US Selumetinib Registry

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Total Enrollment

37 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-01-04

Study Completion Date

2024-11-13

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors

NCT03095248

Neurofibromatosis 2 Vestibular Schwannoma Meningioma +2 more

TERMINATED PHASE2

Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST

NCT06735820

Malignant Peripheral Nerve Sheath Tumor (MPNST) Neurofibromatosis 1 (NF1) Atypical Neurofibroma

NOT_YET_RECRUITING PHASE1/PHASE2

A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors

NCT05985161

Wilms Tumor Rhabdoid Tumor Malignant Peripheral Nerve Sheath Tumors +4 more

RECRUITING PHASE2

Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

NCT00727233

Neurofibromatosis Type I Plexiform Neurofibroma

COMPLETED PHASE1

Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas

NCT05253131

MPNST NF1 Sarcoma

NOT_YET_RECRUITING PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Neurofibromatosis Type 1 Plexiform Neurofibromas

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Cohort 1

Participants who started selumetinib and discontinued selumetinib before enrollment (the "discontinued" cohort).

No interventions assigned to this group

Cohort 2

Participants who started selumetinib before enrollment and are continuing to receive selumetinib at the time of enrollment (the "continuing" cohort).

No interventions assigned to this group

Cohort 3

Participants who started selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment (the "initiating" cohort).

No interventions assigned to this group

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.