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, Germany36 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 range1 Year – 6 Years
SexALL
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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.
✓. 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.
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
✕. 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.