Low-Grade Glioma Clinical Trial
Official title:
SJ901: Phase 1/2 Evaluation of Single Agent Mirdametinib (PD-0325901), a Brain-Penetrant MEK1/2 Inhibitor, for the Treatment of Children, Adolescents, and Young Adults With Low-Grade Glioma
This is an open-label, multi-center, Phase 1/2 study of the brain-penetrant MEK inhibitor, mirdametinib (PD-0325901), in patients with pediatric low-grade glioma (pLGG).
Status | Recruiting |
Enrollment | 132 |
Est. completion date | June 2031 |
Est. primary completion date | June 2031 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 24 Years |
Eligibility | Inclusion Criteria: Screening Phase - Participants with histologically confirmed or suspected low-grade glioma, including neuronal and mixed neuronal-glial tumors - Participant must have adequate tumor tissue from primary and/or relapsed tumor for central pathology review - For Phase 1: Projected to be = 2 years and < 25 years at the time of study enrollment - Participant's body surface area (BSA) at time of study enrollment must fall within the range outlined in the protocol for the specific dose level under evaluation: - Phase 1: Dose Finding/Dose-escalation - For Phase 1 participant's BSA must fall within the range specified in the protocol for the specific dose level under evaluation. - Phase 2: All Cohorts: - For Phase 2 of the study the upper BSA restrictions will be removed. - Participant and/or guardian can understand and is willing to sign a written informed consent document according to institutional guidelines Exclusion Criteria: Screening Phase - Participants with known current retinal pathology that is consistent with or a precursor for central serous retinopathy, retinal vein occlusion (RVO), or neovascular macular degeneration - Participants with a known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of mirdametinib (e.g., gastric bypass, lap band, or other gastric procedures) - Participant with a known history of liver disease or known hepatic or biliary abnormalities (except for Gilbert's syndrome or asymptomatic gallstones) - Participants with a clinically significant history of chronic interstitial lung disease (such as bronchopulmonary dysplasia, chronic bronchiolitis, obliterative bronchiolitis, chronic aspiration pneumonia, surfactant protein disorder, or other serious chronic pulmonary condition). Participants with a history of asthma, reactive airways disease, or viral pneumonitis are not to be excluded if disease has resolved or is well-controlled. Inclusion Criteria: Phase 1 and Phase 2, All Cohorts - Participant must be = 2 years and < 25 years of age at the time of enrollment - Participant's BSA at time of study enrollment must fall within the range outlined below for the specific dose level under evaluation: - Phase 1: Dose-finding/Dose-escalation - For Phase 1 participant's BSA must fall within the range specified in the protocol for the specific dose level under evaluation. - Phase 2: All Cohorts - For Phase 2 of the study the upper BSA restrictions will be removed. - Participant must have confirmation of one of the following diagnosis per St. Jude Children's Research Hospital central pathology review of primary and/or relapsed tumor: - Eligible tumors include: - Low-grade glioma/astrocytic tumor/glioneuronal tumor/neuroepithelial tumor, not otherwise specified (NOS) or not elsewhere classified (NEC) - Pilocytic astrocytoma - Pilomyxoid astrocytoma - Pleomorphic xanthroastrocytoma - Ganglioglioma - Gangliocytoma - Diffuse glioma, diffuse astrocytoma, oligodendroglioma, or oligoastrocytoma - Papillary glioneuronal tumor - Rosette-forming glioneuronal tumor - Diffuse leptomeningeal glioneuronal tumor - Central neurocytoma, extraventricular neurocytoma - Angiocentric glioma - Dysembryoplastic neuroepithelial tumor (DNET), septal DNET, myxoid glioneuronal tumor - Tectal glioma - Desmoplastic infantile astrocytoma / ganglioglioma - Polymorphous low-grade neuroepithelial tumor of the young - Multinodular and vacuolating neuronal tumor - In addition, tumor on central review must show evidence supporting MAPK pathway activation as defined by IHC, FISH and/or DNA/RNA sequencing (i.e. BRAF fused or rearranged, FGFR1/2/3 aberration, PTPN11, SOS1, RAF1 mutations, MYB or MYBL1 fused or rearranged, etc.) or occur in a participant with known NF1, NF2, SOS1, RAF1, or PTPN11 germline mutation. (Note: tests that show evidence supporting MAPK pathway activation that have been already performed do not need to be repeated as long as deemed acceptable by central review). - Participant must have measurable or evaluable disease (as defined in the protocol) - Note: Participants with metastatic disease or multiple independent primary LGGs are allowed on study. - Participants who are receiving corticosteroids must be on a stable or decreasing dose for at least 1 week prior to enrollment with no plans for escalation. - Participant must