Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

Part of paid clinical trials in Akron, Ohio.

Sponsor
AstraZeneca
Study ID
NCT05309668
Phase
PHASE1/PHASE2
Status
Active Not Recruiting

Conditions

  • Neurofibromatosis Type 1

Eligibility Criteria

Sex
ALL
Age
1 Year - 6 Years
Healthy Volunteers
Not accepted

Interventions

  • Selumetinib granule formulation — DRUG
    Selumetinib granule formulation will be administered using BSA-based dosing. The granule formulation dose schema to be used in the study will be established in the dose finding phase. At enrolment participants must have a BSA within the range 0.40 to 1.09 m2; once participants attain a BSA between 1.10 and 1.29 m2 they will be encouraged to transition to the capsule formulation, if feasible, although all participants must remain on the granule formulation until after they have completed their third cycle of treatment.
  • Selumetinib capsule formulation — DRUG
    Selumetinib capsule formulation will be administered using BSA-based dosing. Once participants attain a BSA between 1.10 and 1.29 m2 they will be encouraged to transition to the capsule formulation, if feasible, although all participants must remain on the granule formulation until after they have completed their third cycle of treatment.

Study Details

This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints. The study will inform the benefit risk profile of the granule formulation in children aged ≥ 1 to \< 7 years with NF1 related symptomatic, inoperable PN.

Key Dates

Start date
Jan 21, 2022
Status verified
May 2026
Primary completion
Nov 30, 2027
Completion
Apr 28, 2028

Study Design

Enrollment
36 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Selumetinib single arm
    This study consists of a screening period (up to 28 days), a treatment period (25 cycles) and a long term safety follow-up for participants until they are 5 years old or commence an alternative systemic NF1-PN treatment, whichever is the earlier. Participants may continue treatment with selumetinib throughout the long term safety follow-up as long as they are considered to be receiving clinical benefit in the opinion of their Investigator. A safety follow up assessment will be performed 30 days after the last dose of study intervention for all study participants.

Primary Outcome Measure

Selumetinib AUC0-12 Derived After Single Dose Administration [ Time Frame: Pre-dose and 1, 2, 3, 4, 6, 8 and 10-12 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1) (each cycle is 28 days) ]

Locations (4)

FacilityCityStateZIPSite coordinators
Research SiteAkronOhio44308-
Research SitePhiladelphiaPennsylvania19104-
Research SiteHoustonTexas77030-
Research SiteRichmondVirginia23219-

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