Phase 2 Brain Cancer Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who have been diagnosed with, or are suspected to have, a low-grade brain tumour (a slow-growing type of brain tumour), including certain types of neuronal or mixed brain tumours, confirmed by a specialist review of tumour tissue
• People whose tumour tissue is available from either the original diagnosis or a later recurrence, so that a central specialist team can review it
• People aged 2 years and older and under 25 years at the time of joining (for Phase 1); people aged 2 years and older and under 25 years at the time of joining (for Phase 2)
• People whose body surface area (a measurement based on height and weight) falls within the range required for the specific dose being studied (confirm with trial site for exact ranges; upper limits are removed in Phase 2)
• People whose tumour has been confirmed as one of a specific list of low-grade brain tumour types by the St. Jude Children's Research Hospital central pathology team (this includes types such as pilocytic astrocytoma, ganglioglioma, diffuse glioma, and many others — confirm with trial site for the full list)
• People whose tumour shows signs of a specific biological pathway being active (called the MAPK pathway), shown through specialist laboratory testing, or who have a known inherited genetic condition affecting this pathway (such as NF1)
• People who have measurable or assessable disease on imaging or clinical assessment (those with spread of tumour or multiple tumours may also be considered)
• People taking steroid medications who have been on a stable or reducing dose for at least one week before joining, with no planned increases
• People with a performance score of 50 or above on the standard scales used for children and young people (a measure of how well someone is functioning day-to-day), and who are expected to live at least six weeks
• People with adequate blood counts, without recent transfusions or growth factor support (specific blood test thresholds apply — confirm with trial site)
• People with electrolyte levels (such as potassium, calcium, and magnesium) that are within acceptable limits, or that can be corrected with supplements before starting treatment
• People with liver enzyme levels and bilirubin (a liver-related substance) within acceptable ranges
• People with kidney function within acceptable limits based on age and sex (specific creatinine thresholds apply — confirm with trial site)
• People with adequate heart function, including a heart pumping measurement above 50% and a normal heart rhythm measurement (specific thresholds apply — confirm with trial site)
• People with blood pressure within acceptable limits for their age, height, and sex (blood pressure medication is permitted)
• People who are capable of having children and who agree to use contraception during the trial and for 16 weeks after stopping treatment
• People (and/or their guardian) who are willing and able to sign a consent form

For Phase 1 only (progressive or recurrent tumour, no prior MEK inhibitor treatment):

• People whose tumour has clearly grown back or worsened during or after their most recent treatment (chemotherapy or radiotherapy), confirmed by imaging or clinical signs
• People who have received no more than three prior treatment courses involving chemotherapy or biological agents, and/or focal radiotherapy
• People who have not received any MEK inhibitor medicines previously
• People whose last chemotherapy dose was at least 21 days before joining (or at least 42 days for a type called nitrosourea)
• People whose last dose of a monoclonal antibody or long-acting biological agent was at least 28 days before joining, and who have recovered from any side effects
• People whose last radiotherapy treatment was at least 3 months before joining

For Phase 2, Cohort 1 (newly diagnosed or previously untreated, except surgery):

• People who have had no prior cancer treatment other than surgery
• People whose tumour, in the treating doctor's opinion, requires treatment — for example, because it is unsafe to monitor, is clearly growing on scans, or is causing or is at high risk of causing problems with brain function or vision

For Phase 2, Cohort 2 (progressive or recurrent tumour, no prior MEK inhibitor treatment):

• People whose tumour has clearly grown back or worsened during or after their most recent treatment, confirmed by imaging or clinical signs
• People who have not received any MEK inhibitor medicines previously
• People whose last chemotherapy dose was at least 21 days before joining (or 42 days for nitrosourea), whose last biological agent dose was at least 28 days before joining with side effects resolved, and whose last radiotherapy was at least 3 months before joining
• There is no limit on the number of prior treatments for this group, and prior whole-brain-and-spine radiotherapy is permitted

For Phase 2, Cohort 3A (prior MEK inhibitor treatment — tumour responded):

• People who previously received at least 6 cycles of a MEK inhibitor (including mirdametinib) and whose tumour did not grow while actively taking that medicine, but whose tumour has since clearly grown back after stopping the MEK inhibitor
• People who did not stop their previous MEK inhibitor due to unacceptable side effects, and who the treating doctor believes could tolerate further MEK inhibitor treatment
• People whose last chemotherapy was at least 21 days before joining (or 42 days for nitrosourea), whose last biological agent was at least 28 days before joining with side effects resolved, whose last MEK inhibitor dose was at least 3 weeks before joining, and whose last radiotherapy was at least 3 months before joining
• There is no limit on the number of prior treatments, and prior whole-brain-and-spine radiotherapy is permitted

For Phase 2, Cohort 3B (prior MEK inhibitor treatment — tumour did not respond):

• People who previously received a MEK inhibitor other than mirdametinib and whose tumour clearly grew or worsened while actively taking that medicine
• People regardless of how many prior cycles of MEK inhibitor were received or whether there was any prior response
• People whose last chemotherapy was at least 21 days before joining (or 42 days for nitrosourea), whose last biological agent was at least 28 days before joining with side effects resolved, whose last MEK inhibitor (other than mirdametinib) was at least 3 weeks before joining, and whose last radiotherapy was at least 3 months before joining
• There is no limit on the number of prior treatments, and prior whole-brain-and-spine radiotherapy is permitted

Who may not be able to join:

• People whose tumour is classified as high-grade (WHO grade III or IV) on specialist review
• People whose tumour is a specific type not eligible for this trial, including subependymal giant cell astrocytoma, ependymoma, or tumours with certain genetic changes (Histone H3 K27M/K28M or G34/G35 mutation, BRAF V600 mutation, NTRK1/2/3, ALK, or ROS1 fusion, or IDH 1/2 mutation)
• People currently receiving any other cancer treatment or investigational medicine, or who are still recovering from side effects of a previous treatment (one specific imaging agent is permitted — confirm with trial site)
• People with known current eye conditions affecting the retina, including central serous retinopathy, retinal vein occlusion, retinal detachment, or uncontrolled glaucoma (including eye pressure above acceptable limits if testing is feasible)
• People with known eye conditions that could be early signs of or lead to central serous retinopathy, retinal vein occlusion, or a type of macular degeneration
• People with a known condition that affects how the body absorbs medicines through the gut, such as having had gastric bypass surgery or similar stomach procedures
• People with a known history of significant liver disease or liver and bile duct problems (except for a mild inherited condition called Gilbert's syndrome, or gallstones without symptoms)
• People with a significant history of serious chronic lung disease (such as certain long-term lung conditions affecting lung tissue); people with a history of asthma or similar airway conditions that have resolved or are well-controlled are not excluded
• People with other significant medical conditions (such as serious infections or significant heart, lung, liver, psychiatric, or other organ problems) that the treating doctor believes could affect their ability to safely receive or absorb the study treatment, or that could interfere with the study
• People who are sexually active and capable of having children but have not agreed to use effective contraception during the trial and for 16 weeks after stopping treatment
• People who are unable to follow the study procedures and guidelines
• People currently taking certain medicines that affect how mirdametinib is processed by the body (called P-gp and BCRP inhibitors), unless those medicines were last taken at least one week or five half-lives before starting the study drug (whichever is longer)

Important: Always verify eligibility with the trial site directly before applying.