Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients

Recruiting now · Phase 3

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria (PNH)

In brief

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.

Key facts

Study ID

NCT06934967

Run by

Novartis Pharmaceuticals

People needed

12

Starts

2025-10-28

Expected to finish

2031-11-13

Last updated by the study team

2026-08-07

Who can join

Age: 2 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Childrens Healthcare of Atlanta — Atlanta, Georgia, United States (enrolling)
• Cancer Institute of New Jersey — New Brunswick, New Jersey, United States (enrolling)
• Childrens Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• St Jude Childrens Research Hospital — Memphis, Tennessee, United States (enrolling)
• Novartis Investigative Site — Brasília, Federal District, Brazil (enrolling)
• Novartis Investigative Site — Natal, Rio Grande do Norte, Brazil (enrolling)
• Novartis Investigative Site — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)
• Novartis Investigative Site — Santo André, São Paulo, Brazil (enrolling)
• Novartis Investigative Site — São Paulo, São Paulo, Brazil (enrolling)
• Novartis Investigative Site — São Paulo, São Paulo, Brazil (enrolling)
• Novartis Investigative Site — Cali, Valle del Cauca Department, Colombia (enrolling)
• Novartis Investigative Site — Cologne, North Rhine-Westphalia, Germany (enrolling)
• Novartis Investigative Site — Berlin, Germany (enrolling)
• Novartis Investigative Site — Genova, GE, Italy (enrolling)
• Novartis Investigative Site — Utrecht, Netherlands (enrolling)

Full record on ClinicalTrials.gov

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