Trial · NCT04924608
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
NCT04924608, Phase 3, Active not recruiting, AstraZeneca; results first posted 2025-10-20
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas. Trials, record NCT04924608. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04924608
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
- NCT number
- NCT04924608
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 145 participants, actual
- Lead sponsor
- AstraZeneca
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma (PN)
- Interventions
- Selumetinib, Placebo
- First posted
- 2021-06-14
- Last updated
- 2026-11-12
- Other registries' IDs
- eudract:2020-005607-39
- Results first posted
- 2025-10-20
Source ClinicalTrials.gov
Registered outcomes29
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN. | From first dose up until progression (if it occurs prior to the end of Cycle 16), or the last evaluable assessment up to and including the end of Cycle 16, excluding MRI during prolonged study intervention interruption (defined as interruption >= 28 days) | Objective response rate is defined as the proportion of participants who have a confirmed CR (defined as disappearance of the target PN, confirmed by a consecutive scan within 3 to 6 months after the first response) or confirmed PR (defined as a target PN volume decrease ≥ 20%, compared to baseline, confirmed by a consecutive scan within 3 to 6 months after the first response) by end of Cycle 16 as determined by ICR per REiNS criteria. Increase in the volume of the target PN by 20% or more compared to baseline or the time of best response after documenting a PR is considered as PD. |
| Secondary | (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Primary Analysis | Baseline and end of cycle 12 of study intervention | The PAINS-pNF (Pain Intensity Scale for Plexiform Neurofibroma) chronic target PN pain intensity measures the participants' experience of chronic PN-related pain intensity with score from 0 (no chronic tumor pain) to 10 (worst chronic tumor pain). Baseline PAINS-pNF chronic target PN pain score is defined as the average of the available daily PAINS-pNF chronic target PN pain scores in the screening perio, while for Cycle 12, it is defined as the average of the available daily scores for the 28-days cycle up to and including the last day assessment of cycle 12. The difference of the means in the change from baseline at Cycle 12 between selumetinib and placebo in participants with a PAINS-pNF chronic target PN pain score of ≥ 3 at baseline is presented. |
| Secondary | (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Supplemental Analysis | Baseline and end of cycle 12 of study intervention | The PAINS-pNF (Pain Intensity Scale for Plexiform Neurofibroma) chronic target PN pain intensity measures the participants' experience of chronic PN-related pain intensity with score from 0 (no chronic tumor pain) to 10 (worst chronic tumor pain). Baseline PAINS-pNF chronic target PN pain score is defined as the average of the available daily PAINS-pNF chronic target PN pain scores in the screening period, while for Cycle 12, it is defined as the average of the available daily scores for the 28-days cycle up to and including the last day assessment of cycle 12. The difference of the means in the change from baseline at Cycle 12 between selumetinib and placebo participants is presented. |
| Secondary | (Second Key Secondary Endpoint) The Difference of the Means in the Change From Baseline in PlexiQoL Total Score at Cycle 12 | Baseline and end of Cycle 12 of study intervention | PlexiQoL (Plexiform Neurofibroma Quality of Life scale) is a patient-derived QoL measure specific to adults with NF1-associated PNs. It assesses the impact of PNs on patients' ability to fulfil their human needs. The measure consists of 18 dichotomous items with 0 =Not True and 1 = True. PlexiQoL total scores were calculated by the sum of all items to a maximum of 18, with lower scores indicating better quality of life. The change from baseline to the end of each cycle in PlexiQoL total score was derived as the PlexiQoL total score at the cycle 12 minus baseline PlexiQoL total score and presented. |
| Secondary | Confirmed Partial and Complete Response Rate (ORR) Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | Objective response rate is defined as the proportion of participants who have a confirmed CR (defined as disappearance of the target PN, confirmed by a consecutive scan within 3 to 6 months after the first response) or confirmed PR (defined as a target PN volume decrease ≥ 20%, compared to baseline, confirmed by a consecutive scan within 3 to 6 months after the first response) by end of study (final DCO) as determined by ICR per REiNS criteria. Increase in the volume of the target PN by 20% or more compared to baseline or the time of best response after documenting a PR is considered as PD. |
| Secondary | Duration of Response by Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | The duration of response is defined as the time from the date of first documented response (which is subsequently confirmed) until date of documented progression by ICR per REiNS criteria or death due to any cause. Duration of response is derived using while on-treatment MRI volumetric assessments. The percentage of participants with event are provided. |
| Secondary | Progression Free Survival (PFS) Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | Progression free survival (PFS) is the time from randomisation date until the date of progression by ICR per REiNS criteria or death due to any cause, or last evaluable MRI assessment for subjects that do not progress. For subjects that progress after two or more consecutive missed MRI assessments, the subject is censored at the time of the latest evaluable MRI assessment prior to the missed visits. The percentage of participants with event are provided. |
