US Selumetinib Registry

NCT05683678 | Terminated

• 1 other identifier: ALX-NF1-501
• Study Type: observational
• Target: 37
• Locations: 1 country
• Sites: 10

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 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

Neurofibromatosis Type 1; Plexiform Neurofibromas

Keywords

neurofibromatosis type 1; plexiform neurofibromas; NF1; PNs

Outcome Measures

Primary Outcomes (4)

• 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

  Skeletal manifestations may include changes in the angle of spine curvature.

  Up to Month 60

• Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)

  Up to Month 60

Secondary Outcomes (4)

• 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 Patient Global Impression of Change (GIC) Scale at Month 60

  Baseline, Month 60

• Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60

  Baseline, Month 60

Study Arms (3)

Cohort 1

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

Cohort 2

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

Cohort 3

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

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Approximately 200 participants who meet the eligibility criteria will be enrolled from 20-25 sites.

You may qualify if:

• 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.

You may not qualify if:

• Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (10)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027-6062, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Arnold Palmer Hospital For Children

Orlando, Florida, 32806, United States

Location

Ochsner Medical Center

New Orleans, Louisiana, 70121, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Albany Medical Center

Albany, New York, 12208, United States

Location

Waters Center for Childrens Cancer and Blood Disorders

Syracuse, New York, 13210, United States

Location

The Children's Hospital at Montefiore (CHAM)

The Bronx, New York, 10467, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma, Plexiform

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 5, 2023
• First Posted: January 13, 2023
• Study Start: January 4, 2019
• Primary Completion: November 13, 2024
• Study Completion: November 13, 2024
• Last Updated: April 4, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

US (10)