A Phase 1 interventional study of Selumetinib in Neurofibromatosis 1 and Neurofibroma Plexiform, sponsored by AstraZeneca. Active, not recruiting at 2 sites in China. Open to participants aged 3 Years to 99 Years. Per ClinicalTrials.gov, last updated 2026-08-18.
Sponsored by AstraZeneca · Phase 1, Interventional, and Treatment
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).
Paediatric and adult patients with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) will be evaluated in the screening visit to confirm eligibility. Approximately 16 paediatric and 16 adult qualified patients will receive oral selumetinib 25 mg/m\^2 twice a day (approximately every 12 hours) continuously until disease progression or unacceptable drug-related toxicity, whichever occurs first. Once a patient has discontinued the study treatment then the patient will be followed for specified period for safety assessment
Exclusion Criteria:
Current or past history of retinal pigment epithelial detachment/central serous retinopathy or retinal vein occlusion; Intraocular pressure >21 mmHg (or ULN adjusted by age) or uncontrolled glaucoma (irrespective of IOP); Subjects with known glaucoma and increased IOP who do not have meaningful vision (light perception only or no light perception) and are not experiencing pain related to the glaucoma, may be eligible after discussion with the study physician; Any other significant abnormality on ophthalmic examination that would make the subject unsuitable for enrolment into the study, as assessed by the investigator.
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.
Drug: Selumetinib
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.
Also known as: Koselugo
Adverse events
* Occurrence/frequency. * Relationship to IP as assessed by investigator. * Common Terminology Criteria for Adverse Events (CTCAE) grade. * Seriousness. * Death. * Adverse events leading to discontinuation of IP. * Adverse events of special interest.
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.
Area under the concentration-time curve from zero to the last measurable concentration (AUC0-t) of selumetinib and its metabolite (N-desmethyl selumetinib) in Chinese paediatric and adult subjects with NF 1 and inoperable Plexiform Neurofibromas
AUC0-t after single dose and multiple doses administration
Time frame: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
Maximum plasma concentration (Cmax) of selumetinib and its metabolite (N-desmethyl selumetinib) in Chinese paediatric and adult subjects with NF 1 and inoperable Plexiform Neurofibromas
Cmax after single dose and multiple doses administration
Time frame: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
Terminal half-life (t1/2) of selumetinib and its metabolite (N-desmethyl selumetinib) in Chinese paediatric and adult subjects with NF 1 and inoperable Plexiform Neurofibromas
t1/2 after single dose and multiple doses administration
Time frame: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
objective response rate (ORR) of selumetinib in Chinese paediatric and adult subjects with Neurofibromatosis Type 1 and inoperable Plexiform Neurofibromas
measured by 3D volumetric magnetic resonance imaging (MRI) of the target and nontarget PN
Time frame: First patient first dose until up to 2 years after last subject dosed
duration of response (DoR) of selumetinib in Chinese paediatric and adult subjects with Neurofibromatosis Type 1 and inoperable Plexiform Neurofibromas
measured by 3D volumetric magnetic resonance imaging (MRI) of the target and nontarget PN
Time frame: First patient first dose until up to 2 years after last subject dosed
progression-free survival (PFS) of selumetinib in Chinese paediatric and adult subjects with Neurofibromatosis Type 1 and inoperable Plexiform Neurofibromas
measured by 3D volumetric magnetic resonance imaging (MRI) of the target and nontarget PN
Time frame: First patient first dose until up to 2 years after last subject dosed
time to progression (TTP) of selumetinib in Chinese paediatric and adult subjects with Neurofibromatosis Type 1 and inoperable Plexiform Neurofibromas
measured by 3D volumetric magnetic resonance imaging (MRI) of the target and nontarget PN
Time frame: First patient first dose until up to 2 years after last subject dosed
time to response (TTR) of selumetinib in Chinese paediatric and adult subjects with Neurofibromatosis Type 1 and inoperable Plexiform Neurofibromas
measured by 3D volumetric magnetic resonance imaging (MRI) of the target and nontarget PN
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures of Physical function via Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures health-related quality of life (HRQoL) via PedsQL (paediatric cohort, self-and parent-reported)
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures of pain via FLACC scale
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures health-related quality of life (HRQoL) via EORTC QLQ-C30 (adult cohort)
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures health-related quality of life (HRQoL) via PlexiQoL (adult cohort)
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures of pain via Faces Pain Scale (revised)
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures of pain via NRS-11
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures of pain via PII
Time frame: First patient first dose until up to 2 years after last subject dosed
Measures of pain via Pain Medication Survey
Time frame: First patient first dose until up to 2 years after last subject dosed
Plan to share: Yes — Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
This study is active, not recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
AstraZeneca