have a Lansky (<16 years) or Karnofsky (=16 years) performance score of = 50 and, in the opinion of the investigator, a minimum life expectancy of at least 6 weeks. - Note: Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score. - Participant must have adequate bone marrow and organ function as defined as: - ANC = 1.0 x 10^9/L without growth factor support within 7 days - Platelet count = 75x 10^9/L without support of a platelet transfusion within 7 days - Hemoglobin =8.0 g/dL without support of a blood transfusion within 7 days - Potassium, total calcium (corrected for serum albumin), magnesium, sodium and phosphorus must be = grade 1 or corrected to = grade 1 with supplements before first dose of study medication - Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) = 2.5 x ULN. For the purposes of this study the ULN of ALT and AST is 45 U/L. - Total bilirubin = ULN; or if > ULN then direct bilirubin = 1.5 x ULN - Adequate renal function defined as: - Serum creatinine = the maximum serum creatinine based on age/gender: Age: 2 to < 6 years: maximum serum creatinine (mg/dL) 0.8 (male, female), Age: 6 to <10 years: maximum serum creatinine (mg/dL) 1 (male, female), Age: 10 to <13 years: maximum serum creatinine (mg/dL) 1.2 (male, female), Age: 13 to <16 years: maximum serum creatinine (mg/dL) 1.5 (male); 1.4 (female), Age: = 16 years: maximum serum creatinine (mg/dL) 1.7 (male); 1.4 (female) - Adequate cardiac function defined as: - LVEF > 50% by ECHO - QTc interval = 450 msec for male participants, = 470 msec for female participants after electrolytes have been corrected. - Hypertension: - Patients 2-12.99 years of age must have a blood pressure that is = 95th percentile +10 mmHg for age, height, and gender at the time of enrollment (with or without the use of anti-hypertensive medications). - Patients = 13 years of age must have a blood pressure = 140/90 mmHg at the time of enrollment (with or without the use of anti-hypertensive medications). - Note for patients of all ages: Adequate blood pressure can be achieved using medication for the treatment of hypertension. - Participants of childbearing/child-fathering potential must agree to use contraception. - Participants and/or guardian have the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines. - Participants who are receiving P-gp and BCRP inhibitors must have received their last dose a week or 5 half-lives (whichever is greater) prior to the first mirdametinib dose. Inclusion Criteria: Phase 1: Progressive or Recurrent Low-Grade Glioma without Previous MEKi exposure - Participant's tumor must have unambiguously progressed, relapsed, or recurred during or after the most recent prior therapy (chemotherapy or radiotherapy) and pseudoprogression or treatment-related tumor changes have, in the opinion of the investigator, been thoroughly vetted. - Progression may be radiographic or clinical (i.e. vision deterioration thought to be related to tumor in patients with optic pathway tumors, or neurologic deterioration thought to be related to tumor) but it must be unequivocal and sufficient to warrant treatment in the opinion of the investigator. - Prior therapy: - Patients who have received the following: - = 3 prior treatment regimens with either myelosuppressive chemotherapy or biologic agents and/or - focal radiotherapy - Note that a treatment regimen is defined as a single agent (chemotherapeutic or biologic), or a sequential combination of therapies that can include radiotherapy (with or without concurrent radiosensitizer, chemotherapy, or biologic therapy) followed by maintenance therapy (either single or combination) given over a period of time at either diagnosis or relapse. - Chemotherapy: - Participant must have received their last dose of myelosuppressive anticancer chemotherapy at least 21 days prior to study enrollment or at least 42 days if nitrosourea. - Monoclonal antibody treatment and agents with known prolonged half-lives: - Patient must have recovered from any acute toxicity potentially related to the agent and received their last dose of the agent = 28 days prior to study enrollment - MEK inhibitors: - Patients must not have received prior exposure to any MEK inhibitors - XRT/External Beam Irradiation including Protons: - Participant must have had their last fraction of radiation = 3 months prior to study enrollment Inclusion Criteria: Phase 2, Cohort 1: Newly Diagnosed and/or previously untreated (except surgery) Low-Grade Glioma - No prior anti-cancer treatment except surgery. - In the opinion of the investigator tumor must warrant treatment defined as any of the following: unsafe to observe, unequivocally progressing on serial imaging, tumor is causing or at high risk of causing neurologic or vision-related deficits. Inclusion Criteria: Phase 2, Cohort 2: Progressive or Recurrent Low-Grade Glioma without Previous MEK Inhibitor Exposure - Participant's tumor must