Clinical trial · Observational
US Selumetinib Registry
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Sponsor decision.
Summary
Brief summary (as posted)
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020 in a real-world setting.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neurofibromatosis Type 1 | — | UNRESOLVED | — |
| Plexiform Neurofibromas | — | UNRESOLVED | — |
Interventions
Interventions (0)
Data not yet available
Design
Arms and outcomes
Arms (3)
- label
- Cohort 1
- description
- Participants who started selumetinib and discontinued selumetinib before enrollment (the "discontinued" cohort).
- label
- Cohort 2
- description
- Participants who started selumetinib before enrollment and are continuing to receive selumetinib at the time of enrollment (the "continuing" cohort).
- label
- Cohort 3
- description
- Participants who started selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment (the "initiating" cohort).
Primary outcomes (4)
- measure
- Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
- timeFrame
- Up to Month 60
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 2 Years
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Participants diagnosed with NF1 and PN * Started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020, including patients who: * Discontinued selumetinib before enrollment; or * Initiated treatment before enrollment and are currently on selumetinib; or * Initiated selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment * Able to give written informed consent. Participant or participant's parent/legal guardian must be willing and able to give written informed consent and the participant (if minor) must be willing to give written informed assent. Exclusion Criteria: * Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.
References
Publications (0)
Data not yet available