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This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901). Eligible participants may continue in a long-term follow-up phase.
Neurofibromas are non-malignant peripheral nerve sheath tumors, which are classified as plexiform neurofibromas (PNs) if they extend longitudinally along a nerve and involve multiple fascicles. PNs are a major cause of morbidity and disfigurement in individuals with NF1, and as the tumor growth progresses, can cause a multitude of clinical problems including pain and impaired physical function. PNs have the potential to undergo malignant transformation to Malignant Peripheral Nerve Sheet Tumors (MPNST).
Mirdametinib (PD-0325901) is an orally delivered, highly selective small-molecule inhibitor of the dual specificity kinases, MEK1 and MEK2 (MAPK/ERK Kinase) which prevents the phosphorylation and subsequent activation of mitogen-activated protein kinase (MAPK).
Previous studies of mirdametinib (PD-0325901) demonstrated PN shrinkage and sustained inhibition of pERK. Reduced tumor volume indicated that cell proliferation or cell death may be altered in PNs with administration of mirdametinib (PD-0325901).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mirdametinib (PD-0325901) | Experimental | Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m^2 (maximum dose of 4 mg) by mouth twice daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mirdametinib (PD-0325901) oral capsule or dispersible tablet | Drug | Mirdametinib (PD-0325901) capsule or dispersible tablet |
|
| Measure | Description | Time Frame |
|---|---|---|
| Confirmed Objective Response Rate at the End of the Treatment Phase. | 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. | Up to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Patients With Treatment-Emergent Adverse Events. | 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. | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Localized Strength (Dynamometer) at Cycle 13. | 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. |
Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Responsible | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham/Children's of Alabama | Birmingham | Alabama | 35233 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39514826 | Derived | Moertel CL, Hirbe AC, Shuhaiber HH, Bielamowicz K, Sidhu A, Viskochil D, Weber MD, Lokku A, Smith LM, Foreman NK, Hajjar FM, McNall-Knapp RY, Weintraub L, Antony R, Franson AT, Meade J, Schiff D, Walbert T, Ambady P, Bota DA, Campen CJ, Kaur G, Klesse LJ, Maraka S, Moots PL, Nevel K, Bornhorst M, Aguilar-Bonilla A, Chagnon S, Dalvi N, Gupta P, Khatib Z, Metrock LK, Nghiemphu PL, Roberts RD, Robison NJ, Sadighi Z, Stapleton S, Babovic-Vuksanovic D, Gershon TR; ReNeu Trial Investigators; ReNeu Study Investigators. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma. J Clin Oncol. 2025 Feb 20;43(6):716-729. doi: 10.1200/JCO.24.01034. Epub 2024 Nov 8. |
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We are committed to enhancing public health through responsible sharing of clinical trial data. Following approval of a new product or a new indication for an approved product in both the US and European Union, the study sponsor and/or its affiliated companies will share study protocols, anonymized patient data and study level data, and redacted clinical study reports with qualified scientific and medical researchers, upon request, as necessary for conducting legitimate research. Further information on how to request data can be found on our website bit.ly/IPD21
IPD from completed trials will be publicly available within 6 months after all of the following events:
Qualified researchers may propose access to IPD from sponsored trials via https://vivli.org/members/ourmembers/
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 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 |
| FG001 | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Phase |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 17, 2022 | Mar 12, 2025 |
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All participants will receive mirdametinib (PD-0325901) at a dose of 2 mg/m^2 twice daily (maximum dose of 4 mg twice daily), calculated based on body surface area. Dose will be administered in a 3-week on, 1-week off schedule.
