Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT ID: NCT04747613
Last Updated: 2025-03-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
208 participants
INTERVENTIONAL
2021-07-27
2027-10-19
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Iptacopan
Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy
Iptacopan
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
Interventions
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Iptacopan
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
* History of hematopoietic stem cell transplantation
18 Years
100 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Novartis Pharmaceuticals
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
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City Of Hope
Duarte, California, United States
USC Norris Cancer Center
Los Angeles, California, United States
Univ of California Irvine (Chao Family Comprehensive Cancer Center)
Orange, California, United States
Lakes Research
Miami Lakes, Florida, United States
Augusta University
Augusta, Georgia, United States
Montefiore Medical Center
The Bronx, New York, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
Prisma Health Upstate
Greenville, South Carolina, United States
Novartis Investigative Site
Santo André, São Paulo, Brazil
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São Paulo, São Paulo, Brazil
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Beijing, , China
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Tianjin, , China
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Tianjin, , China
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Ostrava, Poruba, Czechia
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Brno Bohunice, , Czechia
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Lille, , France
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Nantes, , France
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Nice, , France
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Paris, , France
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Toulouse, , France
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Aachen, , Germany
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Dresden, , Germany
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Essen, , Germany
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Hamburg, , Germany
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Riesa, , Germany
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Ulm, , Germany
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Ascoli Piceno, AP, Italy
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Avellino, AV, Italy
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Florence, FI, Italy
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Milan, MI, Italy
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Roma, RM, Italy
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Torino, TO, Italy
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Bassano del Grappa, Vicenza, Italy
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Nagoya, Aichi-ken, Japan
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Fukushima, Fukushima, Japan
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Kanazawa, Ishikawa-ken, Japan
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Isehara, Kanagawa, Japan
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Suwa, Nagano, Japan
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Suita, Osaka, Japan
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Shinjuku Ku, Tokyo, Japan
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Kyoto, , Japan
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Niigata, , Japan
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Vilnius, , Lithuania
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Kota Kinabalu, Sabah, Malaysia
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Kuching, Sarawak, Malaysia
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Nijmegen, , Netherlands
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Singapore, , Singapore
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Seoul, , South Korea
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Seoul, , South Korea
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Donostia / San Sebastian, Basque Country, Spain
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Barcelona, Catalonia, Spain
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Santiago de Compostela, Galicia, Spain
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Hualien City, , Taiwan
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Taipei, , Taiwan
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Istanbul, , Turkey (Türkiye)
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Leeds, West Yorkshire, United Kingdom
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London, , United Kingdom
Countries
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References
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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 Jul 22. doi: 10.1007/s40271-025-00755-5. Online ahead of print.
Other Identifiers
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CLNP023C12001B
Identifier Type: -
Identifier Source: org_study_id
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