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

• Register: JPRN
• Last refreshed on: 1 July 2024
• Main ID: JPRN-jRCT2051200132
• Date of registration: 09/02/2021
• Prospective Registration: Yes
• Primary sponsor: Suzuki Kazuyuki
• Public title: Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
• Scientific title: Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment - APPLY-PNH
• Date of first enrolment: 26/03/2021
• Target sample size: 91
• Recruitment status: Complete
• URL: https://jrct.niph.go.jp/latest-detail/jRCT2051200132
• Study type: Interventional
• Study design: randomized controlled trial, open(masking not used), active control, parallel assignment, treatment purpose
• Phase: 3

Countries of recruitment

Japan, Netherlands

Contacts

• Name: Kazuyuki Suzuki
• Affiliation: Novartis Pharma. K.K.

• Name: Kazuyuki Suzuki
• Affiliation: Novartis Pharma. K.K.

Key inclusion & exclusion 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.

Age minimum: >= 18age old
Age maximum: Not applicable
Gender: Both

Health Condition(s) or Problem(s) studied

Paroxysmal nocturnal hemoglobinuria
PNH

Intervention(s)

Arm A : LNP023 monotherapy
Arm B : Anti-C5 antibody treatment(Eculizumab or Ravulizumab)

Primary Outcome(s)

- Percentage of participants achieving a sustained increase in hemoglobin levels of >= 2 g/dL in the absence of red blood cell transfusions
- Percentage of participants achieving sustained hemoglobin levels >= 12 g/dL in the absence of red blood cell transfusions

Secondary ID(s)

NCT04558918

Ethics review

Status: Approval
Approval date:
Contact:

[e-mail]

Institutional review board of Osaka University Hospital

[phone]

[e-mail]

Results

• Results available: Yes
• Date Completed: 06/03/2023