Status:
RECRUITING
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Eligibility:
All Genders
2-18 years
Phase:
PHASE3
Brief Summary
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 w...
Detailed Description
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 e...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
October 28 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 19 2031
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06934967
Start Date
October 28 2025
End Date
December 19 2031
Last Update
December 23 2025
Active Locations (12)
Enter a location and click search to find clinical trials sorted by distance.
1
Cancer Institute of New Jersey
New Brunswick, New Jersey, United States, 08901
2
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104 4399
3
Novartis Investigative Site
Brasília, Federal District, Brazil, 70684-831
4
Novartis Investigative Site
Natal, Rio Grande do Norte, Brazil, 59012 300