Trial · NCT03962543
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
NCT03962543, Phase 2, Active not recruiting, SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany; results first posted 2025-08-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas. Trials, record NCT03962543. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03962543
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
- NCT number
- NCT03962543
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 114 participants, actual
- Lead sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
- Conditions
- Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
- Interventions
- Mirdametinib (PD-0325901) oral capsule or dispersible tablet
- First posted
- 2019-05-24
- Last updated
- 2026-10-06
- Results first posted
- 2025-08-07
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Confirmed Objective Response Rate at the End of the Treatment Phase. | Up to 24 months | Response will be determined by a blinded centralized review of volumetric MRI. The confirmed objective response rate (complete or partial response) by the end of Treatment Phase (i.e., Cycle 24) is defined as the proportion of participants who have a confirmed ≥ 20% reduction in target tumor volume as compared to baseline as assessed by a BICR, and the response needs to be confirmed by BICR in a consecutive tumor assessment within 2 - 6 months. Partial response is defined as a ≥ 20% reduction in target tumor volume from baseline. Complete response is defined as the complete resolution of the target tumor. |
| Secondary | Percentage of Patients With Treatment-Emergent Adverse Events. | All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment, an average of 1 year and 10 months and up to 3 years and 10 months. | All adverse events were coded using MedDRA Version 24.0. Adverse events will be assessed according to toxicities graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. |
| Secondary | Duration of Response (DOR) for Participants Who Meet Criteria for Confirmed Objective Response. | Starting on the onset of confirmed objective response in the Treatment Phase and afterwards on the 15th day of every 4 cycles (each cycle is 28 days) until disease progression or death, whichever comes first, assessed up to approximately 3 years | Duration of response is defined as the time in months between the first instance of response that is subsequently confirmed, until the date of radiographic disease progression or death, whichever occurs first. For participants who enter the LTFU Phase, all MRI assessments in both the Treatment Phase and LTFU Phase will be used to determine duration of response. Participants without radiographic disease progression or death while on study will have their results censored to their most recent adequate (i.e. evaluable) tumor assessment date. |
| Secondary | Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The PedsQL consists of a 23-item core measure of global QOL that can be completed in approximately 5 minutes. There is a total score and four subscales: physical functioning, emotional functioning, social functioning and school/work functioning. Participants ≥ 5 years of age complete an age-appropriate self-report; and parents/guardians of children ages 2-17 complete a parent proxy report of the age-specific QOL. The recall period is 7 days. PedsQL items are answered on a Likert scale with responses ranging from 0 to 4 (where 0 means it is never a problem and 4 means it is almost always a problem). These items are then reverse scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. On this scale, higher scores indicate better outcomes. Overall scale scores are calculated as the mean of the scores for the questions in said scale (or of all questions for the total score). The change from baseline is modelled using a mixed-model for repeated measures. |
| Secondary | Change From Baseline in Pain as Measured by the Numeric Rating Scale-11 (NRS-11) at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The NRS-11 is a self-reported 11-point numerical scale that assesses pain severity. Participants ≥ 8 years of age are asked to select a number from 0 (no pain) to 10 (worst pain you can imagine) that best describes their worst pain. The recall period is 24 hours. The change from baseline is modelled using a mixed-model for repeated measures. |
| Secondary | Change From Baseline in Pain as Measured by the Pain Interference Index (PII) at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The PII assesses relevant aspects of one's life, including pain interference with activities, spending time with family/friends, mood, sleep and attention. Participants ≥ 6 years of age complete a self-report, and parents/guardians of children aged 6-17 complete a parent proxy report. The recall period is 24 hours. The PII consists of 6 questions, each asking the responder to select one number from 0 to 6 that best describes how their/their proxy's pain has impacted various items over the past 24 hours with 0 representing "Not at all" and 6 representing "Completely". The mean of the completed items is taken as the PII score for a single assessment. The PII score presented each visit will be taken as the average of the PII scores over the 7 consecutive days up to and including visit day, with no transformation applied. The change from baseline is modelled using a mixed-model for repeated measures. |
| Other | Change From Baseline in Localized Strength (Dynamometer) at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | Assessment of strength will be conducted only in the area affected by the target tumor. Measurements will be obtained using Medical Research Council (MRC) grading followed by quantitative assessments using the sponsor provided MicroFET2 dynamometer. Dynamometer assessment (in lbs) must be conducted in accordance with the study reference manual. For each participant, the sum of the dynamometer scores for the muscle groups tested will be taken as an overall score for each visit with higher scores indicating better localized strength. To assess the change from baseline at a visit, a participant will need assessments in which all the following categories, as assessed at baseline, match at that visit: side affected, position during assessment (sitting/supine/lateral decubitus), muscle group assessed. The change from baseline in localized strength measured by the sum of the dynamometer scores for the muscle groups tested will be assessed at Cycle 13. |
| Other | Change From Baseline in Range of Motion of PN-Associated Functional Impairment at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | If motor dysfunction or weakness is evident, range of motion of the affected areas and/or joints will be measured by a goniometer. |
| Other | Change From Baseline in Endurance at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | If airway or lower extremity motor dysfunction is evident, endurance will be measured by completion of a 6-minute walking test. |
| Other | Time to Response Defined as the Time Between First Dose and the First Date of Objective Response That is Subsequently Confirmed. | Up to 24 months | Response will be determined by a blinded centralized review of volumetric MRI. Time to response is defined as the time in months from the start of treatment to the date of first response that was subsequently confirmed. |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Confirmed Objective Response Rate at the End of the Treatment Phase. | Up to 24 months | The Full Analysis Set is defined as patients who received at least one dose of Mirdametinib. | percentage of patients | NUMBER |
