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

ConditionsNeurofibromatosis 1Plexiform Neurofibroma
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-8
SponsorUniversity of Alabama at Birmingham

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age: > 1 (>12 months) and ≤8 years of age at the time of study enrollment.

Diagnosis: Participants with a diagnosis of NF1 based on the 2021 revised consensus criteria [52] and

No known PN (prior to enrollment on Part 1). Participants for whom there is clinical suspicion for a PN (e.g., subtle facial asymmetry or large overlying hyperpigmented area) may be included in the study after discussion with the Study Chair so long as they have not previously had an MRI of the region of concern and are otherwise asymptomatic.

Physical exam at your institution within 1 year prior to consent.

Disqualifiers

Presence of a known, symptomatic PN with or without previous MRI imaging.

Patients who have had previous whole-body MRI (WBMRI) are excluded from the study. However, patients who have had regional MRI(s) for an indication other than a PN and did not have a PN identified on previous MRI may still be eligible for the study.

Inability to undergo MRI and/or contraindication for MRI examinations following the MRI protocol.

Prior treatment with selumetinib or another specific MEK1/2 inhibitor.

Trial design

Design model

Sequential

Treatments tested in this trial

  • Selumetinib

    Drug

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

Treatment groups

200 Participants
are divided into 3 treatment groups
Group A: Part 1: WBMRI for NF1 patients with no known PNNo intervention 0 interventions
Group B: Part 2: Treatment randomization to selumetinib vs observationExperimental treatment 1 intervention
Group C: Part 3: Part 2 participants with growing or symptomatic PNExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Progression free survival (PFS)

Progression free survival (PFS) in the group treated with selumetinib compared to those in the observation group

Time frame
60 months

Secondary outcomes

1

Participants found to have a previously unknown measurable PN

Proportion of participants found to have a previously unknown measurable PN in any location on WBMRI imaging

Time frame
60 months
2

PFS

2\) PFS through one year after transitioning from continuous to an intermittent dosing schedule

Time frame
60 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Alabama at Birmingham

Lead sponsor

Children's Hospital of Philadelphia

Collaborator

Congressionally Directed Medical Research Programs

Collaborator

Source ClinicalTrials.gov
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