Neurofibromatosis 1Phase 1NCT04590235
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
A Phase 1 study testing Selumetinib in people with Neurofibromatosis 1 and Neurofibroma Plexiform, run by AstraZeneca at 2 sites in China.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 3 to 99 years
- Conditions the study is for
- Neurofibromatosis 1, Neurofibroma Plexiform
- What is being tested?
- Selumetinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 2 sitesChina
- Study dates
- 16 Dec 2020to 31 Aug 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Paediatric cohort: Chinese subjects ≥3 years and <18 years of age
- Adult cohort: Chinese subjects ≥18 years of age at the time of study enrollment
- Subjects must be diagnosed with (i) NF1 as per NIH Consensus Development Conference Statement and（ii) PN is defined as a neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches. (iii) inoperable PN
- Subjects must have at least one measurable typical or nodular PN
- Absolute neutrophil count ≥1.5×10^9/L, haemoglobin ≥9g/dL, and platelet count ≥100×10^9/L. Subject must be without growth factor support and platelet transfusion support 7 days before the screening assessment.
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2×upper limit of normal (ULN), total bilirubin ≤1.5×ULN except in the case of subjects with documented Gilbert's disease (≤2.5×ULN).
Who cannot join
- Evidence of malignant peripheral nerve sheath tumour.
- Clinically significant cardiovascular disease
- Prior malignancy (except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the subject had been disease free for ≥2 years or which would not have limited survival to <2 years) or other cancer requiring treatment with chemotherapy or radiation therapy.
- Subjects with the following ophthalmological findings/conditions:
- 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.
Trial locations and contacts
2 sites in 1 country.
About this study
Official title: 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)
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).
Read the detailed description
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
- Conditions
- Neurofibromatosis 1, Neurofibroma Plexiform
- Being tested
- Selumetinib
- Keywords
- Neurofibromatosis Type 1, Plexiform Neurofibromas, Phase 1, Selumetinib, Chinese, Paediatric, Adult
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelumetinibDrug
Also called Koselugo
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 group | Role | What they receive |
|---|---|---|
| Selumetinib | Experimental | 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. Receives: Drug: Selumetinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: 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
Measured: 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
Measured: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
Secondary outcomes (14)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: First patient first dose until up to 2 years after last subject dosed
Measures of pain via FLACC scale
Measured: 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)
Measured: First patient first dose until up to 2 years after last subject dosed
Measures health-related quality of life (HRQoL) via PlexiQoL (adult cohort)
Measured: First patient first dose until up to 2 years after last subject dosed
Measures of pain via Faces Pain Scale (revised)
Measured: First patient first dose until up to 2 years after last subject dosed
Measures of pain via NRS-11
Measured: First patient first dose until up to 2 years after last subject dosed
Measures of pain via PII
Measured: First patient first dose until up to 2 years after last subject dosed
Measures of pain via Pain Medication Survey
Measured: First patient first dose until up to 2 years after last subject dosed
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2020
- Study start16 Dec 2020
- Primary completion16 Aug 2022
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Neurofibromatosis 1Studies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Merck Sharp & Dohme LLC
- Responsible party
- Sponsor
- Investigators
- Qingfeng Li, Principal investigator, Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04590235
- Sponsor's protocol ID
- D1346C00011
- Status last verified
- Aug 2026
- First submitted
- 28 Jul 2020
- Last updated
- 18 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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.
Publications
Zhang X, Sui W, Zheng H, Chen J, Yuan X. Durable responses to long-term selumetinib in Chinese pediatric NF1 patients with inoperable plexiform neurofibromas. Front Pharmacol. 2026 Jun 18;17:1855171. doi: 10.3389/fphar.2026.1855171. eCollection 2026.
PubMed 42394972
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT04590235
- Acronym / sponsor ID
- D1346C00011
- Conditions
- Neurofibromatosis 1, Neurofibroma Plexiform
- Keywords
- Neurofibromatosis Type 1, Plexiform Neurofibromas, Phase 1, Selumetinib, Chinese, Paediatric, Adult
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 32 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Collaborators
- Merck Sharp & Dohme LLC
- Study start
- 16 Dec 2020 (actual)
- Primary completion
- 16 Aug 2022 (actual)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 19 Oct 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Selumetinib | Experimental | Drug: SelumetinibAll 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. |
Who may qualify
- Ages
- 3 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Paediatric cohort: Chinese subjects ≥3 years and <18 years of age
- Adult cohort: Chinese subjects ≥18 years of age at the time of study enrollment
- Subjects must be diagnosed with (i) NF1 as per NIH Consensus Development Conference Statement and（ii) PN is defined as a neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches. (iii) inoperable PN
- Subjects must have at least one measurable typical or nodular PN
- Absolute neutrophil count ≥1.5×10^9/L, haemoglobin ≥9g/dL, and platelet count ≥100×10^9/L. Subject must be without growth factor support and platelet transfusion support 7 days before the screening assessment.
Key exclusion criteria (5 listed)
- Evidence of malignant peripheral nerve sheath tumour.
- Clinically significant cardiovascular disease
- Prior malignancy (except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the subject had been disease free for ≥2 years or which would not have limited survival to <2 years) or other cancer requiring treatment with chemotherapy or radiation therapy.
- Subjects with the following ophthalmological findings/conditions:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SelumetinibAlso: KoselugoAll 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. | Drug | Selumetinib |
Primary outcome
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.
* 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.
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
Time frame: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
AUC0-t after single dose and multiple doses administration
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
Time frame: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
Cmax after single dose and multiple doses administration
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
Time frame: From the first consent patient first dose to last patient steady state PK collection. Expected duration is approximately 1 year.
t1/2 after single dose and multiple doses administration
14 secondary outcomes
objective response rate (ORR) of selumetinib in Chinese paediatric and adult subjects with Neurofibromatosis Type 1 and inoperable Plexiform Neurofibromas
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
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
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
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
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2026.
- Derived from this study: PubMed 42394972Zhang X, Sui W, Zheng H, Chen J, Yuan X. Durable responses to long-term selumetinib in Chinese pediatric NF1 patients with inoperable plexiform neurofibromas. Front Pharmacol. 2026 Jun 18;17:1855171. doi: 10.3389/fphar.2026.1855171. eCollection 2026.
- Clinical Study Protocol RedactedLink provided by the sponsor.
- SAP RedactedLink provided by the sponsor.
- CSR Synopsis RedactedLink provided by the sponsor.
- Study ResultsLink provided by the sponsor.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Qingfeng Li principal investigator
Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Selumetinib in people with Neurofibromatosis 1 and Neurofibroma Plexiform, run by AstraZeneca at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 18 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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.). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04590235, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Aug 2026.