Phase 2 Peripheral Neuropathy Trial, Recruiting

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

Who might be able to join this trial:

Part 1 (Observation phase — all participants start here):

• Children who are older than 12 months and up to 8 years old at the time of joining the study
• Children who have been diagnosed with Neurofibromatosis Type 1 (NF1) based on the updated 2021 diagnostic guidelines
• Children who have not previously been found to have a plexiform neurofibroma (a specific type of nerve tumour associated with NF1) — children where there is some suspicion of one may still be considered after discussion with the Study Chair, provided no MRI of that area has been done and there are no symptoms
• Children who have had a physical exam at the enrolling hospital within the past year
• Children whose legal guardian is willing and able to provide written consent on their behalf

Part 2 (Monitoring and possible treatment phase — for those who completed Part 1):

• Children already enrolled in Part 1 who completed their full-body MRI scan within 6 weeks of joining Part 2
• Children found to have a measurable plexiform neurofibroma (at least 3 mL in size) located in a high-risk area, such as the head or neck, near major nerves in the shoulder or lower back, or near important structures like major blood vessels, airways, the spinal cord, or vital organs — this must be confirmed by the Study Committee
• Children with a body surface area of at least 0.55 m² (a measure of body size) (confirm with trial site regarding availability of an alternative formulation)
• Children who are physically active enough to meet a standard childhood activity score of 70% or above (children who use a wheelchair due to an unrelated NF1 complication, such as a bone or spine condition, are considered to meet this requirement)
• Children who are able to swallow whole capsules (confirm with trial site regarding availability of an alternative formulation)
• Children whose blood counts are within acceptable ranges: enough white blood cells, red blood cells, and platelets, without needing a transfusion
• Children whose liver function test results are within acceptable limits for their age (an exception applies for a harmless condition called Gilbert syndrome)
• Children whose kidney function is within acceptable limits for their age
• Children whose heart is functioning normally, with a pumping strength of at least 53% and a normal heart rhythm measurement on an ECG
• Children whose blood pressure is at or below the normal range for their age, height, and gender — blood pressure medication is acceptable if the child has been on a stable dose for at least 30 days
• Families who are willing to limit the child's sun exposure and use sunscreen when outdoors
• Families who are willing to avoid giving the child grapefruit, Seville oranges, or products made from these fruits during the study, as these can affect how the study medication works

Part 3 (Treatment phase — for those in Part 2 whose tumour grew or caused new symptoms):

• Children already in Part 2 whose plexiform neurofibroma grew by more than 20%, or who developed new symptoms related to it, during the observation period
• Children who continue to meet the same body size, physical activity, swallowing ability, blood count, liver, kidney, heart, and blood pressure requirements listed under Part 2 above
• Families who are willing to follow the same sun exposure and dietary restrictions listed under Part 2 above

Who may not be able to join:

Part 1:

• Children who already have a known plexiform neurofibroma that is causing symptoms, whether or not it has been seen on an MRI
• Children who have previously had a whole-body MRI scan (though children who have had a scan of a specific body region for a different reason, and no neurofibroma was found, may still be considered)
• Children who are unable to have an MRI scan, or for whom an MRI would be medically unsafe
• Children who have previously been treated with selumetinib or another medication from the same drug class (MEK1/2 inhibitors)
• Children who have a brain tumour affecting the optic pathway, another low-grade or high-grade brain tumour, a malignant nerve sheath tumour, or any other cancer currently requiring chemotherapy, targeted therapy, or radiation
• Children currently receiving radiation therapy, chemotherapy, hormone therapy targeting a tumour, immunotherapy, or other biological cancer treatments
• Children whose doctor determines it would not be appropriate for them to participate

Parts 2 and 3:

• Children with any of the tumour or cancer conditions listed above under Part 1 exclusions
• Children currently receiving the cancer treatments listed above under Part 1 exclusions
• Children who have a metal prosthesis, orthopaedic implant, or dental braces that would interfere with MRI measurements of the tumour
• Children who have taken an experimental drug or treatment within the past 30 days (Part 2 only)
• Children with a serious uncontrolled illness, an active infection, an active bleeding disorder, or a history of kidney transplant, or who are known to have hepatitis B, hepatitis C, or HIV
• Children who, in the opinion of the investigating doctor, may not be able to safely follow the study's monitoring and safety requirements
• Children with persistent severe nausea and vomiting, a chronic bowel condition (such as inflammatory bowel disease), or who have had significant bowel surgery that would affect the body's ability to absorb the study medication
• Children taking vitamin E supplements at more than 100% of the recommended daily amount — any multivitamin containing vitamin E would need to be stopped before starting the study medication
• Children whose blood pressure cannot be brought to an acceptable level even with blood pressure medication
• Children with certain heart conditions, including inherited coronary artery disease, symptomatic heart failure, a history of cardiomyopathy (weakened heart muscle), severe heart valve disease, or a history of atrial fibrillation (an irregular heart rhythm)
• Children with certain eye conditions, including a history of a specific type of fluid build-up behind the retina (central serous retinopathy or retinal pigment epithelial detachment), a history of a blocked vein in the retina, a history of radiation treatment that included the eye area, uncontrolled glaucoma or significantly raised eye pressure — though some exceptions may be considered by the Study Chair
• Children with a known severe allergy to selumetinib or any of its ingredients, or a history of serious allergic reactions to similar medications
• Children who have had major surgery within the 4 weeks before starting the study medication
• Children who still have unresolved side effects rated as moderate or above from a previous treatment (hair loss is an exception)
• Children currently taking herbal supplements or medications that are known to significantly affect how the body processes certain drugs (specifically strong or moderate inhibitors or inducers of CYP2C19 and CYP3A4 enzymes, or fluconazole), unless those can be safely stopped at least 14 days or 5 half-lives before starting the study medication — whichever is the longer period

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