Neurofibromatosis Type 1Phase 1NCT05101148
Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
A Phase 1 study testing Selumetinib in people with Neurofibromatosis Type 1, run by AstraZeneca at 9 sites in 4 countries.
Trial summary
This study in adolescent participants with NF1 who have inoperable PN is designed to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI tolerability when selumetinib is dosed under fed and fasted conditions; and potentially, to confirm an appropriate dosing… Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Neurofibromatosis Type 1
- What is being tested?
- Selumetinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 9 sitesin 4 countries
- Study dates
- 21 Jul 2021to 28 Apr 2028
- 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 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female participants aged ≥ 12 to < 18 years at the time of signing the informed consent.
- All study participants must be diagnosed with (i) NF1 per NIH Consensus Development Conference Statement and (ii) inoperable PN. In addition to PN, participants must have at least 1 other diagnostic criterion for NF1 as defined in protocol.
- Participants must require treatment for NF1 and inoperable PN due to actual symptoms or because of the potential to develop significant clinical complications, as judged by the Investigator, as defined in the protocol.
- Participants who have had prior treatment with any MEKi (including selumetinib) may be considered for inclusion in this study.
- Participants must have a BSA ≥ 1.3 and ≤ 2.5 m2
Who cannot join
- Evidence or suspicion of optic glioma, malignant glioma, MPNST, or other cancer requiring treatment with chemotherapy or radiation therapy
- Prior malignancy requiring active treatment (except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the participant had been disease free for ≥ 2 years or which would not have limited survival to < 2 years).
- A life-threatening illness, medical condition, organ system dysfunction of laboratory finding which, in the Investigator's opinion, could compromise the participant's safety, interfere with the absorption or metabolism of selumetinib, or put the study outcomes at undue risk.
- Participants with clinically significant cardiovascular disease as listed in the protocol.
- Liver function tests: bilirubin > 1.5 × the ULN for age (with the exception of those with Gilbert syndrome) or AST/ALT > 2 × upper limit of normal.
- Renal Function: Creatinine clearance or radioisotope glomerular filtration rate < 30 mL/min/1.73 m2 or a serum creatinine > 1.2 mg/dL (for participants aged between 12 and 15 years) or > 1.5 mg/dL for participants aged > 15 years).
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- Participants with abnormal ophthalmological findings/conditions as listed in the protocol.
- Have any unresolved chronic toxicity, associated with previous therapy for NF1-PN: Gastrointestinal toxicity of CTCAE Grade 1 or higher; Have any other unresolved chronic toxicity with CTCAE Grade ≥ 2, except hair changes (such as alopecia or hair lightening).
- Participants who have previously been treated with a MEKi (including selumetinib) and either discontinued treatment or required a dose reduction due to toxicity
- Have had recent major surgery within a minimum of 4 weeks prior to starting study intervention, with the exception of surgical placement for vascular access. Have planned major surgery during the treatment period.
- Any multivitamin containing vitamin E must be stopped at least 7 days prior to initiation of selumetinib.
Trial locations and contacts
9 sites in 4 countries.
9 sites in 4 countries
Poland3 sites
Research Site
Bydgoszcz, 85-094
Research Site
Gdansk, 80-952
Research Site
Warsaw, 02-091
United States2 sites
Research Site
Akron, Ohio, 44308
Research Site
Rochester, Minnesota, 55905
About this study
Official title: A Phase I, Single-Arm, Sequential Study to Evaluate the Effect of Food on the Gastrointestinal Tolerability and Pharmacokinetics of Selumetinib After Multiple Doses in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
This study in adolescent participants with NF1 who have inoperable PN is designed to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI tolerability when selumetinib is dosed under fed and fasted conditions; and potentially, to confirm an appropriate dosing recommendation of selumetinib with a low fat meal that maintains efficacy with acceptable safety. These results may support labelling statements with regard to posology and food.
Read the detailed description
Selumetinib is approved by the FDA for the treatment of paediatric patients 2 years of age and older with NF1 who have symptomatic, inoperable PN.
