A Phase I/II, Single-Arm, Open-label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged >= 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) - SPRINKLE

Astra Zeneca0 个研究点目标入组 5 人开始时间: 待定最近更新: 2024年4月15日

适应症

试验速览

阶段: 2 期
状态: 已完成
发起方
入组人数: 5

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional

入排标准

年龄范围: 2 至 11（—）

入选标准

•- 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 participants must have a diagnosis of NF1 with symptomatic
•inoperable PN where: a) Participants must have PN and at least one other
•diagnostic criterion for NF1 b) Inoperable is defined as a PN that cannot be
•completely surgically removed without a risk of substantial morbidity due to
•encasement of, or close proximity to, vital structures, invasiveness, or high
•vascularity of the PN; or unacceptable risk from the general anaesthetic as
•assessed by the investigator c) Symptomatic is defined as clinically
•significant symptoms or complications caused by the PN, as judged by the
•investigator; symptoms may include, but are not limited to, pain, motor
•dysfunction and disfigurement (examples of complications include PN displacing
•trachea, or PN causing bladder obstruction and hydronephrosis). - 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. - 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, when

排除标准

•- Participants with confirmed suspected malignant glioma or MPNST. Participants
•with optic glioma not requiring chemotherapy or radiation 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 judged by the Investigator,
•any evidence of disease (such as severe or uncontrolled systemic disease, known
•moderate or severe hepatic impairment, active infection, active bleeding
•diatheses, or renal transplant, including any participant known to have
•hepatitis B, hepatitis C, or HIV) which, in the Investigator*s opinion, makes
•it undesirable for the participant to take part in the study. - Participants
•with the following ophthalmological findings/conditions: • Current or past
•history of RPED/CSR or RVO; • Intraocular pressure >21 mmHg (or ULN adjusted by
•age) or uncontrolled glaucoma (irrespective of IOP). • Participants with known
•glaucoma and increased IOP who do not have meaningful vision (light perception
•only or no light perception) and are not experiencing pain related to the
•glaucoma, may be eligible after discussion with the Medical Monitor. •
•Participants with any other significant abnormality on ophthalmic examination
•should be discussed with the Sponsor for potential eligibility. •
•Ophthalmological findings secondary to long*standing optic pathway glioma (such
•as visual loss, optic nerve pallor or strabismus) or longstanding orbito*
•temporal PN (such as visual loss, strabismus) will NOT be considered a
•significant abnormality for the purposes of the study. - Have any unresolved
•chronic toxicity with CTCAE Grade >= 2 which are associated with previous
•therapy for NF1*PN (except hair changes such as alopecia or hair lightening) -
•Participants who have previously been treated with a MEKi (including
•selumetinib) and have had disease progression, or due to toxicity have either
•discontinued treatment and/or required a dose reduction. - Have had major
•surgery within 4 weeks of the first dose of study intervention, with the
•exception of surgical placement for vascular access. Have planned major surgery
•during the treatment period. - Have received or are receiving an IMP or other
•systemic NF1*PN target treatment (including MEKi) within 4 weeks prior to the
•first dose of study intervention, or within a period during which the IMP or
•systemic PN target treatment has not been cleared from the body (eg, a period
•of 5 'half*lives'), whichever is longer. - Has received radiotherapy in the 6
•weeks prior to start of study intervention or any prior radiotherapy directed
•at the target or non*target PN. - Has received growth facto

研究者

发起方

Astra Zeneca

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