Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours

Sponsor
Great Ormond Street Hospital for Children NHS Foundation Trust
Study ID
NCT03326388
Phase
PHASE1/PHASE2
Status
Completed

Conditions

  • Neurofibromatosis Type 1
  • Optic Nerve Glioma
  • Plexiform Neurofibroma

Eligibility Criteria

Sex
ALL
Age
3 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

Phase I and II study of the MEK inhibitor Selumetinib given twice daily on 5 out of 7 days in children with NF1 and inoperable plexiform neurofibromas or progressive/relapsed optic pathway gliomas. This study will test the early and late toxicities of selumetinib when it is given in this intermittent schedule (in 5 out of 7 days) and will also test the effectiveness of the drug in reducing the size of plexiform neurofibromas and optic pathway gliomas in children with NF1. It will also test the effectiveness of the drug in improving the participants function in day to day life.

Key Dates

Start date
Sep 26, 2019
Status verified
Apr 2024
Primary completion
Feb 14, 2024
Completion
Feb 14, 2024

Study Design

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

Arms

  • Experimental: Selumetinib Intermittent Dosing
    Phase 1 of the study to evaluate Intermittent Dosing (Selumetinib given twice daily on 5 out of 7 days) in children with NF1 and inoperable plexiform neurofibromas. The Maximum tolerated dose will define the Recommended phase 2 dose of selumetinib.

Primary Outcome Measure

Phase 1: To evaluate the Maximum Tolerated Dose [ Time Frame: 6 months ]

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