The efficacy of selumetinib in the treatment of NF1 related inoperable PN in paediatric participants was demonstrated in the SPRINT study in which selumetinib was taken at 25 mg/m2 bid under fasted conditions (fast for 2 hours before each dose and 1 hour after each dose). Safety data from this study showed that selumetinib has a generally predictable and manageable safety profile in this population. Gastrointestinal AEs were commonly reported but were predominantly mild/moderate in severity, predictable, and generally did not affect the ability of participants to remain on treatment. However, GI AEs in this paediatric study were reported at a higher frequency than that reported in an analysis of selumetinib in adult oncology participants. Dosing in a fed state is known to reduce GI tolerability for some oncology drugs; such a dosing regimen has an added benefit of improving compliance and adherence to dosing posology.
Food-effect studies conducted in healthy participants (Study D1532C00069) and adult participants with cancer (Study D1532C00020) show that consumption of a high fat meal reduces absorption of selumetinib: in healthy participants Cmax was reduced by 50% and AUC by 16%; and in participants with cancer Cmax was reduced by 62% and AUC by 19%. A further study in adult healthy participants (D1532C00089) found that a low fat, low calorie meal reduces the rate of absorption (Cmax reduced by 65% and tmax delayed by 2.5 hours) and the extent of absorption (AUC reduced by 38%) after a single dose of selumetinib. The finding that a low fat meal had a greater impact on exposure than a high fat meal is unusual and without a clear explanation although in vitro dissolution data suggest that the capsule shell disintegration may be impacted by food.
The study is designed to evaluate the steady state systemic exposure and safety (especially GI tolerability) of selumetinib 25 mg/m2 bid given with a low fat meal versus the same dose given in a fasted state. A third treatment period (T3) will be initiated if there is a significant reduction in exposure (AUC0-12, SS between T2 vs T1) when selumetinib is given with a low fat meal compared with a fasted state; T3 will evaluate the PK and safety of an adjusted dose of selumetinib when given with a low fat meal. The recommendation as to whether to initiate T3, and the dose to be used in T3, will be made by a DRC. This recommendation will be reviewed with FDA before dosing in T3 is initiated.
Approximately 20 participants will be enrolled to achieve 16 evaluable participants completing T2. An evaluable participant is defined as having received study treatment and provided the last required PK sample in T2.
A Data Review Committee(DRC) will review the PK and safety data from T1 and T2 and determine whether T3 is required. If T3 is required the DRC will select the dose to be used. The recommendation will be reviewed with FDA before dosing in T3 is initiated.
- Conditions
- Neurofibromatosis Type 1
- Being tested
- Selumetinib
- Keywords
- Neurofibromatosis Type 1
- Medical subject headings
- Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelumetinibDrug
The dosing regimen for selumetinib (25 mg/m2 bid) will be based on BSA, during T1 and T2 the dose will be 25mg/m2, consistent with the approved prescribing information in the US. If T3 is required, appropriate dose of selumetinib will be defined by Data Review Committee for T3.
| Study group | Role | What they receive |
|---|---|---|
| selumetinib single arm | Experimental | This is a sequential study consisting of a screening period lasting up to 28 days, a 28 day (1 cycle) treatment period (T1) in a fed state, a 7 day washout period, a further 1 cycle treatment period (T2) in a fasted state and an extension to T2 until results from the primary analysis are available. During Treatment Period 1 and 2 all participants will receive selumetinib (25 mg/m2 bid). If a third treatment period (T3) is required, participants will enter a 7 day washout period followed by a treatment period in a fed state at an adjusted dose for 3 cycles. Receives: Drug: Selumetinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Selumetinib area under the plasma concentration-time curve from zero to 12 hours post-dose (AUC0-12)
To compare the AUC0-12, SS of the fed (same dose and dose adjustment if necessary) versus fasted state
Measured: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
Gastrointestinal Adverse Events graded by CTCAE Ver 5.0 (Grade 1 to 5)
To investigate the gastrointestinal toxicities of selumetinib capsules after multiple doses
Measured: from screening until 30 days after last dose
Assessing change of Gastrointestinal toxicity diary: Modified Bristol Stool Form Scale for Children (mBSFS-C)
To investigate the gastrointestinal toxicities of selumetinib capsules after multiple doses
Measured: At screening (at least 14 days), Cycle 1 of each treatment period 1, 2 and 3 (1 cycle is 28 days)
Assessing change of Gastrointestinal toxicity diary: Nausea and Vomiting Symptom Rating Scale (adapted from the Children's Cancer and Leukaemia Group)
To investigate the gastrointestinal toxicities of selumetinib capsules after multiple doses
Measured: At screening (at least 14 days), Cycle 1 of each treatment period 1, 2 and 3 (1 cycle is 28 days)
Number of patients who take each gastrointestinal medication
Collecting gastrointestinal concomitant medications taken including, but not restricted to, medications used to treat diarrhoea, nausea and vomiting.
Measured: From screening until 30 days after last dose
Proportion of patients who take each gastrointestinal medication
Collecting gastrointestinal concomitant medications taken including, but not restricted to, medications used to treat diarrhoea, nausea and vomiting.
Measured: From screening until 30 days after last dose
Secondary outcomes (5)
Adverse events(AEs) graded by CTCAE Version 5.0
To further assess the safety and tolerability of selumetinib capsules
Measured: From screening until 30 days after last dose
Maximum Peak plasma concentration (Cmax) of selumetinib and N-desmethyl selumetinib
To further evaluate the PK of selumetinib and N-desmethyl selumetinib metabolite after multiple doses under fed conditions, compared to fasted conditions
Measured: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
Area under the concentration-time curve from time zero to time of last measurable concentration (AUClast) of selumetinib and N-desmethyl selumetinib
To further evaluate the PK of selumetinib and N-desmethyl selumetinib metabolite after multiple doses under fed conditions, compared to fasted conditions
Measured: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
Time to maximum concentration (tmax) of selumetinib and N-desmethyl selumetinib
To further evaluate the PK of selumetinib and N-desmethyl selumetinib metabolite after multiple doses under fed conditions, compared to fasted conditions
Measured: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
Time to last measurable concentration (tlast) of selumetinib and N-desmethyl selumetinib
To further evaluate the PK of selumetinib and N-desmethyl selumetinib metabolite after multiple doses under fed conditions, compared to fasted conditions
Measured: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2021
- Study start21 Jul 2021
- Primary completion6 Apr 2022
- Study completion28 Apr 2028 (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 Type 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
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Merck Sharp & Dohme LLC
- Responsible party
- Sponsor
- Investigators
- Study physician Study physician, MD, Study director, AstraZeneca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05101148
- Sponsor's protocol ID
- D1346C00015
- Other IDs
- 2024-517216-29-00, 2020-005648-52
- Status last verified
- Aug 2026
- First submitted
- 16 Jun 2021
- Last updated
- 20 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. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Publications
Viskochil D, Wysocki M, Learoyd M, Sun P, So K, Evans A, Lai F, Hernandez HS. Effect of food on selumetinib pharmacokinetics and gastrointestinal tolerability in adolescents with neurofibromatosis type 1-related plexiform neurofibromas. Neurooncol Adv. 2024 Mar 16;6(1):vdae036. doi: 10.1093/noajnl/vdae036. eCollection 2024 Jan-Dec.
PubMed 38721358
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Single-Arm, Sequential Study to Evaluate the Effect of Food on the Gastrointestinal Tolerability and Pharmacokinetics of Selumetinib After Multiple Doses in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
- ClinicalTrials.gov ID
- NCT05101148
- Acronym / sponsor ID
- D1346C00015
- Conditions
- Neurofibromatosis Type 1
- Keywords
- Neurofibromatosis Type 1
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 24 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Collaborators
- Merck Sharp & Dohme LLC
- Study start
- 21 Jul 2021 (actual)
- Primary completion
- 6 Apr 2022 (actual)
- Study completion
- 28 Apr 2028 (estimated)
- First posted
- 1 Nov 2021 (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.
This study in adolescent participants with NF1 who have inoperable PN is designed to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI tolerability when selumetinib is dosed under fed and fasted conditions; and potentially, to confirm an appropriate dosing recommendation of selumetinib with a low fat meal that maintains efficacy with acceptable safety. These results may support labelling statements with regard to posology and food.
Detailed description
Selumetinib is approved by the FDA for the treatment of paediatric patients 2 years of age and older with NF1 who have symptomatic, inoperable PN.