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| Duration of Response (DOR) for Participants Who Meet Criteria for Confirmed Objective Response. | 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. | 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 |
| Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version. | 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. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| Change From Baseline in Pain as Measured by the Numeric Rating Scale-11 (NRS-11) at Cycle 13. | 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. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| Change From Baseline in Pain as Measured by the Pain Interference Index (PII) at Cycle 13. | 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. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| Change From Baseline in Range of Motion of PN-Associated Functional Impairment at Cycle 13. | If motor dysfunction or weakness is evident, range of motion of the affected areas and/or joints will be measured by a goniometer. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| Change From Baseline in Endurance at Cycle 13. | If airway or lower extremity motor dysfunction is evident, endurance will be measured by completion of a 6-minute walking test. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| Time to Response Defined as the Time Between First Dose and the First Date of Objective Response That is Subsequently Confirmed. | 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. | Up to 24 months |
| Time to Progression, From the First Dose to the First Date of a ≥ 20% Increase in Tumor Volume From Baseline. | Evidence of progression or tumor growth will be determined by a blinded centralized review of volumetric MRI. Time to progression is defined as the time in months between the first treatment date until the date of radiographic disease progression. For participants who enter the LTFU Phase, all MRI assessments in both the Treatment Phase and LTFU Phase will be used to determine time to progression. Participants without radiographic disease progression will have their results censored to their most recent adequate (i.e. evaluable) tumor assessment date. | On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed, assessed up to approximately 3.5 years |
| Progression Free Survival, Defined as the Time in Months From the First Dose to the Date of the First ≥ 20% Increase in Tumor Volume From Baseline or Death. | Evidence of progression or tumor growth will be determined by a blinded centralized review of volumetric MRI. Progression free survival is defined as the time in months between the first treatment date, 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 progression free survival. 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. | On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, assessed up to approximately 3.5 years |
| Change From Baseline in PN-Associated Disfigurement Using Standardized Photography, Centrally Reviewed. | For participants with a PN that is visible and amenable to photography, changes in visible tumor aspects will be evaluated by a centralized reviewer. | Up to 24 months |
| Comparison of Tumor Response to Levels of pERK and Biomarkers Indicative of Inhibition of Downstream Targets of MEK (eg, ERK Phosphorylation). | Measured in tumor biopsies in participants ≥ 18 years of age. | Up to 24 months |
| Acceptability of the Dispersible Tablet Formulation as Measured by the Pediatric Oral Medicine Acceptability Questionnaire (P-OMAQ) | The Pediatric Oral Medicine Acceptability Questionnaire (P-OMAQ) uses a 5-point numerical rate scale to measure acceptability of use of the tablet formulation with questions related to taste, smell and administration of study medication. Participants ≥ 8 years of age complete a 12-item self-report; adult parents/caregivers responsible for oversight of study drug administration for participants ages 6 months to 17 years complete a 19-item caregiver report. Each questionnaire is provided with a "past 7 days" recall with all items using a 5-point Numerical Rate Scale (1 to 5) with higher item-scores reflecting greater oral treatment acceptability. The overall P-OMAQ-P (pediatric self-report) and P-OMAQ-C (adult caregiver) scores for a participant in the study will be taken as the mean of all answered questions on the respective questionnaire. | Completed only once through study completion |
| Change From Baseline in Localized Strength (Medical Research Council (MRC) Scale Grading) at Cycle 13. | If motor dysfunction or weakness is evident, strength of the affected muscle groups will be measured by dynamometer and assessed by a muscle grading scale. Medical Research Council (MRC) Scale grading (0-5) must be conducted in accordance with the study reference manual. MRC Grading is assessed on a scale of 0 to 5, with 0 representing no movement being observed, and 5 representing the muscle contracting normally against full resistance. 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 MRC Grade visit score is calculated for each participant separately, and these scores are then averaged. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| 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. | 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. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| 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. | 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. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| 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. | 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. | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| Mayo Clinic Arizona |
| Phoenix |
| Arizona |
| 98195 |
| United States |
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| University of California, Los Angeles (UCLA) | Los Angeles | California | 90095 | United States |
| Children's Hospital of Orange County | Orange | California | 92868 | United States |
| University of California - Irvine Health | Orange | California | 92868 | United States |
| Lucile Packard Children's Hospital Stanford | Palo Alto | California | 94304 | United States |
| University of California - Davis | Sacramento | California | 95817 | United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Nemours A. I. duPont Hospital for Children | Wilmington | Delaware | 19803 | United States |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | United States |
| University of Florida Clinical Research Center | Gainesville | Florida | 32610 | United States |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | United States |
| Florida Hospital Cancer Institute | Orlando | Florida | 32804 | United States |
| Arnold Palmer Hospital for Children | Orlando | Florida | 32806 | United States |
| Johns Hopkins All Children's Hospital | St. Petersburg | Florida | 33701 | United States |
| Moffitt | Tampa | Florida | 33612 | United States |
| University of Illinois Hospital and Health Systems | Chicago | Illinois | 60612 | United States |
| Indiana University | Indianapolis | Indiana | 46202 | United States |
| University of Iowa Health Care | Iowa City | Iowa | 52242 | United States |
| University of Michigan Comprehensive Cancer Center | Ann Arbor | Michigan | 48109 | United States |
