Danicopan PMS in Korea

May 21, 2026 updated by: AstraZeneca

Danicopan Regulatory Post-Marketing Study in Korea

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.

Study Overview

Status

Recruiting

Conditions

Detailed Description

The objectives of this study are to assess the safety and effectiveness of Danicopan in a real world setting in patients who are prescribed with the study drug under the approved indication in Korea.

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

Secondary objective(s) To assess effectiveness of Danicopan as add- on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patients with PNH at 12 weeks.

Study Type

Observational

Enrollment (Estimated)

8

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • South Korea
      • 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
        • Recruiting
        • Research Site
      • Seoul, South Korea
        • Not yet recruiting
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-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.

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety (adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs), AESI
Time Frame: Patient data will be gathered for up to 12 weeks from the first dose of the study drug.
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in hemoglobin (Hgb) at Week 12
Time Frame: Effectiveness variables will be assessed at Week 12 or end of treatment (if treatment is discontinued before Week 12).
Hemoglobin (Hgb) will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).
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
Time Frame: Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)
Absolute reticulocyte count (ARC) will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).
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
Time Frame: Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)
FACIT Fatigue scores will be summarized with descriptive statistics (mean, standard deviation, median, minimum, and maximum).
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)
Time Frame: Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)
The proportion of patients with transfusion avoidance will be summarized. Patients with transfusion avoidance will be considered to show effectiveness and the number and percentage of subjects corresponding to this classification will be presented, along with the 95% confidence interval (CI) for the percentage calculated using the Clopper-Pearson method.
Effectiveness variables will be assessed at Week 12 orend of treatment (if treatment is discontinued before Week 12)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AstraZeneca

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 10, 2026

Primary Completion (Estimated)

June 27, 2028

Study Completion (Estimated)

June 27, 2028

Study Registration Dates

First Submitted

February 13, 2026

First Submitted That Met QC Criteria

March 3, 2026

First Posted (Actual)

March 9, 2026

Study Record Updates

Last Update Posted (Actual)

May 22, 2026

Last Update Submitted That Met QC Criteria

May 21, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D7332R00002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

IPD Sharing 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.

IPD Sharing 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.

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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