Trial · NCT05309668
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
NCT05309668, Phase 1, Phase 2, Active not recruiting, AstraZeneca; results first posted 2025-05-14
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN. Trials, record NCT05309668. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05309668
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
- NCT number
- NCT05309668
- Status
- Active not recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 36 participants, actual
- Lead sponsor
- AstraZeneca
- Conditions
- Neurofibromatosis Type 1
- Interventions
- Selumetinib granule formulation, Selumetinib capsule formulation
- First posted
- 2022-04-04
- Last updated
- 2026-08-13
- Other registries' IDs
- eudract:2020-005608-20
- Results first posted
- 2025-05-14
- Limitations and caveats
The outcome measure Primary Outcome: Selumetininb AUC0-12₂ Derived After Single Dose Administration is one of two co-primary endpoints. The second co-primary endpoint - Adverse Events Graded by CTCAE Version 5.0 - is still ongoing. As a result, the study has not yet reached the Primary Completion Date at the time these results were submitted. Full results, including data for the second co-primary endpoint, will be submitted upon completion of the study.
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Selumetinib AUC0-12 Derived After Single Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8 and 10-12 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1) (each cycle is 28 days) | To determine the pharmacokinetics of selumetinib after administration of the selumetinib granule formulation |
| Primary | Adverse Events Graded by CTCAE Ver 5.0 | from screening until 30 days after last dose | To assess the safety and tolerability of the selumetinib granule formulation. |
| Secondary | Palatability Using the Parent-reported Observer Palatability Questionnaire | Twice daily (morning and evening), from the first day of study treatment (Cycle 1 Day 1) for one week, from Cycle 7 Day 1 for one week (each cycle is 28 days) | To assess the palatability of the selumetinib granule formulation |
| Secondary | Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | To further evaluate the PK of the granule formulation. |
| Secondary | Selumetinib and N-desmethyl Selumetinib Cmax Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | To further evaluate the PK of the granule formulation. |
| Secondary | Selumetinib and N-desmethyl Selumetinib AUC0-6 Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4 and 6 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4 and 6 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | To further evaluate the PK of the granule formulation. |
| Secondary | Objective Response Rate | At screening, at week 17 (Cycle5 Day1), week 33 (Cycle9 Day1), week 49 (Cycle13 Day1), week 73 (Cycle19 Day1) and week 97 (Cycle25 Day1), end of treatment (each cycle is 28 days) | To evaluate the efficacy of the selumetinib granule formulation by assessment of Objective Response Rate |
| Secondary | Selumetinib and N-desmethyl Selumetinib AUClast Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | To further evaluate the PK of the granule formulation. |
| Secondary | Selumetinib and N-desmethyl Selumetinib Tmax Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | To further evaluate the PK of the granule formulation. |
| Secondary | Selumetinib and N-desmethyl Selumetinib Tlast Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | To further evaluate the PK of the granule formulation. |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Selumetinib AUC0-12 Derived After Single Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8 and 10-12 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1) (each cycle is 28 days) | Pharmacokinetic analysis set was used for analysis. Japan Cohort was not considered for the primary endpoint analysis. Some records were excluded for given visits and analytes due to missing concentration data, issues taking the medication and usage of prohibited medications. | h*ng/mL | GEOMETRIC_MEAN |
| PRIMARY | Adverse Events Graded by CTCAE Ver 5.0 | from screening until 30 days after last dose | |||
| SECONDARY | Palatability Using the Parent-reported Observer Palatability Questionnaire | Twice daily (morning and evening), from the first day of study treatment (Cycle 1 Day 1) for one week, from Cycle 7 Day 1 for one week (each cycle is 28 days) | |||
| SECONDARY | Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | Pharmacokinetic analysis set was used for analysis. Some records were excluded for given visits and analytes due to missing concentration data, issues taking the medication and usage of prohibited medications. | h*ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Selumetinib and N-desmethyl Selumetinib Cmax Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | Pharmacokinetic analysis set was used for analysis. Some records were excluded for given visits and analytes due to missing concentration data, issues taking the medication and usage of prohibited medications. | ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Selumetinib and N-desmethyl Selumetinib AUC0-6 Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4 and 6 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4 and 6 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | Pharmacokinetic analysis set was used for analysis. Some records were excluded for given visits and analytes due to missing concentration data, issues taking the medication and usage of prohibited medications. | h*ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Objective Response Rate | At screening, at week 17 (Cycle5 Day1), week 33 (Cycle9 Day1), week 49 (Cycle13 Day1), week 73 (Cycle19 Day1) and week 97 (Cycle25 Day1), end of treatment (each cycle is 28 days) | |||
| SECONDARY | Selumetinib and N-desmethyl Selumetinib AUClast Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | Pharmacokinetic analysis set was used for analysis. Some records were excluded for given visits and analytes due to missing concentration data, issues taking the medication and usage of prohibited medications. | h*ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Selumetinib and N-desmethyl Selumetinib Tmax Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | Pharmacokinetic analysis set was used for analysis. Some records were excluded for given visits and analytes due to missing concentration data, issues taking the medication and usage of prohibited medications. | h | MEDIAN |
| SECONDARY | Selumetinib and N-desmethyl Selumetinib Tlast Derived After Single and Multiple Dose Administration | Pre-dose and 1, 2, 3, 4, 6, 8, 10-12 and 18-24 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1). Pre-dose, 1, 2, 3, 4, 6, 8, and 10-12 hours post-dose on Cycle 2 Day 1 (each cycle is 28 days) | Pharmacokinetic analysis set was used for analysis. Some records were excluded for given visits and analytes due to missing concentration data, issues taking the medication and usage of prohibited medications. | h | MEDIAN |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group299
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Selumetinib AUC0-12 Derived After Single Dose Administration | Global Cohort 1 | 1902 | 95% Confidence Interval 1647 to 2197 | h*ng/mL | |
| Selumetinib AUC0-12 Derived After Single Dose Administration | Global Cohort 2 | 1699 | 95% Confidence Interval 1436 to 2009 | h*ng/mL | |
| Selumetinib AUC0-12 Derived After Single Dose Administration | Total in Global | 1790 | 95% Confidence Interval 1609 to 1993 | h*ng/mL | |
| Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Global Cohort 1 | Cycle 1 Day 1: Selumetinib | 1902 | Geometric Coefficient of Variation 24.19 | h*ng/mL |
| Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Global Cohort 2 | Cycle 1 Day 1: Selumetinib | 1699 | Geometric Coefficient of Variation 31.02 | h*ng/mL |
| Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Total in Global | Cycle 1 Day 1: Selumetinib | 1790 | Geometric Coefficient of Variation 28.15 | h*ng/mL |
| Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Japan Cohort | Cycle 1 Day 1: Selumetinib | 1526 | Geometric Coefficient of Variation 25.09 | h*ng/mL |
| Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Global Cohort 1 | Cycle 1 Day 1: N-desmethyl selumetinib | 156.1 | Geometric Coefficient of Variation 25.76 | h*ng/mL |
| Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Global Cohort 2 | Cycle 1 Day 1: N-desmethyl selumetinib | 134.0 | Geometric Coefficient of Variation 34.21 | h*ng/mL |
| Selumetinib and N-desmethyl Selumetinib AUC0-12 Derived After Single and Multiple Dose Administration | Total in Global | Cycle 1 Day 1: N-desmethyl selumetinib | 144.6 | Geometric Coefficient of Variation 30.69 | h*ng/mL |
1 to 10 of 299Source ClinicalTrials.gov
See all 299 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Global Cohort 1 | 15 | |
| Overall Study | STARTED | Global Cohort 2 | 17 | |
| Overall Study | STARTED | Japan Cohort | 4 | |
| Overall Study | COMPLETED | Global Cohort 1 | 1 | |
| Overall Study | COMPLETED | Global Cohort 2 | 0 | |
| Overall Study | COMPLETED | Japan Cohort | 0 | |
| Overall Study | NOT COMPLETED | Global Cohort 1 | 14 | |
| Overall Study | NOT COMPLETED | Global Cohort 2 | 17 | |
| Overall Study | NOT COMPLETED | Japan Cohort | 4 |
Source ClinicalTrials.gov
Adverse events670
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain upper | Gastrointestinal disorders | Global Cohort 1 | 1 | 15 | 1 |
| other | Abdominal pain upper | Gastrointestinal disorders | Global Cohort 2 | 1 | 17 | 1 |
| other | Abdominal pain upper | Gastrointestinal disorders | Total in Global | 2 | 32 | 2 |
| other | Abdominal pain upper | Gastrointestinal disorders | Japan Cohort | 0 | 4 | 0 |
| other | Abdominal pain upper | Gastrointestinal disorders | Total in Study | 2 | 36 | 2 |
| other | Irritability | Psychiatric disorders | Global Cohort 1 | 1 | 15 | 1 |
| other | Irritability | Psychiatric disorders | Global Cohort 2 | 1 | 17 | 1 |
| other | Irritability | Psychiatric disorders | Total in Global | 2 | 32 | 2 |
| other | Irritability | Psychiatric disorders | Japan Cohort | 0 | 4 | 0 |
| other | Irritability | Psychiatric disorders | Total in Study | 2 | 36 | 2 |
1 to 10 of 670Source ClinicalTrials.gov
See all 670 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Global Cohort 1 | 0 | 15 |
| Total, all-cause mortality | Global Cohort 2 | 0 | 17 |
| Total, all-cause mortality | Total in Global | 0 | 32 |
| Total, all-cause mortality | Japan Cohort | 0 | 4 |
| Total, all-cause mortality | Total in Study | 0 | 36 |
| Total, other adverse events | Global Cohort 1 | 15 | 15 |
| Total, other adverse events | Global Cohort 2 | 17 | 17 |
| Total, other adverse events | Total in Global | 32 | 32 |
| Total, other adverse events | Japan Cohort | 4 | 4 |
| Total, other adverse events | Total in Study | 36 | 36 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 41592259 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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Read this trial from software.
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GET https://conductscience.org/api/trials/v1/NCT05309668/registered?page=2