OncoMatch/Clinical Trials/NCT06934967
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
Is NCT06934967 recruiting? Yes, currently enrolling (May 2026). This Phase 3 trial studies LNP023 for paroxysmal nocturnal hemoglobinuria (pnh).
Treatment: LNP023 — 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.
Check if I qualifyExtracted eligibility criteria
Prior therapy
Must have received: anti-C5 therapy — maintenance
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
Cannot have received: hematopoietic stem cell transplantation
History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1)
Lab requirements
Blood counts
Patients with laboratory evidence of bone marrow failure (reticulocytes < 100 x 10^9/L; platelets < 30 × 10^9/L; neutrophils < 0.5 × 10^9/L) are excluded
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)
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- Cancer Institute of New Jersey · New Brunswick, New Jersey
- Childrens Hospital of Philadelphia · Philadelphia, Pennsylvania
Showing up to 5 US sites. See all sites on ClinicalTrials.gov →
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