A Phase I/II, Single-Arm, Open-label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged >= 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) - SPRINKLE

Astra Zeneca0 sites5 target enrollmentTBD

Overview

No summary available.

Registry

who.int

Start Date

TBD

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional

Sponsor

Astra Zeneca

•\- Male and female participants aged \>\= 1 to \< 7 years of age at the time their
•legally authorised representative (parent or guardian) signs the informed
•consent. \- All participants must have a diagnosis of NF1 with symptomatic
•inoperable PN where: a) Participants must have PN and at least one other
•diagnostic criterion for NF1 b) Inoperable is defined as a PN that cannot be
•completely surgically removed without a risk of substantial morbidity due to
•encasement of, or close proximity to, vital structures, invasiveness, or high
•vascularity of the PN; or unacceptable risk from the general anaesthetic as
•assessed by the investigator c) Symptomatic is defined as clinically
•significant symptoms or complications caused by the PN, as judged by the

•\- Participants with confirmed suspected malignant glioma or MPNST. Participants
•with optic glioma not requiring chemotherapy or radiation therapy are
•permitted. \- History of malignancy except for malignancy treatment with
•curative intent with no known active disease \>\= 2 years before the first dose of
•study intervention and of low potential risk of recurrence. \- Refractory nausea
•and vomiting, chronic gastrointestinal disease, inability to swallow the
•formulated product, or previous significant bowel resection that would preclude
•adequate absorption, distribution, metabolism, or excretion of selumetinib. \- A
•life\*threatening illness, medical condition, organ system dysfunction or
•laboratory finding which, in the Investigator's opinion, could compromise the

Not specified