have unambiguously progressed, relapsed, or recurred during or after the most recent prior therapy (chemotherapy or radiotherapy) and pseudoprogression or treatment-related tumor changes have, in the opinion of the investigator, been thoroughly vetted. - Progression may be radiographic or clinical (i.e. vision deterioration thought to be related to tumor in patients with optic pathway tumors, or neurologic deterioration thought to be related to tumor) but it must be unequivocal and sufficient to warrant treatment in the opinion of the investigator. - Prior therapy: - Chemotherapy: - Participant must have received their last dose of myelosuppressive anticancer chemotherapy at least 21 days prior to study enrollment or at least 42 days if nitrosourea. - Monoclonal antibody treatment and agents with known prolonged half-lives: - Patient must have recovered from any acute toxicity potentially related to the agent and received their last dose of the agent = 28 days prior to study enrollment - MEK inhibitors: - Patients must not have received prior exposure to any MEK inhibitors - XRT/External Beam Irradiation including Protons: - Participant must have had their last fraction of radiation = 3 months prior to study enrollment. - Note that for this cohort, there are no limitations on number of prior treatment regimens and participants who have received craniospinal radiation are eligible Inclusion Criteria: Phase 2, Cohort 3: Progressive or Recurrent Low-Grade Glioma with Previous MEK inhibitor Exposure - Cohort 3A (MEKi responders): Patients who previously received 6 or more cycles of any MEK inhibitor (including mirdametinib) and did NOT progress while on active MEK inhibitor therapy. - The progression must have occurred off MEK inhibitor therapy - Participant's tumor must have unambiguously relapsed or clinically progressed. Progression may be radiographic or clinical (i.e. vision deterioration thought to be related to tumor in patients with optic pathway tumors, or neurologic deterioration thought to be related to tumor) but it must be unequivocal and sufficient to warrant treatment in the opinion of the investigator. - Patient must not have discontinued MEKi (specifically mirdametinib) for unacceptable toxicity, and in the opinion of the PI be able to tolerate subsequent courses of MEKi therapy. - Patients must have received treatment with a MEK inhibitor for =6 cycles and showed no signs of progression while on active MEK inhibitor therapy. - Patients who received additional anti-tumor therapy following discontinuation of MEK inhibitor can be enrolled in this cohort. - Prior Therapy: - Chemotherapy: - Participant must have received their last dose of myelosuppressive anticancer chemotherapy at least 21 days prior to study enrollment or at least 42 days if nitrosourea. - Monoclonal antibody treatment and agents with known prolonged half-lives: - Patient must have recovered from any acute toxicity potentially related to the agent and received their last dose of the agent = 28 days prior to study enrollment. - MEK inhibitors: - Participant must have received their last dose of MEKi at least 3 weeks prior to study enrollment. - XRT/External Beam Irradiation including Protons: - Participant must have had their last fraction of radiation = 3 months prior to study enrollment. - Note that for this cohort, there are no limitations on number of prior treatment regimens and participants who have received craniospinal radiation are eligible - Cohort 3B (MEKi non-responders): Patients with previous exposure to alternative MEK inhibitors (excluding mirdametinib) who progressed while on active MEK inhibitor therapy - Participant's tumor must have unambiguously relapsed or clinically progressed. - Progression may be radiographic or clinical (i.e. vision deterioration thought to be related to tumor in patients with optic pathway tumors, or neurologic deterioration thought to be related to tumor) but it must be unequivocal and sufficient to warrant treatment in the opinion of the investigator. Progression or recurrence must have occurred while on active MEK inhibitor therapy (excluding mirdametinib) - Participants are eligible regardless of how many prior cycles were received or prior history of response (i.e. PR, Major Response, or CR) - Patients who received additional anti-tumor therapy following discontinuation of MEK inhibitor can be enrolled in this cohort as long as they meet the above criteria. - Prior Therapy: - Chemotherapy: - Participant must have received their last dose of myelosuppressive anticancer chemotherapy at least 21 days prior to study enrollment or at least 42 days if nitrosourea. - Monoclonal antibody treatment and agents with known prolonged half-lives: - Patient must have recovered from any acute toxicity potentially related to the agent and received their last dose of the agent = 28 days prior to study enrollment. - Alternative MEK