Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients who are naïve to complement inhibitor therapy.

Phase 1

• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH).; MedDRA version: 21.1Level: PTClassification code 10034042Term: Paroxysmal nocturnal haemoglobinuriaSystem Organ Class: 10038359 - Renal and urinary disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2020-003172-41-IT
• Lead Sponsor: OVARTIS PHARMA AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-17
• Last Update Posted: 24 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

- Male and female participants = 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with RBCs and white blood cells (WBCs) (granulocyte/monocyte) clone size = 10%.
- Mean hemoglobin level <10 g/dL confirmed by central laboratory assessment during Screening and prior to starting study treatment:
a. By two hemoglobin measurements (mean < 10 g/dL), two to eight weeks apart, for patients not receiving a RBC transfusion during Screening.
b. By one hemoglobin measurement (<10 g/dL) carried at the first Screening visit for patients receiving a RBC transfusion after which he/she will be eligible.
- LDH > 1.5 x Upper Limit of Normal (ULN) for at least two central laboratory measurements two to eight weeks apart during the screening period.
- Vaccination against Neisseria meningitidis infection is required prior to the start of treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local
regulations, at least 2 weeks prior to first dosing.
- If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given, if available and according to local regulations. The vaccines should be given at least 2 weeks prior to first dosing.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 36
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

Exclusion Criteria

- Prior treatment with a complement inhibitor, including anti-C5 antibody.
- Known or suspected hereditary complement deficiency at screening.
- History of hematopoietic stem cell transplantation.
- Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <500x106/L).
- Active systemic bacterial, viral (incl. COVID-19) 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., New York Heart Association (NYHA) class IV), severe pulmonary disease (e.g., severe
pulmonary hypertension (World Health Organization (WHO) class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified