Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children aged ≥1 to <7 years with NF1-related Symptomatic, Inoperable PN - SPRINKLE

Study identifier:D1346C00004

ClinicalTrials.gov identifier:NCT05309668

EudraCT identifier:2020-005608-20

CTIS identifier:2023-506357-38-00

Recruitment Complete

Official Title

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)

Medical condition

Neurofibromatosis Type 1

Phase

Phase 1/2

Healthy volunteers

No

Study drug

Selumetinib granule formulation, Selumetinib capsule formulation

Sex

All

Actual Enrollment

36

Study type

Interventional

Age

1 Years - 6 Years

Date

Study Start Date: 21 Jan 2022
Primary Completion Date: 08 Apr 2024
Estimated Study Completion Date: 28 Apr 2028

Study design

Allocation: N/A
Endpoint Classification: -
Intervention Model: Single Group Assignment
Masking: -
Primary Purpose: Treatment

Verification:

Verified 01 Aug 2024 by AstraZeneca

Sponsors

AstraZeneca

Collaborators

Merck Sharp & Dohme Corp.

Inclusion and exclusion criteria