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Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

NCT06188741 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 200

Last updated 2026-03-12

No results posted yet for this study

Summary

Plexiform neurofibromas (PN) are known to cause significant morbidity in children with NF1. The recent FDA approval for selumetinib in children 2 years and older with inoperable symptomatic PN was based on the finding that selumetinib shrinks the majority of PN in children with NF1 and results in clinically meaningful benefit such as improvement in pain or range of motion. However, many morbidities, such as blindness or nerve damage, cannot be fully reversed with PN shrinkage. Therefore, there remains a critical need in this patient population to determine if young participants with PN in high-risk locations may benefit from early medical intervention prior to the development of clinical problems. This study will determine whether participants with asymptomatic PN in high-risk locations can potentially benefit from early treatment with selumetinib.

Conditions

  • Neurofibromatosis 1
  • Plexiform Neurofibroma

Interventions

DRUG

Selumetinib

Selumetinib (KoselugoTM) at the FDA approved dose of 25 mg/m2/dose PO BID

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
1 Year
Max Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-27
Primary Completion
2031-09-01
Completion
2032-09-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06188741 on ClinicalTrials.gov