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Study to assess the pharmacokinetics, safety, and tolerability of iptacopan in pediatric PNH patients
EU CTIS ID: 2024-515926-10-00
What this study is testing
To evaluate the safety and tolerability of iptacopan during the 26-week treatment period. To characterize the PK of iptacopan.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
- Participants 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.
- Participants who are anti-C5 treatment naive: mean hemoglobin level < 10 g/dL confirmed by central laboratory assessment during screening.
- Participants 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.
You likely can't join if
- 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).
- Participants with laboratory evidence of bone marrow failure (reticulocytes < 100 × 10^9/L, platelets < 30 × 10^9/L, neutrophils < 0.5 × 10^9/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.
The study team makes the final eligibility decision.
Where it's taking place
- Colombia
- United States
- Brazil
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Colombia; United States; Brazil. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.