Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody.

ClinicalTrials.gov Identifier: NCT04558918

Novartis Reference Number: CLNP023C12302

Last Update: Oct 28, 2021

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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

Condition 
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 
Phase 3
Overall status 
Recruiting
Enrollment count 
91 participants
Start date 
Jan 25, 2021
Completion date 
Jan 23, 2023
Gender 
All
Age(s)
18 Years and older (Adult, Older Adult)

Interventions

Drug
LNP023
Taken Orally b.i.d. Dosage Supplied: 200 mg Dosage form: Hard gelatin capsule Route of Administration: Oral
Drug
Eculizumab
Administered as intravenous infusion every 2 weeks as per the stable regimen, the maintenance dose is a fixed dose. Dosage Supplied: 300 mg/30mL Dosage form: Concentrate solution for infusion
Drug
Ravulizumab
Administered as intravenous infusion every 8 weeks, the maintenance dose is based on body weight. Dosage Supplied: 300 mg/30mL Dosage form: Concentrate solution for infusion

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 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

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/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

Study Locations

United States
Novartis Investigative Site
Recruiting
Duarte, 91010
California
United States
Novartis Investigative Site
Recruiting
Orange, 92868
California
United States
Novartis Investigative Site
Recruiting
Miami Lakes, 33014
Florida
United States
Novartis Investigative Site
Recruiting
Augusta, 30912
Georgia
United States
Novartis Investigative Site
Recruiting
Boston, 02115
Massachusetts
United States
Novartis Investigative Site
Recruiting
Cleveland, 44195
Ohio
United States
Brazil
Novartis Investigative Site
Recruiting
Porto Alegre, 90050-170
Rio Grande Do Sul
Brazil
Novartis Investigative Site
Recruiting
Rio de Janeiro, 22640-000
RJ
Brazil
Novartis Investigative Site
Recruiting
Ribeirao Preto, 14048-900
SP
Brazil
Novartis Investigative Site
Recruiting
Santo Andre, 09090-790
SP
Brazil
Novartis Investigative Site
Recruiting
São Paulo, 01323-900
SP
Brazil
Czech Republic
Novartis Investigative Site
Recruiting
Ostrava Poruba, 708 52
Czech Republic
Czech Republic
France
Novartis Investigative Site
Recruiting
Clermont Ferrand, 63003
-
France
Novartis Investigative Site
Recruiting
Lille, 59037
-
France
Novartis Investigative Site
Recruiting
Paris Cedex 10, 75475
-
France
Novartis Investigative Site
Recruiting
Pierre Benite Cedex, 69495
-
France
Novartis Investigative Site
Recruiting
Toulouse, 31059
-
France
Novartis Investigative Site
Recruiting
Vandoeuvre les Nancy, 54511
-
France
Germany
Novartis Investigative Site
Recruiting
Aachen, 52074
-
Germany
Novartis Investigative Site
Recruiting
Essen, 45147
-
Germany
Novartis Investigative Site
Recruiting
Hamburg, 20246
-
Germany
Novartis Investigative Site
Recruiting
Riesa, 01589
-
Germany
Novartis Investigative Site
Recruiting
Ulm, 89081
-
Germany
Hungary
Novartis Investigative Site
Recruiting
Budapest, 1088
-
Hungary
Italy
Novartis Investigative Site
Recruiting
Ascoli Piceno, 63100
AP
Italy
Novartis Investigative Site
Recruiting
Avellino, 83100
AV
Italy
Novartis Investigative Site
Recruiting
Firenze, 50139
FI
Italy
Novartis Investigative Site
Recruiting
Milano, 20122
MI
Italy
Novartis Investigative Site
Recruiting
Palermo, 90146
PA
Italy
Novartis Investigative Site
Recruiting
Bassano Del Grappa, 36061
Vicenza
Italy
Japan
Novartis Investigative Site
Recruiting
Nagoya, 453-8511
Aichi
Japan
Novartis Investigative Site
Recruiting
Fukushima city, 960 1295
Fukushima
Japan
Novartis Investigative Site
Recruiting
Kanazawa-city, 920-8641
Ishikawa
Japan
Novartis Investigative Site
Recruiting
Isehara, 259-1193
Kanagawa
Japan
Novartis Investigative Site
Recruiting
Suwa, 392-8510
Nagano
Japan
Novartis Investigative Site
Recruiting
Osaka Sayama, 589 8511
Osaka
Japan
Novartis Investigative Site
Recruiting
Suita city, 565 0871
Osaka
Japan
Novartis Investigative Site
Recruiting
Shinagawa ku, 141 8625
Tokyo
Japan
Novartis Investigative Site
Recruiting
Shinjuku-ku, 160-0023
Tokyo
Japan
Novartis Investigative Site
Recruiting
Kyoto, 606 8507
-
Japan
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 03080
-
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 06351
-
Korea, Republic of
Netherlands
Novartis Investigative Site
Recruiting
Nijmegen, 6500 MB
-
Netherlands
Spain
Novartis Investigative Site
Recruiting
Sevilla, 41013
Andalucia
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08036
Catalunya
Spain
Novartis Investigative Site
Recruiting
Santiago de Compostela, 15706
Galicia
Spain
Novartis Investigative Site
Recruiting
San Sebastian, 20080
Pais Vasco
Spain
Novartis Investigative Site
Recruiting
Madrid, 28040
-
Spain
Taiwan
Novartis Investigative Site
Recruiting
Hualien, 970
-
Taiwan
Novartis Investigative Site
Recruiting
Taipei, 10002
-
Taiwan
United Kingdom
Novartis Investigative Site
Recruiting
London, SE5 9RS
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
Name: 
Novartis Pharmaceuticals
Phone: 

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