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NCT06934967 · ClinicalTrials.gov registry record · Phase 3
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
A Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Novartis Pharmaceuticals.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 12
- Enrollment target
- 12
- Study locations
NCT06934967: Recruiting Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Novartis Pharmaceuticals.
NCT06934967 is a Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria (PNH) that is actively recruiting participants, run by Novartis Pharmaceuticals. The registered enrollment target is 12 participants, below the 123-participant average among 5 other Paroxysmal Nocturnal Hemoglobinuria (PNH) trials with a reported enrollment target (90% lower). The trial reports 12 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06934967, a Phase 3 study of Paroxysmal Nocturnal Hemoglobinuria (PNH), is actively recruiting participants, sponsored by Novartis Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 12 participants
- Enrollment target
- 12
- Study locations
Study 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 when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to \< 18 years of age.
Primary Outcome
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.
Conditions Studied
Interventions
- DRUG LNP023
Study Locations (12)
São Paulo
- Novartis Investigative Site - Santo André
- Novartis Investigative Site - São Paulo
- Novartis Investigative Site - São Paulo
Other
- Novartis Investigative Site - Berlin
- Novartis Investigative Site - Utrecht
New Jersey
- Cancer Institute of New Jersey - New Brunswick
Pennsylvania
- Childrens Hospital of Philadelphia - Philadelphia
Federal District
- Novartis Investigative Site - Brasília
Rio Grande do Norte
- Novartis Investigative Site - Natal
Rio Grande do Sul
- Novartis Investigative Site - Porto Alegre
Valle del Cauca Department
- Novartis Investigative Site - Cali
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2025-10-28 |
| Est. Completion | 2031-12-19 |
| Phase | Phase 3 |
What NCT06934967 shows while recruiting
NCT06934967 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 123-participant average among 5 other Paroxysmal Nocturnal Hemoglobinuria (PNH) trials with a reported enrollment target (90% lower).
The record links to 1 condition, with Paroxysmal Nocturnal Hemoglobinuria (PNH) appearing as the primary indexed condition, and to 1 intervention - of which LNP023 is the first listed.
NCT06934967 lists 12 locations in 9 states (São Paulo, Other, New Jersey).
Frequently Asked Questions
What is clinical trial NCT06934967 about?
NCT06934967 is a clinical study titled "Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients". 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 \< 1...
What is the current status of trial NCT06934967?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 12 participants. The study started on 2025-10-28. Estimated completion is 2031-12-19.
What conditions does trial NCT06934967 study?
This clinical trial studies the following conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH).
What interventions are being tested in trial NCT06934967?
The interventions under investigation include: LNP023 (DRUG).
Who is sponsoring clinical trial NCT06934967?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06934967 being conducted?
This trial has 12 study locations across New Jersey, Pennsylvania, Federal District, Rio Grande do Norte, Rio Grande do Sul. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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