The efficacy of selumetinib in the treatment of NF1 related inoperable PN in paediatric participants was demonstrated in the SPRINT study in which selumetinib was taken at 25 mg/m2 bid under fasted conditions (fast for 2 hours before each dose and 1 hour after each dose). Safety data from this study showed that selumetinib has a generally predictable and manageable safety profile in this population. Gastrointestinal AEs were commonly reported but were predominantly mild/moderate in severity, predictable, and generally did not affect the ability of participants to remain on treatment. However, GI AEs in this paediatric study were reported at a higher frequency than that reported in an analysis of selumetinib in adult oncology participants. Dosing in a fed state is known to reduce GI tolerability for some oncology drugs; such a dosing regimen has an added benefit of improving compliance and adherence to dosing posology.
Food-effect studies conducted in healthy participants (Study D1532C00069) and adult participants with cancer (Study D1532C00020) show that consumption of a high fat meal reduces absorption of selumetinib: in healthy participants Cmax was reduced by 50% and AUC by 16%; and in participants with cancer Cmax was reduced by 62% and AUC by 19%. A further study in adult healthy participants (D1532C00089) found that a low fat, low calorie meal reduces the rate of absorption (Cmax reduced by 65% and tmax delayed by 2.5 hours) and the extent of absorption (AUC reduced by 38%) after a single dose of selumetinib. The finding that a low fat meal had a greater impact on exposure than a high fat meal is unusual and without a clear explanation although in vitro dissolution data suggest that the capsule shell disintegration may be impacted by food.
The study is designed to evaluate the steady state systemic exposure and safety (especially GI tolerability) of selumetinib 25 mg/m2 bid given with a low fat meal versus the same dose given in a fasted state. A third treatment period (T3) will be initiated if there is a significant reduction in exposure (AUC0-12, SS between T2 vs T1) when selumetinib is given with a low fat meal compared with a fasted state; T3 will evaluate the PK and safety of an adjusted dose of selumetinib when given with a low fat meal. The recommendation as to whether to initiate T3, and the dose to be used in T3, will be made by a DRC. This recommendation will be reviewed with FDA before dosing in T3 is initiated.
Approximately 20 participants will be enrolled to achieve 16 evaluable participants completing T2. An evaluable participant is defined as having received study treatment and provided the last required PK sample in T2.
A Data Review Committee(DRC) will review the PK and safety data from T1 and T2 and determine whether T3 is required. If T3 is required the DRC will select the dose to be used. The recommendation will be reviewed with FDA before dosing in T3 is initiated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| selumetinib single arm | Experimental | Drug: SelumetinibThis is a sequential study consisting of a screening period lasting up to 28 days, a 28 day (1 cycle) treatment period (T1) in a fed state, a 7 day washout period, a further 1 cycle treatment period (T2) in a fasted state and an extension to T2 until results from the primary analysis are available. During Treatment Period 1 and 2 all participants will receive selumetinib (25 mg/m2 bid). If a third treatment period (T3) is required, participants will enter a 7 day washout period followed by a treatment period in a fed state at an adjusted dose for 3 cycles. |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Male and female participants aged ≥ 12 to < 18 years at the time of signing the informed consent.
- All study participants must be diagnosed with (i) NF1 per NIH Consensus Development Conference Statement and (ii) inoperable PN. In addition to PN, participants must have at least 1 other diagnostic criterion for NF1 as defined in protocol.
- Participants must require treatment for NF1 and inoperable PN due to actual symptoms or because of the potential to develop significant clinical complications, as judged by the Investigator, as defined in the protocol.
- Participants who have had prior treatment with any MEKi (including selumetinib) may be considered for inclusion in this study.
- Participants must have a BSA ≥ 1.3 and ≤ 2.5 m2
Key exclusion criteria (11 listed)
- Evidence or suspicion of optic glioma, malignant glioma, MPNST, or other cancer requiring treatment with chemotherapy or radiation therapy
- Prior malignancy requiring active treatment (except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the participant had been disease free for ≥ 2 years or which would not have limited survival to < 2 years).