inhibitor: - Participant must have received their last dose of MEKi (excluding mirdametinib) at least 3 weeks prior to study enrollment. - XRT/External Beam Irradiation including Protons: - Participant must have had their last fraction of radiation = 3 months prior to study enrollment. - Note that for this cohort, there are no limitations on number of prior treatment regimens and participants who have received craniospinal radiation are eligible. Exclusion Criteria: Phase 1 and Phase 2, All Cohorts - Participants whose tumor on central review is any of the following: - High-grade (WHO III or IV) - Subependymal giant cell astrocytoma - Ependymoma - Histone H3 K27M/K28M or G34/G35-mutant - BRAF V600 mutant - NTRK1/2/3, ALK, or ROS1 fusion-positive - IDH 1/2 mutant - Participant who is currently receiving any other anticancer or investigational agents (^11C-methionine allowed) or still recovering from acute toxicity potentially related to the agent. - Ophthalmologic Conditions - Patients with central serous retinopathy - Patients with retinal vein occlusion or retinal detachment - Patients with uncontrolled glaucoma - If checking pressure is clinically indicated and feasible per patient's age and ability to complete exam, patients with IOP > 22 mmHg or ULN adjusted by age are not eligible - Participants with other clinically significant medical disorders (i.e. serious infections or significant cardiac, pulmonary, hepatic, psychiatric, or other organ dysfunction) that in the investigator's judgement could compromise their ability to tolerate or absorb protocol therapy or would interfere with the study procedures or results. - Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 16 weeks after stopping study therapy are not eligible. - Participants are excluded if unable to comply with protocol guidelines. |
Country | Name | City | State |
---|---|---|---|
United States | St. Jude Children's Research Hospital | Memphis | Tennessee |
Lead Sponsor | Collaborator |
---|---|
St. Jude Children's Research Hospital | SpringWorks Therapeutics, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Phase 1: Estimate the maximum tolerated dose (MTD)/recommended phase 2 dose (RP2D) of mirdametinib dosed twice daily on a continuous schedule in pediatric patients with progressive or recurrent low-grade glioma. | The maximum tolerated dose (MTD) is empirically defined as the highest dose level at which six patients have been treated with at most one patient experiencing a dose-limiting toxicity (DLT) and the next higher dose level determined to be too toxic. The MTD estimate will not be available if the lowest dose level studied is too toxic or the highest dose level studied is considered safe. In the latter case, the highest studied safe dose may be considered as the recommended phase 2 dose (RP2D). The MTD estimation will be limited to evaluable patients and toxicity assessments from course 1 (28 days). We will require that at least 12 DLT evaluable subjects are assessed before the MTD/RP2D is declared. | 1 month after start of mirdametinib treatment | |
Primary | Phase 1: Determine the safety and tolerability of mirdametinib dosed twice daily on a continuous schedule in pediatric patients with progressive or recurrent low-grade glioma. | Incidence of adverse event data at least possibly related to treatment will be summarized in cohort specific tables by grade and attribution throughout treatment. | Up to 25 months after start of mirdametinib treatment | |
Primary | Characterize the maximum plasma concentration and area under the concentration-time curve (AUC0-8h) of mirdametinib. | Mirdametinib plasma concentration will be provided and area under the curve (AUC0-8h) estimated based on course 1, days 1 and 15 PK samples | Course 1: Days 1 and 15 | |
Primary | Phase 2, Cohort 1: Objective response rate observed anytime during active treatment and sustained for at least 8 weeks | The response rate, defined as the rate of minor response, partial response (PR), major response, or complete response (CR) will be calculated as the percentage of confirmed responders among all response assessable patients. These rates as well as their exact confidence intervals will be provided and will be summarized by each response category (i.e., PR, major response, and CR). Subjects without an assessment will be considered non-responders. | Up to 24 months after start of mirdametinib treatment | |
Primary | Phase 2, Cohort 2: Objective response rate observed anytime during active treatment and sustained for at least 8 weeks | The response rate, defined as the rate of minor response, partial response (PR), major response, or complete response (CR) will be calculated as the percentage of confirmed responders among all response assessable patients. These rates as well as their exact confidence intervals will be provided and will be summarized by each response category (i.e., PR, major response, and CR). Subjects without an assessment will be considered non-responders. | Up to 24 months after start of mirdametinib treatment | |
