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04747613

Layout table for location contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Layout table for location information
Novartis Investigative Site Recruiting
Brno - Bohunice, Czechia, 625 00
Novartis Investigative Site Recruiting
Lille Cedex, France, 59 037
Novartis Investigative Site Recruiting
Paris Cedex 10, France, 75475
Novartis Investigative Site Recruiting
Toulouse, France, 31059
Novartis Investigative Site Recruiting
Aachen, Germany, 52074
Novartis Investigative Site Recruiting
Essen, Germany, 45147
Novartis Investigative Site Recruiting
Hamburg, Germany, 20246
Novartis Investigative Site Recruiting
Riesa, Germany, 01589
Novartis Investigative Site Recruiting
Ascoli Piceno, AP, Italy, 63100
Novartis Investigative Site Recruiting
Avellino, AV, Italy, 83100
Novartis Investigative Site Recruiting
Firenze, FI, Italy, 50134
Novartis Investigative Site Recruiting
Milano, MI, Italy, 20122
Novartis Investigative Site Recruiting
Bassano Del Grappa, VI, Italy, 36061
Novartis Investigative Site Recruiting
Nagoya, Aichi, Japan, 453-8511
Novartis Investigative Site Recruiting
Fukushima city, Fukushima, Japan, 960 1295
Novartis Investigative Site Recruiting
Kanazawa-city, Ishikawa, Japan, 920-8641
Novartis Investigative Site Recruiting
Isehara, Kanagawa, Japan, 259-1193
Novartis Investigative Site Recruiting
Suwa, Nagano, Japan, 392-8510
Novartis Investigative Site Recruiting
Suita city, Osaka, Japan, 565 0871
Novartis Investigative Site Recruiting
Shinjuku-ku, Tokyo, Japan, 160-0023
Novartis Investigative Site Recruiting
Niigata, Japan, 951 8520
Korea, Republic of
Novartis Investigative Site Recruiting
Seoul, Korea, Republic of, 03080
Novartis Investigative Site Recruiting
Seoul, Korea, Republic of, 06351
Novartis Investigative Site Recruiting
Vilnius, Lithuania, LT-08661
Novartis Investigative Site Recruiting
Kota Kinabalu, Sabah, Malaysia, 88586
Novartis Investigative Site Recruiting
Nijmegen, Netherlands, 6500 MB
Novartis Investigative Site Recruiting
Singapore, Singapore, 119228
Novartis Investigative Site Recruiting
Barcelona, Catalunya, Spain, 08036
Novartis Investigative Site Recruiting
Hualien, Taiwan, 970
Novartis Investigative Site Recruiting
Taipei, Taiwan, 10002
United Kingdom
Novartis Investigative Site Recruiting
London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Novartis Pharmaceuticals
Layout table for additonal information
Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04747613    
Other Study ID Numbers: CLNP023C12001B
First Posted: February 10, 2021    Key Record Dates
Last Update Posted: August 1, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Additional relevant MeSH terms:
Layout table for MeSH terms
Hemoglobinuria, Paroxysmal
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases