Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
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
2 other identifiers
interventional
208
17 countries
60
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jul 2021
Longer than P75 for phase_3
60 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 5, 2021
CompletedFirst Posted
Study publicly available on registry
February 10, 2021
CompletedStudy Start
First participant enrolled
July 27, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 18, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 18, 2027
April 16, 2026
April 1, 2026
6.2 years
February 5, 2021
April 13, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Proportion of participants with adverse events
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 Outcomes (4)
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
60 months
Proportion of participants who remain free from transfusions
60 months
Rate of breakthrough hemolysis (BTH)
60 months
Proportion of participants with Major Adverse Vascular Events MAVEs
60 months
Study Arms (1)
Iptacopan
EXPERIMENTALParticipants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy
Interventions
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
Eligibility Criteria
You may qualify if:
- 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 may not qualify 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (60)
City Of Hope National Med Center
Duarte, California, 91010, United States
City Of Hope
Duarte, California, 91010, United States
USC Norris Cancer Center
Los Angeles, California, 90033, United States
Univ Cali Irvine ALS Neuromuscular
Orange, California, 92868, United States
Univ of California Irvine (Chao Family Comprehensive Cancer Center)
Orange, California, 92868, United States
Lakes Research
Miami Lakes, Florida, 33014, United States
Augusta University Georgia
Augusta, Georgia, 30912, United States
Augusta University
Augusta, Georgia, 30912, United States
Montefiore Medical Center
The Bronx, New York, 10461, United States
Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
Prisma Health Upstate
Greenville, South Carolina, 29615, United States
Novartis Investigative Site
Santo André, São Paulo, 09090-790, Brazil
Novartis Investigative Site
São Paulo, São Paulo, 01323-900, Brazil
Novartis Investigative Site
Beijing, 100730, China
Novartis Investigative Site
Tianjin, 300020, China
Novartis Investigative Site
Tianjin, 300052, China
Novartis Investigative Site
Ostrava, Poruba, 708 52, Czechia
Novartis Investigative Site
Brno, 625 00, Czechia
Novartis Investigative Site
Lille, 59037, France
Novartis Investigative Site
Nantes, 44093, France
Novartis Investigative Site
Nice, 06202, France
Novartis Investigative Site
Paris, 75475, France
Novartis Investigative Site
Toulouse, 31059, France
Novartis Investigative Site
Dresden, Saxony, 01307, Germany
Novartis Investigative Site
Aachen, 52074, Germany
Novartis Investigative Site
Essen, 45147, Germany
Novartis Investigative Site
Hamburg, 20246, Germany
Novartis Investigative Site
Riesa, 01589, Germany
Novartis Investigative Site
Ulm, 89081, Germany
Novartis Investigative Site
Ascoli Piceno, AP, 63100, Italy
Novartis Investigative Site
Avellino, AV, 83100, Italy
Novartis Investigative Site
Florence, FI, 50134, Italy
Novartis Investigative Site
Milan, MI, 20122, Italy
Novartis Investigative Site
Roma, RM, 00161, Italy
Novartis Investigative Site
Torino, TO, 10126, Italy
Novartis Investigative Site
Bassano del Grappa, VI, 36061, Italy
Novartis Investigative Site
Nagoya, Aichi-ken, 453-8511, Japan
Novartis Investigative Site
Kanazawa, Ishikawa-ken, 920 8641, Japan
Novartis Investigative Site
Isehara, Kanagawa, 259-1193, Japan
Novartis Investigative Site
Suwa, Nagano, 392-8510, Japan
Novartis Investigative Site
Suita, Osaka, 565 0871, Japan
Novartis Investigative Site
Shinjuku Ku, Tokyo, 160-0023, Japan
Novartis Investigative Site
Fukushima, 9601295, Japan
Novartis Investigative Site
Kyoto, 6068507, Japan
Novartis Investigative Site
Niigata, 9518520, Japan
Novartis Investigative Site
Vilnius, LT-08406, Lithuania
Novartis Investigative Site
Kota Kinabalu, Sabah, 88586, Malaysia
Novartis Investigative Site
Kuching, Sarawak, 93586, Malaysia
Novartis Investigative Site
Nijmegen, Gelderland, 6500HB, Netherlands
Novartis Investigative Site
Singapore, 119074, Singapore
Novartis Investigative Site
Seoul, Korea, 03080, South Korea
Novartis Investigative Site
Seoul, Seoul, 06351, South Korea
Novartis Investigative Site
Santiago Compostela, A Coruna, 15706, Spain
Novartis Investigative Site
Donostia / San Sebastian, Gipuzkoa, 20014, Spain
Novartis Investigative Site
Barcelona, 08036, Spain
Novartis Investigative Site
Hualien City, 970, Taiwan
Novartis Investigative Site
Taipei, 10002, Taiwan
Novartis Investigative Site
Istanbul, Fatih, 34093, Turkey (Türkiye)
Novartis Investigative Site
Leeds, West Yorkshire, LS9 7TF, United Kingdom
Novartis Investigative Site
London, SE5 9RS, United Kingdom
Related Publications (1)
DeCastro C, Sheinberg P, Han B, Vallow S, Bermann G, Dhalke M, Kumar R, Dickie G, Galipeau N, Lamoureux R, Rupinski K, Lowe C, Nieves A, de Fontbrune FS, de Latour RP. Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan. Patient. 2025 Nov;18(6):699-712. doi: 10.1007/s40271-025-00755-5. Epub 2025 Jul 22.
PMID: 40694294DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 5, 2021
First Posted
February 10, 2021
Study Start
July 27, 2021
Primary Completion (Estimated)
October 18, 2027
Study Completion (Estimated)
October 18, 2027
Last Updated
April 16, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
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