NCT07457151

Brief Summary

As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical practice settings. This study is designed to assess the known safety profile or identify previously unsuspected adverse reactions and to evaluate the effectiveness of Danicopan under conditions of routine daily medical practice in Korea.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for all trials

Timeline
26mo left

Started Apr 2026

Typical duration for all trials

Geographic Reach
1 country

4 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress4%
Apr 2026Jun 2028

First Submitted

Initial submission to the registry

February 13, 2026

Completed
24 days until next milestone

First Posted

Study publicly available on registry

March 9, 2026

Completed
1 month until next milestone

Study Start

First participant enrolled

April 10, 2026

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 27, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 27, 2028

Last Updated

April 17, 2026

Status Verified

April 1, 2026

Enrollment Period

2.2 years

First QC Date

February 13, 2026

Last Update Submit

April 16, 2026

Conditions

Keywords

PNH

Outcome Measures

Primary Outcomes (1)

  • Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI

    To assess the safety of Danicopan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab)in patients with PNH in Korea.

    Patient data will be gathered for up to 12 weeks from the first dose of the study drug.

Secondary Outcomes (4)

  • Change from baseline in hemoglobin (Hgb) at Week 12

    Effectiveness variables will be assessed at Week 12 or end of treatment (if treatment is discontinued before Week 12).

  • Change from baseline in absolute reticulocyte count (ARC) at Week 12

    Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)

  • Change from baseline in FACIT Fatigue scores at Week 12

    Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)

  • Proportion of patients with transfusion avoidance* at Week 12(* Transfusion Avoidance: Defined as remaining free from red blood cell transfusions)

    Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients currently treated with or initiating the study drug under approved local label and conditions of routine daily medical practice in Korea will be included in the study. This study will be conducted using a total surveillance method to investigate the safety and effectiveness of all patients being treated with the study drug.

You may qualify if:

  • Patients eligible for and treated with Danicpan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patient with PNH in Korea
  • Provision of a signed and dated written informed consent by the patient or their legally acceptable representative

You may not qualify if:

  • Participation in any concurrent interventional trials during the period of study drug treatment
  • Other off-label indications according to the approved label in Korea

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Research Site

Busan, South Korea

RECRUITING

Research Site

Daegu, South Korea

NOT YET RECRUITING

Research Site

Hwasun-gun, South Korea

NOT YET RECRUITING

Research Site

Seoul, South Korea

NOT YET RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 13, 2026

First Posted

March 9, 2026

Study Start

April 10, 2026

Primary Completion (Estimated)

June 27, 2028

Study Completion (Estimated)

June 27, 2028

Last Updated

April 17, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

Time Frame
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access Criteria
When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
More information

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