MedTrace Logo

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

NCT06449001 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-12-23

No results posted yet for this study

Summary

The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

Conditions

Interventions

DRUG

Danicopan

Participants will receive danicopan on a weight-based dosing regimen.

Sponsors & Collaborators

  • AstraZeneca

    collaborator INDUSTRY

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-11
Primary Completion
2027-05-28
Completion
2028-03-10
FDA Drug
Yes

Countries

  • Canada
  • France
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06449001 on ClinicalTrials.gov