A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)

Sponsor
AstraZeneca
Study ID
NCT04590235
Phase
PHASE1
Status
Active Not Recruiting

Conditions

  • Neurofibroma Plexiform
  • Neurofibromatosis 1

Eligibility Criteria

Sex
ALL
Age
3 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • Selumetinib — DRUG
    All eligible subjects will first receive a single oral dose of selumetinib 25 mg/m\^2. After a washout period of 2 days, oral selumetinib 25 mg/m\^2 twice daily will be administered continuously. Subjects will continue to receive selumetinib until disease progression or unacceptable drug-related toxicity, whichever occurs first. 10 mg and 25 mg capsules strengths available.

Study Details

A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN).

Key Dates

Start date
Dec 16, 2020
Status verified
May 2026
Primary completion
Aug 16, 2022
Completion
Aug 31, 2026

Study Design

Enrollment
32 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Selumetinib
    All eligible subjects will first receive a single oral dose of selumetinib 25 mg/m\^2. Then, selumetinib 25 mg/m\^2 oral twice daily will be administered continuously until disease progression or unacceptable drug-related toxicity, whichever occurs first.

Primary Outcome Measure

Adverse events [ Time Frame: For paediatric cohort: from signing the informed consent form until up to 3 years after last subject dosed; For adult cohort: from signing the informed consent form until up to 2 years+30 days after last subject dosed. ]

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