Real-World Treatment Study of Koselugo (Selumetinib)

Recruiting

• Conditions: Neurofibromatosis 1; Neurofibroma, Plexiform

• Registration Number: NCT06360406
• Lead Sponsor: AstraZeneca

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-04-08
• Study First Submitted QC: 2024-04-08
• Study First Posted: 2024-04-11
• Study Start: 2024-06-15
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-09
• Last Update Posted: 2025-05-11
• Primary Completion: 2031-09-30
• Study Completion: 2031-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 150

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Adverse events (AEs) rate	1 year	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
Physeal dysplasia occurance rate	1 year	To assess the safety of the study drug for patients prescribed with the study drug under the approved indication(s) in Korea

Secondary Outcome Measures

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

Trial Locations

Locations (2)

Research Site; 🇰🇷 Yangsan-si, Korea, Republic of

Research Site; 🇰🇷 Yangsan-si, Korea, Republic of