| Secondary | Time to Progression (TTP) Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | Time to progression (TTP) is the time from randomisation date until the date of first documented objective disease progression by ICR per REiNS criteria, or last evaluable MRI assessment for subjects that do not progress. For subjects that progress after two or more consecutive missed MRI assessments, the subject is censored at the time of the latest evaluable MRI assessment prior to the missed visits. The percentage of participants with event are provided. |
| Secondary | Time to Response (TTR) Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) (Single Arm) | From 1st dose until progression (if before final DCO) or last evaluable assessment up to the last assessment prior to final DCO (up to 3y 3mo from 1st dose), excluding MRI during prolonged study intervention interruption (interruption >=28 days) | Time to response (TTR) is the time from randomisation date until the date of first documented objective partial and complete response (which is subsequently confirmed, cCR or cPR) as determined by ICR per REiNS criteria. Only participant who have achieved a cCR or a cPR are evaluated for TTR. |
| Secondary | Best Percentage Change From Baseline (PCHG) in Target PN During Randomized Period | From randomisation date until end of cycle 12 of study intervention | The best change in volume is the largest decrease from baseline or the smallest increase from baseline in the absence of a reduction and includes all on-treatment MRI volumetric assessments by the end of Cycle 12 Day 28 visit and prior to the earliest of death in the absence of progression, any evidence of progression, or the last evaluable REiNS assessment if the patient has not died or progressed. |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 registered outcomesOutcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN. | From first dose up until progression (if it occurs prior to the end of Cycle 16), or the last evaluable assessment up to and including the end of Cycle 16, excluding MRI during prolonged study intervention interruption (defined as interruption >= 28 days) | Full analysis set - All patients who are randomized to study intervention | Percentage | NUMBER |
| SECONDARY | (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Primary Analysis | Baseline and end of cycle 12 of study intervention | Pain full analysis set - subjects randomised to study intervention with a baseline PAINS-pNF chronic target PN pain intensity score \>= 3. | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Supplemental Analysis | Baseline and end of cycle 12 of study intervention | Full analysis set - subjects randomised to study intervention | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | (Second Key Secondary Endpoint) The Difference of the Means in the Change From Baseline in PlexiQoL Total Score at Cycle 12 | Baseline and end of Cycle 12 of study intervention | Full analysis set - subjects randomised to study intervention | Scores on a scale | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN. | Selumetinib 25 mg/m2 | 19.7 | 95% Confidence Interval 11.2 to 30.9 | Percentage | |
Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN. | Placebo | 5.4 | 95% Confidence Interval 1.5 to 13.3 | Percentage | |
| (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Primary Analysis | Selumetinib 25 mg/m2 | -2.0 | Standard Error 0.30 | Scores on a scale | |
| (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Primary Analysis | Placebo | -1.3 | Standard Error 0.29 | Scores on a scale | |
| (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Supplemental Analysis | Selumetinib 25 mg/m2 | -1.6 | Standard Error 0.22 | Scores on a scale | |
| (First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Supplemental Analysis | Placebo | -0.9 | Standard Error 0.21 | Scores on a scale | |
| (Second Key Secondary Endpoint) The Difference of the Means in the Change From Baseline in PlexiQoL Total Score at Cycle 12 | Selumetinib 25 mg/m2 | -0.4 | Standard Error 0.45 | Scores on a scale | |
| (Second Key Secondary Endpoint) The Difference of the Means in the Change From Baseline in PlexiQoL Total Score at Cycle 12 | Placebo | -0.3 | Standard Error 0.44 | Scores on a scale |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Selumetinib 25 mg/m2 | 71 | |
| Overall Study | STARTED | Placebo | 74 | |
| Overall Study | COMPLETED | Selumetinib 25 mg/m2 | 0 | |
| Overall Study | COMPLETED | Placebo | 0 | |
| Overall Study | NOT COMPLETED | Selumetinib 25 mg/m2 | 71 | |
| Overall Study | NOT COMPLETED | Placebo | 74 |
Source ClinicalTrials.gov
Adverse events104
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 | Vomiting | Gastrointestinal disorders | Selumetinib 25 mg/m2 | 18 | 71 | 33 |
| other | Vomiting | Gastrointestinal disorders | Placebo | 6 | 74 | 7 |
| other | Fatigue | General disorders | Selumetinib 25 mg/m2 | 14 | 71 | 14 |
| other | Fatigue | General disorders | Placebo | 10 | 74 | 10 |
| other | Oedema peripheral | General disorders | Selumetinib 25 mg/m2 | 11 | 71 | 14 |
| other | Oedema peripheral | General disorders | Placebo | 1 | 74 | 1 |
| other | Pyrexia | General disorders | Selumetinib 25 mg/m2 | 5 | 71 | 6 |
| other | Pyrexia | General disorders | Placebo | 3 | 74 | 3 |
| other | Covid-19 | Infections and infestations | Selumetinib 25 mg/m2 | 11 | 71 | 12 |
| other | Covid-19 | Infections and infestations | Placebo | 15 | 74 | 16 |
1 to 10 of 104Source ClinicalTrials.gov
See all 104 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Selumetinib 25 mg/m2 | 0 | 71 |
| Total, all-cause mortality | Placebo | 0 | 74 |
| Total, other adverse events | Selumetinib 25 mg/m2 | 70 | 71 |
| Total, other adverse events | Placebo | 54 | 74 |
| Total, serious adverse events | Selumetinib 25 mg/m2 | 10 | 71 |
| Total, serious adverse events | Placebo | 9 | 74 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40473450 | 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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Selumetinib 25 mg/m2 vs Placebo: deaths from any cause | Trial comparisons |
| Selumetinib 25 mg/m2 vs Placebo: serious adverse events | Trial comparisons |
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04924608
GET https://conductscience.org/api/trials/v1/NCT04924608/registered?page=2