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

An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age

Phase 3 Interventional Novartis · NCT06934967

This trial will try oral iptacopan in children and teens (ages 2 to under 18) with PNH to see how their bodies handle the drug and whether it is safe and well tolerated.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment12 (estimated)
Ages2 Years to 18 Years
SexAll
SponsorNovartis Industry-sponsored
Locations13 sites (New Brunswick, New Jersey and 12 other locations)
Trial IDNCT06934967 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Children and adolescents ages 2 to under 18 with confirmed PNH by high-sensitivity flow cytometry (RBC and WBC/granulocyte/monocyte clone ≥ 10%), meeting cohort weight requirements, and either stable on anti‑C5 therapy or treatment‑naive with low hemoglobin and elevated LDH are eligible.

Not a fit: Patients without a confirmed PNH clone ≥10%, those with other causes of anemia or serious uncontrolled medical conditions, or those unable to travel to the U.S. study sites are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, iptacopan could provide an effective oral option to control hemolysis and anemia in pediatric PNH patients and reduce the need for regular intravenous complement C5 infusions.

How similar studies have performed: Oral iptacopan and related complement inhibitors have produced promising efficacy and biomarker improvements in adults with PNH, but pediatric data remain limited so this trial extends adult findings to children.

Eligibility criteria

Show full inclusion / exclusion 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.

Where this trial is running

New Brunswick, New Jersey and 12 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Paroxysmal Nocturnal HemoglobinuriaIptacopan,PNH,hemoglobin,anemia
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.