NCT06934967 · Novartis Pharmaceuticals
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
What this study is about
The purpose of this where both patients and doctors know the treatment given, single treatment group$1, conducted at multiple hospitals, phase 3 study is to assess the how the drug moves through the body 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.
View original scientific 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.
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.
Primary outcome measures
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 26 weeks
Incidence and severity of AEs and SAEs by treatment group, including changes in vital signs, electrocardiograms (ECGs) and laboratory results qualifying and reported as AEs.
PK parameter (Cmax)
Time frame: Week 2
Cmax is defined as the maximum (peak) observed concentration following a dose.
PK parameter (AUClast)
Time frame: Week 2
AUClast is the area under the plasma concentration-time curve from time zero to the time of last quantifiable concentration (tlast).
PK parameter (AUCtau)
Time frame: Week 2
AUCtau describes the area under the curve limited to the end of a dosing interval.
PK parameter (Ctrough)
Time frame: Weeks 2, 4, 12 and 26
Ctrough is the observed plasma concentration that is just prior to the beginning of, or at the end of a dosing interval.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- New Brunswick, New Jersey
- Philadelphia, Pennsylvania
- Memphis, Tennessee
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 16, 2026 · Source of record for eligibility and locations