Primary | Phase 2, Cohort 3a: Estimate 1-year disease stabilization rate | Rate of stable disease from start of treatment until the time of progression or time of last follow-up. | Up to 12 months (slight departures from this timing allowed based on MRI screening) after start of mirdamentinib treatment | |
Primary | Phase 2, Cohort 3b: Estimate 6-month disease stabilization rate | Rate of stable disease from start of treatment until the time of progression or time of last follow-up. | Up to 6 months (slight departures from this timing allowed based on MRI screening) after start of mirdametinib treatment | |
Primary | Describe the toxicity profile of mirdametinib by cohort. | Incidence of adverse event data at least possibly related to treatment will be summarized in cohort specific tables by grade and attribution throughout treatment. | Up to 25 months after start of mirdametinib treatment | |
Secondary | Progression-free survival (PFS) by cohort | PFS will be measured using Kaplan Meier approaches from the date of initial treatment to the earliest date of progressive disease, death due to any cause, or date last follow-up. | Up to 5 years after the last enrolled patient starts treatment | |
Secondary | Rates of Minor Response, by cohort | Minor Response is defined as a 25-49% reduction in target lesion area relative to baseline measurements. Overall, the patient should be clinically stable or have improved on physical examination and functional or neurological assessment. Responses and duration will be measured for each cohort from the date of initial treatment to the earliest date of progressive disease, or data of last follow-up. | Up to 5 years after the last enrolled patient starts treatment | |
Secondary | Rates of Partial Response (PR), by cohort | Partial Response (PR) is defined as a 50-75% reduction in the target lesion area relative to baseline measurements. Overall, the patient should be clinically stable or have improved on physical examination and functional or neurological assessment. Responses and duration will be measured for each cohort from the date of initial treatment to the earliest date of progressive disease, or data of last follow-up. | Up to 5 years after the last enrolled patient starts treatment | |
Secondary | Rates of Major Response, by cohort | Major Response is defined as more than 75% reduction in target lesion area relative to baseline measurements. Overall, the patient should be clinically stable or have improved on physical examination and functional or neurological assessment. Responses and duration will be measured for each cohort from the date of initial treatment to the earliest date of progressive disease, or data of last follow-up. | Up to 5 years after the last enrolled patient starts treatment | |
Secondary | Rates of Complete Response (CR), by cohort | Complete Response is defined as disappearance of the target lesion and, if applicable, all areas of metastatic disease as compared to reference scan (baseline or best recorded MRI). Overall, the patient should be clinically stable or have improved on physical examination and functional or neurological assessment. Responses and duration will be measured for each cohort from the date of initial treatment to the earliest date of progressive disease, or data of last follow-up. | Up to 5 years after the last enrolled patient starts treatment | |
Secondary | Rates of Stable Disease, by cohort | Stable disease is defined as an increase or decrease in target lesion area that is not sufficient to qualify as progressive or responsive disease (i.e. minor, partial, major, or complete). Overall, the patient should be clinically stable or have improved on physical examination and functional or neurological assessment. Responses and duration will be measured for each cohort from the date of initial treatment to the earliest date of progressive disease, or data of last follow-up. | Up to 5 years after the last enrolled patient starts treatment | |
Secondary | Rates of Progressive Disease (PD), by cohort | Progressive Disease is defined as any of the following: A greater than 25% increase in target lesion area relative to reference scan, the development of a new tumor lesion, substantial growth (>25%) of a measurable metastatic lesion, or worsening of clinical and/or functional assessment directly related to tumor progression. Responses will be measured for each cohort from the date of initial treatment to the earliest date of progressive disease, or data of last follow-up. | Up to 5 years after the last enrolled patient starts treatment | |