| Henry Ford Hospital | Detroit | Michigan | 48202 | United States |
| University of Minnesota/Masonic Cancer Center | Minneapolis | Minnesota | 55455 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Saint Joseph's Regional Medical Center | Paterson | New Jersey | 07503 | United States |
| Albany Medical Center | Albany | New York | 12208 | United States |
| Children's Hospital at Montefiore | The Bronx | New York | 10467 | United States |
| UNC Medical Center | Chapel Hill | North Carolina | 27599 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| UC Health | Cincinnati | Ohio | 45219 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| University of Oklahoma - Health Sciences Center | Oklahoma City | Oklahoma | 73104 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| Children's Hospital of Pittsburgh UPMC | Pittsburgh | Pennsylvania | 15219-2739 | United States |
| Vanderbilt Children's Hospital | Nashville | Tennessee | 37232 | United States |
| University of Texas Southwestern | Dallas | Texas | 75244 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| The University of Utah Health Sciences Center | Salt Lake City | Utah | 84108 | United States |
| University of Virginia - Emily Couric Cancer Center | Charlottesville | Virginia | 22903 | United States |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23510 | United States |
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 |
| COMPLETED |
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| NOT COMPLETED |
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| Long Term Follow-Up Phase |
|
|
Full Analysis Set (Received At Least One Dose of Mirdametinib)
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 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 |
| BG001 | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 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 |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
| ||||||||||||||||
| Karnofsky/Lansky Score | Karnofsky (>= 16 years of age) and Lansky (< 16 years of age) performance scores are intended to be multiples of 10 ranging from 0 to 100 with larger scores indicating better outcomes. | Mean | Standard Deviation | units on a scale |
| ||||||||||||||
| Childbearing Potential at Screening | Based on the number of female patients in the Full Analysis Set | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Confirmed Objective Response Rate at the End of the Treatment Phase. | 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. | Posted | Number | 95% Confidence Interval | percentage of patients | Up to 24 months |
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| Secondary | Percentage of Patients With Treatment-Emergent Adverse Events. | 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. | Posted | Number | percentage of patients | 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. |
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| Secondary | Duration of Response (DOR) for Participants Who Meet Criteria for Confirmed Objective Response. | 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. | Posted | Median | 95% Confidence Interval | months | 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 |
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| Secondary | Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version. | 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. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
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| Secondary | Change From Baseline in Pain as Measured by the Numeric Rating Scale-11 (NRS-11) at Cycle 13. | 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. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
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| Secondary | Change From Baseline in Pain as Measured by the Pain Interference Index (PII) at Cycle 13. | 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. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
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| Other Pre-specified | Change From Baseline in Localized Strength (Dynamometer) at Cycle 13. | 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. | The Full Analysis Set patients eligible for this instrument at baseline. | Posted | Mean | Standard Deviation | lbs | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
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| Other Pre-specified | Change From Baseline in Range of Motion of PN-Associated Functional Impairment at Cycle 13. | If motor dysfunction or weakness is evident, range of motion of the affected areas and/or joints will be measured by a goniometer. | The Full Analysis Set patients who are eligible for this instrument at baseline. | Posted | Mean | Standard Deviation | degrees | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
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| Other Pre-specified | Change From Baseline in Endurance at Cycle 13. | If airway or lower extremity motor dysfunction is evident, endurance will be measured by completion of a 6-minute walking test. | The Full Analysis Set patients eligible for this instrument at baseline | Posted | Mean | Standard Deviation | meters | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
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| Other Pre-specified | Time to Response Defined as the Time Between First Dose and the First Date of Objective Response That is Subsequently Confirmed. | 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. | The Full Analysis Set patients who achieved confirmed objective response. | Posted | Mean | Standard Deviation | months | Up to 24 months |
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| Other Pre-specified | Time to Progression, From the First Dose to the First Date of a ≥ 20% Increase in Tumor Volume From Baseline. | Evidence of progression or tumor growth will be determined by a blinded centralized review of volumetric MRI. Time to progression is defined as the time in months between the first treatment date until the date of radiographic disease progression. For participants who enter the LTFU Phase, all MRI assessments in both the Treatment Phase and LTFU Phase will be used to determine time to progression. Participants without radiographic disease progression will have their results censored to their most recent adequate (i.e. evaluable) tumor assessment date. | The Full Analysis Set defined as all patients who received at least one dose of Mirdametinib. | Posted | Median | 95% Confidence Interval | months | On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed, assessed up to approximately 3.5 years |
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| Other Pre-specified | Progression Free Survival, Defined as the Time in Months From the First Dose to the Date of the First ≥ 20% Increase in Tumor Volume From Baseline or Death. | Evidence of progression or tumor growth will be determined by a blinded centralized review of volumetric MRI. Progression free survival is defined as the time in months between the first treatment date, 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 progression free survival. 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 defined as all patients who received at least one dose of Mirdametinib. | Posted | Median | 95% Confidence Interval | months | On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, assessed up to approximately 3.5 years |
