US Selumetinib Registry

Terminated

• Conditions: Plexiform Neurofibromas; Neurofibromatosis Type 1

• Registration Number: NCT05683678
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

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 participants with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who were aged 2 to 18 years at the time selumetinib was started in a real-world setting.

Detailed Description

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

Study Timeline

• Study Start: 2019-01-04
• Study First Submitted: 2023-01-05
• Study First Submitted QC: 2023-01-05
• Study First Posted: 2023-01-13
• Primary Completion: 2024-11-13
• Study Completion: 2024-11-13
• Status Verified: 2025-03
• Last Update Submitted: 2025-04-02
• Last Update Posted: 2025-04-04

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 37

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician	Up to Month 60
Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician	Up to Month 60
Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations	Up to Month 60	Skeletal manifestations may include changes in the angle of spine curvature.
Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)	Up to Month 60

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60	Baseline, Month 60
Change From Baseline in the Pain Interference Index (PII) Score at Month 60	Baseline, Month 60
Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60	Baseline, Month 60
Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60	Baseline, Month 60

Trial Locations

Locations (10)

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

Nicklaus Children's Hospital; 🇺🇸 Miami, Florida, United States

Arnold Palmer Hospital For Children; 🇺🇸 Orlando, Florida, United States

Ochsner Medical Center; 🇺🇸 New Orleans, Louisiana, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Albany Medical Center; 🇺🇸 Albany, New York, United States

The Children's Hospital at Montefiore (CHAM); 🇺🇸 Bronx, New York, United States

Waters Center for Childrens Cancer and Blood Disorders; 🇺🇸 Syracuse, New York, United States

Cook Children's Medical Center; 🇺🇸 Fort Worth, Texas, United States