Real-World Treatment Study of Koselugo (Selumetinib)

As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients treated with Koselugo (Selumetinib), an oral selective inhibitor of MAPK kinase (MEK) 1 and 2, by physicians in routine clinical practice settings. This study is designed to assess the known safety profile or identify previously unsuspected adverse reactions and evaluate the effectiveness of Koselugo under conditions of routine daily medical practice in Korea.

This study will provide information on the Korean patient population that is treated with the study drug.

Study Overview

• Status: Recruiting
• Conditions: Neurofibromatosis 1; Neurofibroma, Plexiform

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: AstraZeneca Clinical Study Information Center; Phone Number: 1-877-240-9479; Email: information.center@astrazeneca.com

Study Locations

• South Korea
  • Busan, South Korea
    • Recruiting
    • Research Site
  • Chungcheongbuk-do, South Korea
    • Recruiting
    • Research Site
  • Daejeon, South Korea
    • Recruiting
    • Research Site
  • Gyeonggi-do, South Korea
    • Recruiting
    • Research Site
  • Incheon, South Korea
    • Recruiting
    • Research Site
  • Jeonnam, South Korea
    • Recruiting
    • Research Site
  • Seoul, South Korea
    • Suspended
    • Research Site
  • Seoul, South Korea
    • Recruiting
    • Research Site
  • Seoul, South Korea
    • Completed
    • Research Site
  • Seoul, South Korea
    • Not yet recruiting
    • Research Site
  • Wŏnju, South Korea
    • Recruiting
    • Research Site
  • Yangsan, South Korea
    • Recruiting
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Patients treated with Koselugo (Selumetinib) under the approved label in South Korea.

Description

Inclusion Criteria:

1. Patients treated with Koselugo (Selumetinib) under the approved label in South Korea
2. Provision of signed and dated written informed consent by the patient or legally acceptable representative

Exclusion Criteria:

1. Patients treated with Koselugo (Selumetinib) under the approved label in South Korea
2. Provision of signed and dated written informed consent by the patient or legally acceptable representative

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events (AEs) rate	To assess the safety (Adverse events (AEs), serious AEs (SAEs), adverse drug reactions (ADRs), serious ADRs (SADRs), unexpected AEs/ADRs) of the study drug for patients prescribed with the study drug under the approved indication(s) in Korea	1 year
Physeal dysplasia occurance rate	To assess the safety of the study drug for patients prescribed with the study drug under the approved indication(s) in Korea	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Descriptive analysis with the physician qualitative assessments by overall disease status of NF-1 and status of clinically significant PNs, respectively. - Improving - Progression - Stable	To assess the effectiveness of the study drug for patients prescribed with the study drug under the approved indication(s) in Korea	1 year

Collaborators and Investigators

• Sponsor: AstraZeneca

Study record dates

Study Major Dates

• Study Start (Actual): June 15, 2024
• Primary Completion (Estimated): September 30, 2031
• Study Completion (Estimated): September 30, 2031

Study Registration Dates

• First Submitted: April 8, 2024
• First Submitted That Met QC Criteria: April 8, 2024
• First Posted (Actual): April 11, 2024

Study Record Updates

• Last Update Posted (Actual): June 1, 2026
• Last Update Submitted That Met QC Criteria: May 29, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms; Neuromuscular Diseases; Genetic Diseases, Inborn; Peripheral Nervous System Diseases; Neoplasms by Histologic Type; Neurodegenerative Diseases; Neoplasms, Nerve Tissue; Nervous System Neoplasms; Heredodegenerative Disorders, Nervous System; Nerve Sheath Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Peripheral Nervous System Neoplasms; Neurofibroma; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurofibromatoses; Neurofibromatosis 1; Neurofibroma, Plexiform
• Other Study ID Numbers: D1346R00009

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 approved.

• 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 refer 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. A Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No