ClinicalTrials.gov record NCT06934967

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age2 years old to 18 years old
SexAll
LocationBrazil, Colombia, Germany, Italy, Netherlands, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an open‑label, single‑arm Phase 3 study that will enroll children aged 2 to <18 years with PNH to study iptacopan (LNP023). The study includes up to an 8‑week screening period, a 26‑week treatment period, and a 26‑week extension. Patients are enrolled in age‑based cohorts with dosing set by age or weight.

What it is studying. The study measures how iptacopan is processed in children's bodies (pharmacokinetics) and collects data on safety and tolerability when used to treat pediatric PNH. It also records blood measures such as hemoglobin and lactate dehydrogenase (LDH) and tracks transfusion needs as secondary outcomes.

What the researchers hope to learn. To characterize iptacopan's pharmacokinetics in pediatric patients and to evaluate its safety and tolerability in children with PNH. The study also aims to collect data on hematology measures (hemoglobin, LDH) and transfusion avoidance to inform future use in this population.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • LNP023

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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).

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After screening (up to 8 weeks), participants take oral iptacopan during a 26-week treatment period, followed by a possible 26-week extension. Adolescents (12–<18) receive a fixed dose (200 mg twice daily); younger children are dosed by weight starting at Day 1, with dose reassessments at Weeks 12, 26, and 38. Expect regular clinic visits for safety checks, blood samples for drug levels and lab tests, vital signs and ECGs, and reporting of any adverse events. Vaccination against meningococcal and pneumococcal infections is required before starting treatment (or prophylactic antibiotics if treatment must begin sooner); vaccination against Haemophilus influenzae is also recommended.

How the study is structured. Interventional, open‑label (no placebo or blinding), single‑arm study that enrolls children into sequential age cohorts. Adolescents (12 to <18 years) receive a fixed oral dose (200 mg twice daily); younger children receive weight‑based dosing with reassessments at Weeks 12, 26, and 38. The overall plan is screening (up to 8 weeks), 26 weeks of treatment, then a 26‑week extension; primary measurements include drug levels and monitoring for adverse events.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in Brazil, Colombia, Germany, Italy, Netherlands, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on paroxysmal nocturnal hemoglobinuria

Clinical trials44 trials listed for paroxysmal nocturnal hemoglobinuriaEvery trial the registry lists for paroxysmal nocturnal hemoglobinuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in paroxysmal nocturnal hemoglobinuria researchLatest monthly update: September 2026. Includes: Treatment access. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Oct 28, 2025
Primary completion
Oct 14, 2031 (estimated)
Study completion
Nov 13, 2031 (estimated)
Target enrollment
12 participants (estimated)
Last updated on registry
Aug 7, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Novartis Pharmaceuticals

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (15)

  • Childrens Healthcare of AtlantaRecruiting

    Atlanta, Georgia, 30342 · United States

  • Cancer Institute of New JerseyRecruiting

    New Brunswick, New Jersey, 08901 · United States

  • Childrens Hospital of PhiladelphiaRecruiting

    Philadelphia, Pennsylvania, 19104-4399 · United States

  • St Jude Childrens Research HospitalRecruiting

    Memphis, Tennessee, 38105 · United States

  • Novartis Investigative SiteRecruiting

    Brasília, Federal District, 70684-831 · Brazil

  • Novartis Investigative SiteRecruiting

    Natal, Rio Grande do Norte, 59012 300 · Brazil

  • Novartis Investigative SiteRecruiting

    Porto Alegre, Rio Grande do Sul, 90035-003 · Brazil

  • Novartis Investigative SiteRecruiting

    Santo André, São Paulo, 09090-401 · Brazil

  • Novartis Investigative SiteRecruiting

    São Paulo, São Paulo, 01323001 · Brazil

  • Novartis Investigative SiteRecruiting

    São Paulo, São Paulo, 04038-002 · Brazil

Terms used on this page · explanations by MedicalUpdates.ai

open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
single arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
pharmacokinetics
How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
Cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
stable regimen
A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
Principal Investigator
The doctor or scientist responsible for running the study at a site or overall.
treatment naive
Has not previously received the treatment, or the type of treatment, that the study is about.
during screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
HSCT
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials