Neurofibromatosis 1NCT06360406
Real-World Treatment Study of Koselugo (Selumetinib)
An observational study of Neurofibromatosis 1 and Neurofibroma, Plexiform, run by AstraZeneca at 12 sites in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 99 years
- Conditions the study is for
- Neurofibromatosis 1, Neurofibroma, Plexiform
- How many people
- 200 participants
- Where
- 12 sitesSouth Korea
- Study dates
- 15 Jun 2024to 30 Sep 2031
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients treated with Koselugo (Selumetinib) under the approved label in South Korea.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
12 sites in 1 country
South Korea12 sites, 10 recruiting
- Recruiting
Research Site
Busan
- Recruiting
Research Site
Chungcheongbuk-do
- Recruiting
Research Site
Daejeon
- Recruiting
Research Site
Gyeonggi-do
- Recruiting
Research Site
Incheon
- Recruiting
Research Site
Jeonnam
- Suspended
Research Site
Seoul
- Recruiting
Research Site
Seoul
- Completed
Research Site
Seoul
- Not yet recruiting
Research Site
Seoul
- Recruiting
Research Site
Wŏnju
- Recruiting
Research Site
Yangsan
About this study
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.
- Conditions
- Neurofibromatosis 1, Neurofibroma, Plexiform
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma, Plexiform
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 1 year
Secondary outcomes (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
To assess the effectiveness of the study drug for patients prescribed with the study drug under the approved indication(s) in Korea
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2024
- Study start15 Jun 2024
- Primary completion30 Sep 2031 (estimated)
- Study completion30 Sep 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis 1Studies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06360406
- Sponsor's protocol ID
- D1346R00009
- Status last verified
- Aug 2026
- First submitted
- 8 Apr 2024
- Last updated
- 25 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-World Treatment Study of Koselugo (Selumetinib)
- ClinicalTrials.gov ID
- NCT06360406
- Acronym / sponsor ID
- D1346R00009
- Conditions
- Neurofibromatosis 1, Neurofibroma, Plexiform
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 15 Jun 2024 (actual)
- Primary completion
- 30 Sep 2031 (estimated)
- Study completion
- 30 Sep 2031 (estimated)
- First posted
- 11 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Other
Who may qualify
- Ages
- 3 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients treated with Koselugo (Selumetinib) under the approved label in South Korea.
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Adverse events (AEs) rate
Time frame: 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
Time frame: 1 year
To assess the safety of the study drug for patients prescribed with the study drug under the approved indication(s) in Korea
1 secondary outcome
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2026.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
AstraZeneca Clinical Study Information Center contact
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurofibromatosis 1 and Neurofibroma, Plexiform, run by AstraZeneca at 12 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact AstraZeneca Clinical Study Information Center (1-877-240-9479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (AEs) rate (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2026; AzTrials last refreshed its copy on 26 Oct 2026.