Myelodysplastic Syndrome Clinical Trial
Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
The purpose of this Phase 3 study is to determine whether LNP023 is efficacious and safe for the treatment in PNH through demonstration of superiority of LNP023 compared to anti-C5 antibody treatment in adult PNH patients presenting with residual anemia despite treatment with anti-C5 therapy
The purpose of this Phase 3 randomized, multicenter, active-comparator controlled, open-label trial is to determine whether LNP023 is efficacious and safe for the treatment in PNH through demonstration of superiority of LNP023 compared to anti-C5 antibody treatment in adult PNH patients presenting with residual anemia despite treatment with anti-C5 therapy.
The study is planned to randomize approx. 91 patients in various countries.
Male and female participants â‰¥ 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size â‰¥ 10%
Stable regimen of anti-C5 antibody treatment (either eculizumab or ravulizumab) for at least 6 months prior to randomization
Mean hemoglobin level <10 g/dL
Vaccination against Neisseria meningitidis infection is required prior to the start of treatment.
If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given
Participants on a stable eculizumab dose but with a dosing interval of 11 days or less
Known or suspected hereditary complement deficiency at screening
History of hematopoietic stem cell transplantation
Patients with laboratory evidence of bone marrow failure (reticulocytes <100x10E9/L; platelets <30x10E9/L; neutrophils <500x10E6/L).
Active systemic bacterial, viral or fungal infection within 14 days prior to study drug administration
A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
Major concurrent comorbidities including but not limited to severe kidney disease (e.g., dialysis), advanced cardiac disease (e.g., NYHA class IV), severe pulmonary disease (e.g., severe pulmonary) hypertension (WHO class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.
Other protocol-defined inclusion/exclusion criteria may apply
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There are 39 Locations for this study
Duarte California, 91010, United States
Orange California, 92868, United States
Augusta Georgia, 30912, United States
Charlotte North Carolina, 28204, United States
Cleveland Ohio, 44195, United States
Santo Andre SP, 09090, Brazil
Sao Paulo SP, 01323, Brazil
Ostrava Poruba, 708 5, Czechia
Lille , 59037, France
Paris Cedex 10 , 75475, France
Toulouse , 31059, France
Aachen , 52074, Germany
Essen , 45147, Germany
Hamburg , 20246, Germany
Riesa , 01589, Germany
Ulm , 89081, Germany
Ascoli Piceno AP, 63100, Italy
Avellino AV, 83100, Italy
Firenze FI, 50139, Italy
Milano MI, 20122, Italy
Roma RM, 00161, Italy
Torino TO, 10126, Italy
Bassano Del Grappa VI, 36061, Italy
Nagoya Aichi, 453-8, Japan
Fukushima city Fukushima, 960 1, Japan
Kanazawa-city Ishikawa, 920-8, Japan
Suwa Nagano, 392-8, Japan
Suita Osaka, 565 0, Japan
Shinjuku-ku Tokyo, 160-0, Japan
Kyoto , 606 8, Japan
Seoul , 06351, Korea, Republic of
Nijmegen , 6500 , Netherlands
Barcelona Catalunya, 08036, Spain
Santiago de Compostela Galicia, 15706, Spain
San Sebastian Pais Vasco, 20080, Spain
Hualien , 970, Taiwan
Taipei , 10002, Taiwan
Leeds , LS9 7, United Kingdom
London , SE5 9, United Kingdom
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