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

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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 
Not yet recruiting
Enrollment count 
91 participants
Start date 
Dec 15, 2020
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

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
1-888-669-6682
Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Email: 

Have a question?

Call 1-999-669-6682 or email [email protected]