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

Summary

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

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Full Description

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.

View Eligibility Criteria

Eligibility Criteria

Inclusion Criteria:

Male and female participants ≥ 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%
Stable regimen of anti-C5 antibody treatment (either eculizumab or ravulizumab) for at least 6 months prior to randomization
Mean hemoglobin level <10 gdL
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

Exclusion Criteria:

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> 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

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

97

Study ID:

NCT04558918

Recruitment Status:

Active, not recruiting

Sponsor:

Novartis Pharmaceuticals

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There are 40 Locations for this study

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Novartis Investigative Site
Duarte California, 91010, United States
Novartis Investigative Site
Orange California, 92868, United States
Novartis Investigative Site
Augusta Georgia, 30912, United States
Novartis Investigative Site
Charlotte North Carolina, 28204, United States
Novartis Investigative Site
Cleveland Ohio, 44195, United States
Novartis Investigative Site
Santo Andre SP, 09090, Brazil
Novartis Investigative Site
Sao Paulo SP, 01323, Brazil
Novartis Investigative Site
Ostrava Poruba, 708 5, Czechia
Novartis Investigative Site
Lille , 59037, France
Novartis Investigative Site
Paris Cedex 10 , 75475, France
Novartis Investigative Site
Toulouse , 31059, France
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
Firenze FI, 50139, Italy
Novartis Investigative Site
Milano 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, 453-8, Japan
Novartis Investigative Site
Fukushima city Fukushima, 960 1, Japan
Novartis Investigative Site
Kanazawa-city Ishikawa, 920-8, Japan
Novartis Investigative Site
Suwa Nagano, 392-8, Japan
Novartis Investigative Site
Osaka Sayama Osaka, 589 8, Japan
Novartis Investigative Site
Suita Osaka, 565 0, Japan
Novartis Investigative Site
Shinjuku-ku Tokyo, 160-0, Japan
Novartis Investigative Site
Kyoto , 606 8, Japan
Novartis Investigative Site
Seoul , 06351, Korea, Republic of
Novartis Investigative Site
Nijmegen , 6500 , Netherlands
Novartis Investigative Site
Barcelona Catalunya, 08036, Spain
Novartis Investigative Site
Santiago de Compostela Galicia, 15706, Spain
Novartis Investigative Site
San Sebastian Pais Vasco, 20080, Spain
Novartis Investigative Site
Hualien , 970, Taiwan
Novartis Investigative Site
Taipei , 10002, Taiwan
Novartis Investigative Site
Leeds , LS9 7, United Kingdom
Novartis Investigative Site
London , SE5 9, United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

97

Study ID:

NCT04558918

Recruitment Status:

Active, not recruiting

Sponsor:


Novartis Pharmaceuticals

How clear is this clinincal trial information?

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