Last Update: Aug 07, 2026 An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to ClinicalTrials.gov Identifier: [NCT06934967](https://clinicaltrials.gov/ct2/show/NCT06934967) Novartis Reference Number:CLNP023I12201 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") 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 open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to \\< 18 years of age. This is a multicenter, open-label, single arm study comprised of an up to a 8-week Screening Period, and a 26-week Treatment Period followed by a 26-week Extension Treatment Period. This study will enroll a minimum of 12 pediatric patients 2 to \\< 18 years of age in a staggered manner into 3 cohorts: Cohort 1 (adolescents 12 to \\< 18 years of age, approximately 6 patients), Cohort 2a (6 to \\< 12 years of age, approximately 4 patients), and Cohort 2b (2 to \\< 6 years of age, approximately 2 patients). Condition Paroxysmal Nocturnal Hemoglobinuria (PNH) Phase Phase3 Overall Status Recruiting Number of Participants12 Start Date Oct 28, 2025 Completion Date Nov 13, 2031 Gender All Age(s) 2 Years - 18 Years (Child, Adult) ## Interventions Drug ### LNP023 Cohort 1-administered orally a dosing scheme of 200 mg twice-daily (two 100 mg capsules). Cohort 2- administered orally a dosing scheme based on weight at the Day 1, Week 12, 26 and 38. ## Eligibility Criteria Inclusion Criteria: \* Male and female participants 2 to \\< 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes/monocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg. \* Patients being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator. \* Patients who are anti-C5 treatment naive: mean hemoglobin level \\< 10 g/dL confirmed by central laboratory assessment during screening. \* Patients who are anti-C5 treatment naive: lactate dehydrogenase (LDH) \\> 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab. \* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated. \* Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan. Exclusion Criteria: \* History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes. \* Known or suspected hereditary complement deficiency at screening. \* History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1). \* Patients with laboratory evidence of bone marrow failure (reticulocytes \\< 100 x 10 to the ninth/L; platelets \\< 30 × 10 to the ninth/L; neutrophils \\< 0.5 × 10 to the ninth/L). \* Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration. \* Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration. Other protocol-defined inclusion/exclusion criteria may apply. Brazil #### Novartis Investigative Site Recruiting Natal,Rio Grande do Norte,59012 300,Brazil #### Novartis Investigative Site Recruiting Santo André,São Paulo,09090-401,Brazil #### Novartis Investigative Site Recruiting São Paulo,São Paulo,04038-002,Brazil #### Novartis Investigative Site Recruiting Brasília,Federal District,70684-831,Brazil #### Novartis Investigative Site Recruiting São Paulo,São Paulo,01323001,Brazil #### Novartis Investigative Site Recruiting Porto Alegre,Rio Grande do Sul,90035-003,Brazil Colombia #### Novartis Investigative Site Recruiting Cali,Valle del Cauca Department,760012,Colombia Germany #### Novartis Investigative Site Recruiting Cologne,North Rhine-Westphalia,50937,Germany #### Novartis Investigative Site Recruiting Berlin,13353,Germany Italy #### Novartis Investigative Site Recruiting Genova,GE,16147,Italy Netherlands #### Novartis Investigative Site Recruiting Utrecht,3584 cx,Netherlands United States #### Cancer Institute of New Jersey Recruiting New Brunswick,New Jersey,08901,United States ###### Richard DRACHTMAN ###### Serban Morojanu Phone: [+1 732 235 7577]() Email: #### St Jude Childrens Research Hospital Recruiting Memphis,Tennessee,38105,United States ###### Marcin Wlodarski ###### Michelle Brignac Email: #### Childrens Healthcare of Atlanta Recruiting Atlanta,Georgia,30342,United States ###### Robert Uddo Email: ###### Satheesh Chonat #### Childrens Hospital of Philadelphia Recruiting Philadelphia,Pennsylvania,19104-4399,United States ###### Peter Nicholas Phone: [+1 267 425 2136]() Email: ###### Timothy Olson ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: [](mailto:) #### Novartis Pharmaceuticals Phone: [ 1-888-669-6682](tel:1-888-669-6682) Email: