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

March 26, 2024 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

Recruiting

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Estimated)

250

Phase

  • 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 Backup

  • Name: Novartis Pharmaceuticals
  • Phone Number: +41613241111

Study Locations

  • Brazil
    • SP
      • Santo Andre, SP, Brazil, 09090-790
        • Active, not recruiting
        • Novartis Investigative Site
      • Sao Paulo, SP, Brazil, 01323-900
        • Active, not recruiting
        • Novartis Investigative Site
  • China
      • Beijing, China, 100730
        • Active, not recruiting
        • Novartis Investigative Site
      • Tianjin, China, 300052
        • Active, not recruiting
        • Novartis Investigative Site
      • Tianjin, China, 300020
        • Active, not recruiting
        • Novartis Investigative Site
  • Czechia
      • Brno - Bohunice, Czechia, 625 00
        • Active, not recruiting
        • Novartis Investigative Site
    • Poruba
      • Ostrava, Poruba, Czechia, 708 52
        • Active, not recruiting
        • Novartis Investigative Site
  • France
      • Lille Cedex, France, 59 037
        • Recruiting
        • Novartis Investigative Site
      • Paris 10, France, 75475
        • Recruiting
        • Novartis Investigative Site
      • Toulouse, France, 31059
        • Recruiting
        • Novartis Investigative Site
  • Germany
      • Aachen, Germany, 52074
        • Recruiting
        • Novartis Investigative Site
      • Essen, Germany, 45147
        • Recruiting
        • Novartis Investigative Site
      • Hamburg, Germany, 20246
        • Active, not recruiting
        • Novartis Investigative Site
      • Riesa, Germany, 01589
        • Active, not recruiting
        • Novartis Investigative Site
      • Ulm, Germany, 89081
        • Recruiting
        • Novartis Investigative Site
  • Italy
    • AP
      • Ascoli Piceno, AP, Italy, 63100
        • Recruiting
        • Novartis Investigative Site
    • AV
      • Avellino, AV, Italy, 83100
        • Recruiting
        • Novartis Investigative Site
    • FI
      • Firenze, FI, Italy, 50134
        • Recruiting
        • Novartis Investigative Site
    • MI
      • Milano, MI, Italy, 20122
        • Recruiting
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italy, 00161
        • Recruiting
        • Novartis Investigative Site
    • TO
      • Torino, TO, Italy, 10126
        • Completed
        • Novartis Investigative Site
    • VI
      • Bassano Del Grappa, VI, Italy, 36061
        • Recruiting
        • Novartis Investigative Site
  • Japan
      • Kyoto, Japan, 606 8507
        • Active, not recruiting
        • Novartis Investigative Site
      • Niigata, Japan, 951 8520
        • Active, not recruiting
        • Novartis Investigative Site
    • Aichi
      • Nagoya, Aichi, Japan, 453-8511
        • Active, not recruiting
        • Novartis Investigative Site
    • Fukushima
      • Fukushima city, Fukushima, Japan, 960 1295
        • Active, not recruiting
        • Novartis Investigative Site
    • Ishikawa
      • Kanazawa-city, Ishikawa, Japan, 920-8641
        • Active, not recruiting
        • Novartis Investigative Site
    • Kanagawa
      • Isehara, Kanagawa, Japan, 259-1193
        • Active, not recruiting
        • Novartis Investigative Site
    • Nagano
      • Suwa, Nagano, Japan, 392-8510
        • Active, not recruiting
        • Novartis Investigative Site
    • Osaka
      • Suita, Osaka, Japan, 565 0871
        • Active, not recruiting
        • Novartis Investigative Site
    • Tokyo
      • Shinjuku-ku, Tokyo, Japan, 160-0023
        • Active, not recruiting
        • Novartis Investigative Site
  • Korea, Republic of
      • Seoul, Korea, Republic of, 03080
        • Recruiting
        • Novartis Investigative Site
      • Seoul, Korea, Republic of, 06351
        • Recruiting
        • Novartis Investigative Site
  • Lithuania
      • Vilnius, Lithuania, LT-08661
        • Recruiting
        • Novartis Investigative Site
  • Malaysia
    • Sabah
      • Kota Kinabalu, Sabah, Malaysia, 88586
        • Recruiting
        • Novartis Investigative Site
    • Sarawak
      • Kuching, Sarawak, Malaysia, 93586
        • Recruiting
