Study of Efficacy and Safety of Twice Daily Oral iptacopan 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] - Immune System Diseases [C20]

• Registration Number: EUCTR2020-003172-41-CZ
• Lead Sponsor: ovartis Pharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-10
• Last Update Posted: 21 August 2023

Recruitment & Eligibility

• Status: ot Recruiting
• 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 WBCs clone size = 10%
- Mean hemoglobin level <10 g/dL • LDH > 1.5 x Upper Limit of Normal (ULN)
- Vaccination against Neisseria meningitidis infection is required, and is
recommended at least 2 weeks prior to initiation of iptacopan treatment.However, administration of these vaccines less than 2 weeks prior to start of iptacopan treatment or up to 2 weeks (up to Day 14) after iptacopan initiation, is at the discretion of the investigator. If iptacopan treatment is started less than 2 weeks post-vaccination or before a specific vaccination is given, participant must be given prophylactic antibiotic at the start of iptacopan and for at least 2 weeks after vaccination.
-If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae should be given (if available
and according to local/national regulations), and are recommended at
least 2 weeks prior to initiation of iptacopan treatment. However,
administration of these vaccines less than 2 weeks prior to start of
iptacopan treatment or up to 2 weeks (up to Day 14) after iptacopan
initiation, is at the discretion of the investigator. If iptacopan treatment
is started less than 2 weeks post-vaccination or before a specific
vaccination is given, participant must be given prophylactic antibiotic at
the start of iptacopan and for at least 2 weeks after vaccination.

Other protocol defined inclusion/exclusion criteria may apply.
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
- History of hematopoietic stem cell transplantation
- Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L).
- Active systemic bacterial, viral (incl. COVID-19)or fungal infection within 14 days prior to study drug administration
- 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 heart failure), 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 & Design

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