NCT03962543 · STUDY IN CONTEXT

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

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.

The Full Analysis Set is defined as patients who received at least one dose of Mirdametinib.

→

secondary · POSTED

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.

The Full Analysis Set includes patients who received at least one dose of Mirdametinib.

→

secondary · POSTED

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.

The Full Analysis Set patients with confirmed objective response.

→

secondary · POSTED

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.

The Full Analysis Set patients who are eligible for the Peds-QL assessments at baseline.

→

secondary · POSTED

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.

The Full Analysis Set patients eligible for the instrument at baseline.

→

secondary · POSTED

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.

The Full Analysis Set patients eligible for the instrument at baseline.

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

Unnamed quality-of-life measure · Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version.

Score direction: Higher is better, according to the source. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-nerve-sheath-neoplasm
nct id: NCT03962543
qol key: qol-0003
source section id: NCT03962543-om-0003
source outcome index: 3
instrument: Unnamed quality-of-life measure
instrument basis: matched 'quality of life' in the study's own wording
scale title: Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version.
measure definition: 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.
time frame: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role: secondary
reporting status: POSTED
higher is better: 1
direction quote: higher scores indicate better
population note: The Full Analysis Set patients who are eligible for the Peds-QL assessments at baseline.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

PROMIS · Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Pediatric v2.0 Mobility 8a.

Score direction: Higher is better, according to the source. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-nerve-sheath-neoplasm
nct id: NCT03962543
qol key: qol-0016
source section id: NCT03962543-om-0016
source outcome index: 16
instrument: PROMIS
instrument basis: matched 'promis' in the study's own wording
scale title: Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Pediatric v2.0 Mobility 8a.
measure definition: PROMIS measures capability of physical functioning, with questions related to daily activities. Participants 8-17 years complete a self-report of physical function in the upper extremity or lower extremity (mobility), depending on the location of the PN. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher reported mobility. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 14 to 59 for self-report, and 14 to 56 for parent proxy with higher scores representing higher reported mobility and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10.
time frame: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role: exploratory
reporting status: POSTED
higher is better: 1
direction quote: higher scores representing higher reported mobility and better
population note: The Full Analysis Set eligible for the instrument at baseline.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

PROMIS · Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Pediatric v2.0 Upper Extremities 8a.

Score direction: Higher is better, according to the source. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-nerve-sheath-neoplasm
nct id: NCT03962543
qol key: qol-0017
source section id: NCT03962543-om-0017
source outcome index: 17
instrument: PROMIS
instrument basis: matched 'promis' in the study's own wording
scale title: Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Pediatric v2.0 Upper Extremities 8a.
measure definition: PROMIS measures capability of physical functioning, with questions related to daily activities. Participants 8-17 years complete a self-report of physical function in the upper extremity or lower extremity (mobility), depending on the location of the PN. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher physical functioning in the upper extremities. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 10 to 57 for self-report, and 13 to 55 for parent proxy with higher scores representing higher reported physical functioning and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10.
time frame: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role: exploratory
reporting status: POSTED
higher is better: 1
direction quote: higher scores representing higher reported physical functioning and better
population note: The Full Analysis Set eligible for the instrument at baseline.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

PROMIS · Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Adult v2.0 Physical Function 8b.

Score direction: Higher is better, according to the source. Direction is never inferred from an instrument name.

release id: clinical-indications-draft-1
disease state id: ds-nerve-sheath-neoplasm
nct id: NCT03962543
qol key: qol-0018
source section id: NCT03962543-om-0018
source outcome index: 18
instrument: PROMIS
instrument basis: matched 'promis' in the study's own wording
scale title: Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Adult v2.0 Physical Function 8b.
measure definition: PROMIS measures capability of physical functioning, with questions related to daily activities. Participants ≥ 18 years with a target PN impacting physical functioning complete a self-report of physical function. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher reported physical capability. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 20.3 to 60.1 with higher scores representing higher reported physical functioning and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10.
time frame: Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role: exploratory
reporting status: POSTED
higher is better: 1
direction quote: higher scores representing higher reported physical functioning and better
population note: The Full Analysis Set eligible for the instrument at baseline.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z

Open the exact result →

Full draft applicability and source-link record

trial

release id: clinical-indications-draft-1
disease state id: ds-nerve-sheath-neoplasm
nct id: NCT03962543
trial label: MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
applicability: narrower
applicability note: Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z
provenance class: regulator-cited
discovery note: Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
outcome Count: 6
harms Count: 2
qol Count: 4

outcomes

release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0000
source section id
NCT03962543-om-0000
source outcome index
0
endpoint
Confirmed Objective Response Rate at the End of the Treatment Phase.
endpoint definition
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.
time origin
Up to 24 months
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
The Full Analysis Set is defined as patients who received at least one dose of Mirdametinib.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0001
source section id
NCT03962543-om-0001
source outcome index
1
endpoint
Percentage of Patients With Treatment-Emergent Adverse Events.
endpoint definition
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.
time origin
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.
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
The Full Analysis Set includes patients who received at least one dose of Mirdametinib.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0002
source section id
NCT03962543-om-0002
source outcome index
2
endpoint
Duration of Response (DOR) for Participants Who Meet Criteria for Confirmed Objective Response.
endpoint definition
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.
time origin
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
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
The Full Analysis Set patients with confirmed objective response.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0003
source section id
NCT03962543-om-0003
source outcome index
3
endpoint
Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version.
endpoint definition
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.
time origin
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
The Full Analysis Set patients who are eligible for the Peds-QL assessments at baseline.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0004
source section id
NCT03962543-om-0004
source outcome index
4
endpoint
Change From Baseline in Pain as Measured by the Numeric Rating Scale-11 (NRS-11) at Cycle 13.
endpoint definition
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.
time origin
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
The Full Analysis Set patients eligible for the instrument at baseline.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0005
source section id
NCT03962543-om-0005
source outcome index
5
endpoint
Change From Baseline in Pain as Measured by the Pain Interference Index (PII) at Cycle 13.
endpoint definition
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.
time origin
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
The Full Analysis Set patients eligible for the instrument at baseline.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