| SECONDARY | Percentage of Patients With Treatment-Emergent Adverse Events. | All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment, an average of 1 year and 10 months and up to 3 years and 10 months. | The Full Analysis Set includes patients who received at least one dose of Mirdametinib. | percentage of patients | NUMBER |
| SECONDARY | Duration of Response (DOR) for Participants Who Meet Criteria for Confirmed Objective Response. | Starting on the onset of confirmed objective response in the Treatment Phase and afterwards on the 15th day of every 4 cycles (each cycle is 28 days) until disease progression or death, whichever comes first, assessed up to approximately 3 years | The Full Analysis Set patients with confirmed objective response. | months | MEDIAN |
| SECONDARY | Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The Full Analysis Set patients who are eligible for the Peds-QL assessments at baseline. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Pain as Measured by the Numeric Rating Scale-11 (NRS-11) at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The Full Analysis Set patients eligible for the instrument at baseline. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Pain as Measured by the Pain Interference Index (PII) at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The Full Analysis Set patients eligible for the instrument at baseline. | units on a scale | LEAST_SQUARES_MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Localized Strength (Dynamometer) at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The Full Analysis Set patients eligible for this instrument at baseline. | lbs | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Range of Motion of PN-Associated Functional Impairment at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The Full Analysis Set patients who are eligible for this instrument at baseline. | degrees | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Endurance at Cycle 13. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months | The Full Analysis Set patients eligible for this instrument at baseline | meters | MEAN |
| OTHER_PRE_SPECIFIED | Time to Response Defined as the Time Between First Dose and the First Date of Objective Response That is Subsequently Confirmed. | Up to 24 months | The Full Analysis Set patients who achieved confirmed objective response. | months | MEAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group49
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Confirmed Objective Response Rate at the End of the Treatment Phase. | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 51.8 | 95% Confidence Interval 38.0 to 65.3 | percentage of patients | |
| Confirmed Objective Response Rate at the End of the Treatment Phase. | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 41.4 | 95% Confidence Interval 28.6 to 55.1 | percentage of patients | |
| Percentage of Patients With Treatment-Emergent Adverse Events. | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 100 | percentage of patients | ||
| Percentage of Patients With Treatment-Emergent Adverse Events. | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 100 | percentage of patients | ||
| Duration of Response (DOR) for Participants Who Meet Criteria for Confirmed Objective Response. | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | NA | 95% Confidence Interval NA to NA | months | |
| Duration of Response (DOR) for Participants Who Meet Criteria for Confirmed Objective Response. | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | NA | 95% Confidence Interval NA to NA | months | |
| Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version. | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report | Total Score | 4.01 | 95% Confidence Interval -0.74 to 8.76 | units on a scale |
| Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version. | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy | Total Score | 5.64 | 95% Confidence Interval 1.75 to 9.53 | units on a scale |
| Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version. | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report | Total Score | 3.94 | 95% Confidence Interval 0.70 to 7.17 | units on a scale |
| Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version. | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report | School/Work Functioning | -0.02 | 95% Confidence Interval -7.16 to 7.13 | units on a scale |
1 to 10 of 49Source ClinicalTrials.gov
See all 49 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Phase | STARTED | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 56 | |
| Treatment Phase | STARTED | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 58 | |
| Treatment Phase | COMPLETED | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 33 | |
| Treatment Phase | COMPLETED | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 31 | |
| Treatment Phase | NOT COMPLETED | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 23 | |
| Treatment Phase | NOT COMPLETED | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 27 | |
| Long Term Follow-Up Phase | STARTED | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 28 | |
| Long Term Follow-Up Phase | STARTED | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 26 | |
| Long Term Follow-Up Phase | COMPLETED | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 0 | |
| Long Term Follow-Up Phase | COMPLETED | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events154
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 | Dermatitis acneiform | Skin and subcutaneous tissue disorders | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 24 | 56 | |
| other | Dermatitis acneiform | Skin and subcutaneous tissue disorders | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 45 | 58 | |
| other | Dry skin | Skin and subcutaneous tissue disorders | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 10 | 56 | |
| other | Dry skin | Skin and subcutaneous tissue disorders | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 9 | 58 | |
| other | Alopecia | Skin and subcutaneous tissue disorders | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 8 | 56 | |
| other | Alopecia | Skin and subcutaneous tissue disorders | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 7 | 58 | |
| other | Rash | Skin and subcutaneous tissue disorders | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 11 | 56 | |
| other | Rash | Skin and subcutaneous tissue disorders | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 4 | 58 | |
| other | Pruritus | Skin and subcutaneous tissue disorders | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 7 | 56 | |
| other | Pruritus | Skin and subcutaneous tissue disorders | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 7 | 58 |
1 to 10 of 154Source ClinicalTrials.gov
See all 154 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 0 | 56 |
| Total, all-cause mortality | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 1 | 58 |
| Total, other adverse events | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 56 | 56 |
| Total, other adverse events | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 57 | 58 |
| Total, serious adverse events | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 8 | 56 |
| Total, serious adverse events | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 10 | 58 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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 |
|---|---|
| Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) vs Treatment Phase - Adult Cohort Mirdametinib (PD-0325901): deaths from any cause | Trial comparisons |
| Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) vs Treatment Phase - Adult Cohort Mirdametinib (PD-0325901): 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/NCT03962543
GET https://conductscience.org/api/trials/v1/NCT03962543/results?page=2