- A life-threatening illness, medical condition, organ system dysfunction of laboratory finding which, in the Investigator's opinion, could compromise the participant's safety, interfere with the absorption or metabolism of selumetinib, or put the study outcomes at undue risk.
- Participants with clinically significant cardiovascular disease as listed in the protocol.
- Liver function tests: bilirubin > 1.5 × the ULN for age (with the exception of those with Gilbert syndrome) or AST/ALT > 2 × upper limit of normal.
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 |
|---|---|---|
| SelumetinibThe dosing regimen for selumetinib (25 mg/m2 bid) will be based on BSA, during T1 and T2 the dose will be 25mg/m2, consistent with the approved prescribing information in the US. If T3 is required, appropriate dose of selumetinib will be defined by Data Review Committee for T3. | Drug | selumetinib single arm |
Primary outcome
Selumetinib area under the plasma concentration-time curve from zero to 12 hours post-dose (AUC0-12)
Time frame: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
To compare the AUC0-12, SS of the fed (same dose and dose adjustment if necessary) versus fasted state
Gastrointestinal Adverse Events graded by CTCAE Ver 5.0 (Grade 1 to 5)
Time frame: from screening until 30 days after last dose
To investigate the gastrointestinal toxicities of selumetinib capsules after multiple doses
Assessing change of Gastrointestinal toxicity diary: Modified Bristol Stool Form Scale for Children (mBSFS-C)
Time frame: At screening (at least 14 days), Cycle 1 of each treatment period 1, 2 and 3 (1 cycle is 28 days)
To investigate the gastrointestinal toxicities of selumetinib capsules after multiple doses
Assessing change of Gastrointestinal toxicity diary: Nausea and Vomiting Symptom Rating Scale (adapted from the Children's Cancer and Leukaemia Group)
Time frame: At screening (at least 14 days), Cycle 1 of each treatment period 1, 2 and 3 (1 cycle is 28 days)
To investigate the gastrointestinal toxicities of selumetinib capsules after multiple doses
Number of patients who take each gastrointestinal medication
Time frame: From screening until 30 days after last dose
Collecting gastrointestinal concomitant medications taken including, but not restricted to, medications used to treat diarrhoea, nausea and vomiting.
Proportion of patients who take each gastrointestinal medication
Time frame: From screening until 30 days after last dose
Collecting gastrointestinal concomitant medications taken including, but not restricted to, medications used to treat diarrhoea, nausea and vomiting.
5 secondary outcomes
Adverse events(AEs) graded by CTCAE Version 5.0
Time frame: From screening until 30 days after last dose
Maximum Peak plasma concentration (Cmax) of selumetinib and N-desmethyl selumetinib
Time frame: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
Area under the concentration-time curve from time zero to time of last measurable concentration (AUClast) of selumetinib and N-desmethyl selumetinib
Time frame: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
Time to maximum concentration (tmax) of selumetinib and N-desmethyl selumetinib
Time frame: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
Time to last measurable concentration (tlast) of selumetinib and N-desmethyl selumetinib
Time frame: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.
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 20 Aug 2026.
- Derived from this study: PubMed 38721358Viskochil D, Wysocki M, Learoyd M, Sun P, So K, Evans A, Lai F, Hernandez HS. Effect of food on selumetinib pharmacokinetics and gastrointestinal tolerability in adolescents with neurofibromatosis type 1-related plexiform neurofibromas. Neurooncol Adv. 2024 Mar 16;6(1):vdae036. doi: 10.1093/noajnl/vdae036. eCollection 2024 Jan-Dec.
- CSR SynopsisLink 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. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
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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.
Study physician Study physician, MD study director
AstraZeneca
9 sites in 4 countries: 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 Type 1, run by AstraZeneca at 9 sites in 4 countries.
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 5 inclusion and 11 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 20 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 9 sites in Poland, United States, Russia, Spain. 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?
Selumetinib area under the plasma concentration-time curve from zero to 12 hours post-dose (AUC0-12) (time frame: At pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose (Cycle 1 Day 8 of each treatment period 1, 2 and 3); Each treatment period 1 and 2 has 1 cycle (Each cycle is 28 days). If needed, treatment period 3 will be started approximately 5 cycles later.). The study lists 6 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 NCT05101148, 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 20 Aug 2026.