Secondary | Longitudinal change in intellectual function, by cohort | To evaluate the effects of mirdametinib treatment on intellectual function among children treated for low-grade glioma. This will be assessed in children 2.6-5.11 years of age using Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV), in children 6-16.11 years of age using the Wechsler Intelligence Scale for Children (WISC-V), and for children 17 years and older using Wechsler Adult Intelligence Scale (WAIS-IV). Scores for each measure will be summarized by timepoint and cohort using descriptive statistics and appropriate plots. | Baseline (at enrollment), 9 months, 2 years, and 5 years after start of mirdametinib treatment | |
Secondary | Longitudinal change in attention and executive functions, by cohort | To evaluate the effects of mirdametinib treatment on attention and executive function in children treated for low-grade glioma. This will be assessed using different instruments as age appropriate: Cogstate will be used to measure attention, working memory and processing speed in children 4 years and older; WPPSI-IV (4-5.11 years of age), WISC-V (6-16.11 years of age) and WAIS-IV (17 years of age and older) will assess brief attention and working memory; Kaufman Test of Educational Achievement, Third Edition (KTEA-3) will assess verbal fluency in children 4-25 years of age; and Behavior Rating Inventory of Executive Function (BRIEF; BRIEF-2) will assess behavioral manifestations of executive function. Scores for each measure will be summarized by timepoint and cohort using descriptive statistics and appropriate plots. | Baseline (at enrollment), 6 months, 9 months, 2 years, 3 years, 4 years, and 5 years after start of mirdametinib treatment | |
Secondary | Longitudinal change in memory, by cohort | To evaluate the effects of mirdametinib treatment on memory in children treated for low-grade glioma. This will be assessed using different instruments as age appropriate: California Verbal Learning Test, Children's Version (CVLT-C) will be used to measure verbal list learning in children 6-16.11 years of age; the NEPSY-II Memory for Designs subtest (ages 6-16.11) and Wechsler Memory Scale (WMS-IV, ages 17 and older ) will assess nonverbal memory; the Children's Memory Scale (CMS, ages 5-16.11) and WMS-IV (ages 17 and older) Story Memory subtests will assess immediate recall, delayed recall, and recognition of details. Scores for each measure will be summarized by timepoint and cohort using descriptive statistics and appropriate plots. | 9 months, 2 years, and 5 years after start of mirdametinib treatment | |
Secondary | Longitudinal change in processing speed, by cohort | To evaluate the effects of mirdametinib treatment on processing speed in children treated for low-grade glioma. This will be assessed using the age appropriate Wechsler Processing Speed Index [WPPSI-IV (ages 4-5.11), WISC-V (ages 6-16), and WAIS-IV (ages 17 and older)]. Scores for each measure will be summarized by timepoint and cohort using descriptive statistics and appropriate plots. | 9 months, 2 years, and 5 years after start of mirdametinib treatment | |
Secondary | Longitudinal change in academic achievement, by cohort | To evaluate the effects of mirdametinib treatment on academic achievement in children treated for low-grade glioma. Reading and math abilities will be assessed using age-appropriate KTEA-3 subtests: Letter & Word Recognition (ages 4 and older), Word Recognition Fluency (ages 6 and older), Math Computation (ages 5 and older), and Math Fluency (ages 6 and older). Scores for each measure will be summarized by timepoint and cohort using descriptive statistics and appropriate plots. | 2 years and 5 years after start of mirdametinib treatment | |
Secondary | Longitudinal change in psychosocial functioning, by cohort | To evaluate the effects of mirdametinib treatment on psychosocial function in children treated for low-grade glioma. Social-emotional measures will include the Behavior Assessment System for Children, Third Edition (BASC-3, ages 2-20.11) to assess behavioral, emotional, and adaptive functioning, as well as the PedsQL (ages 2 and older) to assess health-related quality of life. Scores for each measure will be summarized by timepoint and cohort using descriptive statistics and appropriate plots. | Baseline (at enrollment), 6 months, 9 months, 2 years, 3 years, 4 years, and 5 years after start of mirdametinib treatment | |
Secondary | Longitudinal change in adaptive behavior, by cohort | To evaluate the effects of mirdametinib treatment on adaptive behavior in children treated for low-grade glioma. This will be assessed using the Adaptive Behavior Assessment System, Third Edition (ABAS-3, ages 2-21.11), which rates the child's ability to independently perform age-appropriate daily living skills. Scores will be summarized by timepoint and cohort using descriptive statistics and appropriate plots. | Baseline (at enrollment), 9 months, 2 years, and 5 years after start of mirdametinib treatment |
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