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

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
University of Alabama at Birmingham
Study ID
NCT06188741
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Neurofibromatosis 1
  • Plexiform Neurofibroma

Eligibility Criteria

Sex
ALL
Age
1 Year - 8 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Aug 27, 2025
Status verified
Sep 2025
Primary completion
Sep 1, 2031
Completion
Sep 1, 2032

Study Design

Enrollment
200 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • No Intervention: Part 1: WBMRI for NF1 patients with no known PN
    To assess the incidence of asymptomatic PN in any location in participants with NF1 and no known PN
  • Experimental: Part 2: Treatment randomization to selumetinib vs observation
    To determine if selumetinib treatment prevents PN growth in young participants with asymptomatic tumors in high-risk locations
  • Experimental: Part 3: Part 2 participants with growing or symptomatic PN
    To assess the proportion of participants who are able to maintain tumor response after transition to an intermittent dosing schedule

Primary Outcome Measure

Progression free survival (PFS) [ Time Frame: 60 months ]

Central Contacts

Locations (15)

FacilityCityStateZIPSite coordinators
Childrens of AlabamaBirminghamAlabama35233
Manjul Virmani, BS
[email protected]
205-638-6259
Britney McKenzie
[email protected]
205-638-6259
Girish Dhall, MD (PRINCIPAL_INVESTIGATOR)
Laura Katie Metrock, MD (SUB_INVESTIGATOR)
Children's Hospital of Los AngelesLos AngelesCalifornia90027
Tena Rosser, MD
[email protected]
323-361-2471
Martha Arellano-Garcia, MS
[email protected]
323-361-5812
Tena Rosser, MD (PRINCIPAL_INVESTIGATOR)
Nathan Robison, MD (SUB_INVESTIGATOR)
Stanford UniversityPalo AltoCalifornia94304
Cynthia Campen, MD
[email protected]
650-497-8953
Cynthia Campen, MD (PRINCIPAL_INVESTIGATOR)
Children's National HospitalWashington D.C.District of Columbia20010
Benjamin Siegel, MD
[email protected]
202-476-5181
Benjamin Siegel, MD (PRINCIPAL_INVESTIGATOR)
Lurie Children's Hospital of ChicagoChicagoIllinois60611
Kelly Bruno
[email protected]
(312)227-4810
Miriam Bornhorst, MD (PRINCIPAL_INVESTIGATOR)
University of ChicagoChicagoIllinois63637
Cynthia MacKenzie
[email protected]
773-702-6487
James Tonsgard, MD (PRINCIPAL_INVESTIGATOR)
Riley Hospital for Children/Indiana UniversityIndianapolisIndiana46202
Steven Rhodes, MD, PhD
[email protected]
(317) 948-6641
Steven Rhodes, MD, PhD (PRINCIPAL_INVESTIGATOR)
Johns Hopkins UniversityBaltimoreMaryland21231
Joshua Roberts, PhD
[email protected]
(410) 955-6827
Jaishri Blakeley, MD (PRINCIPAL_INVESTIGATOR)
National Cancer Institute/ National Institutes of HealthBethesdaMaryland20892
Hemaxi Patel
[email protected]
240-243-8838
Brigitte Widemann, MD (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115
Olivia Clancy
[email protected]
617-919-0291
Nicole Ullrich, MD, PhD (PRINCIPAL_INVESTIGATOR)
Mayo ClinicRochesterMinnesota55905
Meg Connors
[email protected]
507-266-2275
Radhika Dhamija, MBBS (PRINCIPAL_INVESTIGATOR)
Washington University - St. LouisSt LouisMissouri63110
Amy Armstrong, MD
[email protected]
314-454-6018
Amy Armstrong, MD (PRINCIPAL_INVESTIGATOR)
Cincinnati Childrens Hospital Medical CenterCincinnatiOhio45229-
Lori Backus
[email protected]; [email protected]
513-517-2068
Andrea Gross, MD (SUB_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Ratnakar Patti
[email protected]
267-426-5503
Michael Fisher, MD (PRINCIPAL_INVESTIGATOR)
University of Texas, SouthwesternDallasTexas75390
Laura Klesse, MD, PhD
[email protected]
(214) 456-2382
Laura Klesse, MD, PhD (PRINCIPAL_INVESTIGATOR)

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