Real-World Treatment Study of Koselugo (Selumetinib)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Neurofibromatosis 1, Neurofibroma, Plexiform. Basic parameters: 3 years — 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- South Korea
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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.
Primary outcome measures
- Adverse events (AEs) rate [Time frame: 1 year]
- Physeal dysplasia occurance rate [Time frame: 1 year]
Secondary outcome measures (1)
- Descriptive analysis with the physician qualitative assessments by overall disease status of NF-1 and status of clinically significant PNs, respectively. - Improving - Progression - Stable [Time frame: 1 year]
Eligibility criteria
Inclusion criteria
- Patients treated with Koselugo (Selumetinib) under the approved label in South Korea
- Provision of signed and dated written informed consent by the patient or legally acceptable representative
Exclusion criteria
- Patients treated with Koselugo (Selumetinib) under the approved label in South Korea
- Provision of signed and dated written informed consent by the patient or legally acceptable representative
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
South Korea · 12 centers
- Research Site — Busan
- Research Site — Chungcheongbuk-do
- Research Site — Daejeon
- Research Site — Gyeonggi-do
- Research Site — Incheon
- Research Site — Jeonnam
- Research Site — Seoul
- Research Site — Seoul
- … and 4 more centers
Identifiers
NCT: NCT06360406 · D1346R00009