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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 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.

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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.

Conditions Studied

Paroxysmal Nocturnal Hemoglobinuria (PNH)

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

FieldValue
Enrollment Target 12 participants
Start Date 2025-10-28
Est. Completion 2031-12-19
Phase Phase 3

Sponsor

Novartis Pharmaceuticals

1,375 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06934967

The ClinicalTrials.gov registry entry for NCT06934967 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 123-participant average among 5 other Paroxysmal Nocturnal Hemoglobinuria (PNH) trials with a reported enrollment target (90% lower). The listed sponsor is Novartis Pharmaceuticals, which has 1,375 total studies on file at ClinicalTrials.gov.

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 reports 12 study locations spanning 9 distinct geographic areas - top geographies include 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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