        • Novartis Investigative Site
  • Netherlands
      • Nijmegen, Netherlands, 6500 MB
        • Active, not recruiting
        • Novartis Investigative Site
  • Singapore
      • Singapore, Singapore, 119228
        • Recruiting
        • Novartis Investigative Site
  • Spain
    • Catalunya
      • Barcelona, Catalunya, Spain, 08036
        • Active, not recruiting
        • Novartis Investigative Site
    • Galicia
      • Santiago De Compostela, Galicia, Spain, 15706
        • Active, not recruiting
        • Novartis Investigative Site
    • Pais Vasco
      • San Sebastian, Pais Vasco, Spain, 20080
        • Active, not recruiting
        • Novartis Investigative Site
  • Taiwan
      • Hualien, Taiwan, 970
        • Recruiting
        • Novartis Investigative Site
      • Taipei, Taiwan, 10002
        • Recruiting
        • Novartis Investigative Site
  • Turkey
      • Istanbul, Turkey, 34093
        • Recruiting
        • Novartis Investigative Site
  • United Kingdom
      • Leeds, United Kingdom, LS9 7TF
        • Recruiting
        • Novartis Investigative Site
      • London, United Kingdom, SE5 9RS
        • Recruiting
        • Novartis Investigative Site
  • United States
    • California
      • Duarte, California, United States, 91010
        • Recruiting
        • City of Hope
        • Principal Investigator:
          • Vinod Pullarkat
        • Contact:
          • Phone Number: 626-218-0247
      • Los Angeles, California, United States, 90033
        • Recruiting
        • USC Norris Cancer Center
        • Contact:
        • Principal Investigator:
          • Ilene Weitz
      • Orange, California, United States, 92868
        • Recruiting
        • Univ of California Irvine (Chao Family Comprehensive Cancer Center)
        • Contact:
          • Phone Number: 714-509-6558
        • Principal Investigator:
          • Zahra Pakbaz
    • Florida
      • Miami Lakes, Florida, United States, 33014
        • Recruiting
        • Lakes Research
        • Principal Investigator:
          • Eloy Roman
        • Contact:
          • Phone Number: 786-362-5763
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Recruiting
        • Augusta University
        • Principal Investigator:
          • Abdullah Kutlar
        • Contact:
    • New York
      • Bronx, New York, United States, 10461
        • Recruiting
        • Montefiore Medical Center .
        • Principal Investigator:
          • Irina Murakhovskaya
        • Contact:
          • Phone Number: 718-920-2680
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic Foundation
        • Contact:
          • Phone Number: 216-444-0441
        • Principal Investigator:
          • Jaroslaw Maciejewski

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

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
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 Names:
  • LNP023

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
60 months

Secondary Outcome Measures

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: 60 months
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
60 months
Proportion of participants who remain free from transfusions
Time Frame: 60 months
Proportion of participants who remain free from transfusions evaluated over yearly intervals
60 months
Rate of breakthrough hemolysis (BTH)
Time Frame: 60 months
Rate of breakthrough hemolysis (BTH)
60 months
Proportion of participants with Major Adverse Vascular Events MAVEs
Time Frame: 60 months
MAVEs (incl. thrombosis) evaluated over yearly intervals
60 months

Collaborators and Investigators

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

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

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)

July 27, 2021

Primary Completion (Estimated)

January 10, 2028

Study Completion (Estimated)

February 7, 2028

Study Registration Dates

First Submitted

February 5, 2021

First Submitted That Met QC Criteria

February 9, 2021

First Posted (Actual)

February 10, 2021

Study Record Updates

Last Update Posted (Actual)

March 27, 2024

Last Update Submitted That Met QC Criteria

March 26, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLNP023C12001B

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.

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