arms

release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0000
arm key
OG000
arm label
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901)
regimen
Patients aged 2 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
56
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0000#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0000
arm key
OG001
arm label
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)
regimen
Patients aged 18 and above at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
58
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0000#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0001
arm key
OG000
arm label
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901)
regimen
Patients aged 2 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
56
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0001#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0001
arm key
OG001
arm label
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)
regimen
Patients aged 18 and above at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
58
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0001#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0002
arm key
OG000
arm label
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901)
regimen
Patients aged 2 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
29
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0002#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0002
arm key
OG001
arm label
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)
regimen
Patients aged 18 and above at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
24
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0002#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0003
arm key
OG000
arm label
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report
regimen
Patients self-reported results aged 5 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
50
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0003#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0003
arm key
OG001
arm label
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy
regimen
Patients parent proxy results aged 2 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
56
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0003#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0003
arm key
OG002
arm label
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report
regimen
Patients self-reported results aged 18 years and above at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
58
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0003#groups/OG002
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0004
arm key
OG000
arm label
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report
regimen
Patients aged 8 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
37
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0004#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0004
arm key
OG001
arm label
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report
regimen
Patients aged 18 and above at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
58
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0004#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
outcome key
om-0005
arm key
OG000
arm label
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report
regimen
Patients self-reported results aged 6 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
analyzed count
47
analyzed unit
Participants
is comparator
Source null
source pointer
NCT03962543-om-0005#groups/OG000

harms

release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
harm key
EG000
source section id
NCT03962543-ae
event group key
EG000
event group label
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901)
serious affected
8
serious at risk
56
other affected
56
other at risk
56
frequency threshold
5
collection period
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.
event definition
All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment. Medical occurrences that begin before the start of study treatment but after obtaining informed consent will be recorded as AEs.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
harm key
EG001
source section id
NCT03962543-ae
event group key
EG001
event group label
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)
serious affected
10
serious at risk
58
other affected
57
other at risk
58
frequency threshold
5
collection period
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.
event definition
All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment. Medical occurrences that begin before the start of study treatment but after obtaining informed consent will be recorded as AEs.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

quality of life

release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
qol key
qol-0003
source section id
NCT03962543-om-0003
source outcome index
3
instrument
Unnamed quality-of-life measure
instrument basis
matched 'quality of life' in the study's own wording
scale title
Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version.
measure definition
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.
time frame
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role
secondary
reporting status
POSTED
higher is better
1
direction quote
higher scores indicate better
population note
The Full Analysis Set patients who are eligible for the Peds-QL assessments at baseline.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
qol key
qol-0016
source section id
NCT03962543-om-0016
source outcome index
16
instrument
PROMIS
instrument basis
matched 'promis' in the study's own wording
scale title
Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Pediatric v2.0 Mobility 8a.
measure definition
PROMIS measures capability of physical functioning, with questions related to daily activities. Participants 8-17 years complete a self-report of physical function in the upper extremity or lower extremity (mobility), depending on the location of the PN. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher reported mobility. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 14 to 59 for self-report, and 14 to 56 for parent proxy with higher scores representing higher reported mobility and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10.
time frame
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role
exploratory
reporting status
POSTED
higher is better
1
direction quote
higher scores representing higher reported mobility and better
population note
The Full Analysis Set eligible for the instrument at baseline.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
qol key
qol-0017
source section id
NCT03962543-om-0017
source outcome index
17
instrument
PROMIS
instrument basis
matched 'promis' in the study's own wording
scale title
Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Pediatric v2.0 Upper Extremities 8a.
measure definition
PROMIS measures capability of physical functioning, with questions related to daily activities. Participants 8-17 years complete a self-report of physical function in the upper extremity or lower extremity (mobility), depending on the location of the PN. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher physical functioning in the upper extremities. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 10 to 57 for self-report, and 13 to 55 for parent proxy with higher scores representing higher reported physical functioning and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10.
time frame
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role
exploratory
reporting status
POSTED
higher is better
1
direction quote
higher scores representing higher reported physical functioning and better
population note
The Full Analysis Set eligible for the instrument at baseline.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-nerve-sheath-neoplasm
nct id
NCT03962543
qol key
qol-0018
source section id
NCT03962543-om-0018
source outcome index
18
instrument
PROMIS
instrument basis
matched 'promis' in the study's own wording
scale title
Change From Baseline in Physical Function Status as Measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) at Cycle 13: Adult v2.0 Physical Function 8b.
measure definition
PROMIS measures capability of physical functioning, with questions related to daily activities. Participants ≥ 18 years with a target PN impacting physical functioning complete a self-report of physical function. The recall period is 7 days. All PROMIS items are answered on a Likert scale with responses ranging from 1 to 5, with higher scores representing higher reported physical capability. The total raw score will be calculated as the sum of the individual item responses at a given visit (total raw scale range 8-40). The total raw scores for the measure will be converted to T-scores using the applicable score conversion table in the user manual and scoring instructions (PROMIS, 2023) ranging from 20.3 to 60.1 with higher scores representing higher reported physical functioning and better outcomes. A T-score of 50 indicates the population mean with a standard deviation of 10.
time frame
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
analysis role
exploratory
reporting status
POSTED
higher is better
1
direction quote
higher scores representing higher reported physical functioning and better
population note
The Full Analysis Set eligible for the instrument at baseline.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

setting source links

Preserved source evidence · Not medical advice