Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 … (NCT05309668) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
United States, Germany, Italy36 participantsStarted 2022-01-21
Plain-language summary
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints. The study will inform the benefit risk profile of the granule formulation in children aged ≥ 1 to \< 7 years with NF1 related symptomatic, inoperable PN.
Who can participate
Age range
1 Year – 6 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male and female participants aged ≥ 1 to \< 7 years of age at the time their legally authorised representative (parent or guardian) signs the informed consent.
. All study participants must be diagnosed with NF1 with symptomatic inoperable PN as defined in protocol.
. Participants must have at least one measurable PN, defined as a PN of at least 3 cm measured in one dimension, which can be seen on at least 3 imaging slices and have a reasonably well-defined contour. Participants who have undergone surgery for resection of a PN are eligible provided the PN was incompletely resected and is measurable. The target PN will be defined as the clinically most relevant PN, which is symptomatic, inoperable and measurable by volumetric MRI analysis.
. Performance status: Participants must have a Lansky performance of ≥ 70 except in participants who are wheelchair bound or have limited mobility secondary to a need for mechanical breathing support (such as an airway PN requiring tracheostomy or continuous positive airway pressure) who must have a Lansky performance of ≥ 40.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a Phase 1/2 trial testing a granule formulation of selumetinib specifically in children aged 1 to under 7, what does that mean for what's already known about safety compared to the tablet form that older children have used?
2The trial is actively measuring drug levels in the blood after a single dose — what does that tell us about where they are in understanding the right dose for this age group, and how might that uncertainty affect my child?
3The study is no longer enrolling new participants — does that mean results are coming soon, and could my child potentially access selumetinib through another pathway like compassionate use or a different trial instead?
4Given that this trial is focused on inoperable plexiform neurofibromas, what would my child's doctor be watching for in terms of adverse events graded on the CTCAE scale, and what side effects have been seen in older children taking selumetinib?
5Before considering this or a similar trial, should my child try any currently approved standard treatments first, and how would that choice affect eligibility for future studies?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Selumetinib AUC0-12 Derived After Single Dose Administration
Timeframe: Pre-dose and 1, 2, 3, 4, 6, 8 and 10-12 hours after selumetinib single dose on the first day of study treatment (Cycle 1 Day 1) (each cycle is 28 days)
2
Adverse Events Graded by CTCAE Ver 5.0
Timeframe: from screening until 30 days after last dose
. Participants must have a BSA ≥ 0.4 and ≤ 1.09 m2 at study entry (date of ICF signature).
. Mandatory provision of consent for the study signed and dated by a participant's legally authorised representative (parent or guardian) along with the paediatric assent form, if applicable.
Exclusion criteria
. Participants with confirmed or suspected malignant glioma or MPNST. Participants with low grade glioma (including optic glioma) not requiring systemic therapy are permitted.
. History of malignancy except for malignancy treatment with curative intent with no known active disease ≥ 2 years before the first dose of study intervention and of low potential risk of recurrence.
. Refractory nausea and vomiting, chronic gastrointestinal disease, inability to swallow the formulated product, or previous significant bowel resection that would preclude adequate absorption, distribution, metabolism, or excretion of selumetinib.
. A life-threatening illness, medical condition, organ system dysfunction or laboratory finding which, in the Investigator's opinion, could compromise the participant's safety, interfere with the absorption or metabolism of selumetinib, or put the study outcomes at undue risk.
. Participants with clinically significant cardiovascular disease as defined in the protocol.
. Liver function tests: Bilirubin \> 1.5 × the ULN for age with the exception of those with Gilbert syndrome (≥ 3 × ULN) or AST/ALT \> 2 × ULN.
. Renal Function: Creatinine clearance or radioisotope glomerular filtration rate \< 60 mL/min/1.73 m2 or Serum creatinine \> 0.8 mg/dL (for participants aged ≥ 1 to \< 4 years) or \> 1.0 mg/dL (for participants aged ≥ 4 years).
. Participants with ophthalmological findings/condition as listed in the protocol.