| ||||||||||||||||||||||||||||||
| Other Pre-specified | Change From Baseline in PN-Associated Disfigurement Using Standardized Photography, Centrally Reviewed. | For participants with a PN that is visible and amenable to photography, changes in visible tumor aspects will be evaluated by a centralized reviewer. | Not Posted | Up to 24 months | Participants | ||||||||||||||||||||||||||||||||||
| Other Pre-specified | Comparison of Tumor Response to Levels of pERK and Biomarkers Indicative of Inhibition of Downstream Targets of MEK (eg, ERK Phosphorylation). | Measured in tumor biopsies in participants ≥ 18 years of age. | Not Posted | Up to 24 months | Participants | ||||||||||||||||||||||||||||||||||
| Other Pre-specified | Acceptability of the Dispersible Tablet Formulation as Measured by the Pediatric Oral Medicine Acceptability Questionnaire (P-OMAQ) | The Pediatric Oral Medicine Acceptability Questionnaire (P-OMAQ) uses a 5-point numerical rate scale to measure acceptability of use of the tablet formulation with questions related to taste, smell and administration of study medication. Participants ≥ 8 years of age complete a 12-item self-report; adult parents/caregivers responsible for oversight of study drug administration for participants ages 6 months to 17 years complete a 19-item caregiver report. Each questionnaire is provided with a "past 7 days" recall with all items using a 5-point Numerical Rate Scale (1 to 5) with higher item-scores reflecting greater oral treatment acceptability. The overall P-OMAQ-P (pediatric self-report) and P-OMAQ-C (adult caregiver) scores for a participant in the study will be taken as the mean of all answered questions on the respective questionnaire. | The Full Analysis Set eligible for the instrument at baseline | Posted | Mean | Standard Deviation | units on a scale | Completed only once through study completion |
| ||||||||||||||||||||||||||||||
| Other Pre-specified | Change From Baseline in Localized Strength (Medical Research Council (MRC) Scale Grading) at Cycle 13. | If motor dysfunction or weakness is evident, strength of the affected muscle groups will be measured by dynamometer and assessed by a muscle grading scale. Medical Research Council (MRC) Scale grading (0-5) must be conducted in accordance with the study reference manual. MRC Grading is assessed on a scale of 0 to 5, with 0 representing no movement being observed, and 5 representing the muscle contracting normally against full resistance. 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 MRC Grade visit score is calculated for each participant separately, and these scores are then averaged. | The Full Analysis Set patients eligible for this instrument at baseline. | Posted | Mean | Standard Deviation | units on a scale | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| ||||||||||||||||||||||||||||||
| Other Pre-specified | 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. | 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. | The Full Analysis Set eligible for the instrument at baseline. | Posted | Mean | Standard Deviation | T-score | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| ||||||||||||||||||||||||||||||
| Other Pre-specified | 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. | 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. | The Full Analysis Set eligible for the instrument at baseline. | Posted | Mean | Standard Deviation | T-score | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
| ||||||||||||||||||||||||||||||
| Other Pre-specified | 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. | 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. | The Full Analysis Set eligible for the instrument at baseline. | Posted | Mean | Standard Deviation | T-score | Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months |
|
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 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.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) | 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 | 0 | 56 | 8 | 56 | 56 | 56 |
| EG001 | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) | 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 | 1 | 58 | 10 | 58 | 57 | 58 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Gastrointestinal viral infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Rotavirus infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Near drowning | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Diplopia | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Retinal vein occlusion | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Colitis ischaemic | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Squamous cell carcinoma of skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Device breakage | Product Issues | MedDRA 24.0 | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hair colour changes | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Ejection fraction decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Tumour pain | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Head of Clinical Operations | SpringWorks Therapeutics, Inc. | 8772794870 | clinical@springworkstx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 2, 2023 | Mar 12, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D018318 | Neurofibroma, Plexiform |
| D009456 | Neurofibromatosis 1 |
| D017253 | Neurofibromatoses |
| D009455 | Neurofibroma |
| ID | Term |
|---|---|
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D010524 | Peripheral Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
| D009422 | Nervous System Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D009386 | Neoplastic Syndromes, Hereditary |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C506614 | mirdametinib |
Not provided
Not provided
Not provided
| Physician Decision |
|
| Radiographic PD |
|
| Sponsor Request |
|
| Participant Decision |
|
| Positive pregnancy test |
|
| Ongoing in the Long Term Follow-Up Phase Phase |
|
|
|
|
|
|
|
|
|
|
|
| OG001 | Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy | 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 |
| OG002 | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report | 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 |
|
|
| Participants |
|
|
Patients parent proxy 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 |
| OG002 | Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report | 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 |
|
|
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 |
|
|
|
|
|
|
|
|
|
The adult caregiver of Pediatric 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
|
|
|
|
| Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy |
Patients parent proxy 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 |
|
|
| OG001 |
| Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy |
Patients parent proxy 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 |
|
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| Units | Counts |
|---|---|
| Participants |
|
|