Iptacopan for children and teens with PNH to see how the drug behaves in the body and whether it is safe

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is an open-label, single-arm, multicenter Phase 3 trial that enrolls a minimum of 12 pediatric patients in three staggered age cohorts (12 to <18, 6 to <12, 2 to <6 years). After up to an 8-week screening period, participants receive 26 weeks of treatment with iptacopan followed by a 26-week extension phase, with pharmacokinetic sampling and safety monitoring throughout. The study includes patients who are stable on anti‑C5 therapy and those who are treatment‑naive with anemia and elevated LDH, and it focuses on drug exposure, tolerability, and safety endpoints. Sites include major U.S. pediatric centers and dosing and follow-up visits are scheduled at those centers.

Why it matters

Who can join: the full rules

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.

Where it's running

• Childrens Healthcare of Atlanta
  Atlanta, Georgia, United States Recruiting
• Cancer Institute of New Jersey
  New Brunswick, New Jersey, United States Recruiting
• Childrens Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting
• St Jude Childrens Research Hospital
  Memphis, Tennessee, United States Recruiting
• Novartis Investigative Site
  Brasília, Federal District, Brazil Recruiting
• Novartis Investigative Site
  Natal, Rio Grande do Norte, Brazil Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.