Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Co… (NCT05825365) | Clinical Trial Compass
WithdrawnPhase 2
Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, Phaseâ…¡, Double-Blinded, Placebo-Controlled Study
Stopped: Selumetinib launch approval achieved in advance,strategy changesa new study will be generated in place of this study.
0Started 2023-08-31
Plain-language summary
This is a phase II, multicenter, randomised, parallel, double-blind, placebo-controlled study assessing the efficacy and safety of the MEKi selumetinib compared with placebo in Chinese paediatric participants with post-operative NF1-associated PNs.
Who can participate
Age range3 Years – 18 Years
SexALL
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Inclusion criteria
✓. Paediatric subjects: Subjects ≥3 years and \<18 years of age with a BSA ≥0.55 m2 at the time of study enrolment.
✓. All study participants must have either positive genetic testing for NF1 or have at least one other diagnostic criterion for NF1 listed below Six or more macules (≥0.5cm in greatest diameter in pre-pubertal participants or ≥1.5 cm in greatest diameter in post-pubertal participants)； Freckling in the axillary or inguinal regions； Optic pathway glioma; Two or more iris Lisch nodules identified by slit lamp examination or 2 or more choroidal abnormalities-defined as bright, patchy nodules imaged by optical coherence tomography/near-infrared reflectance imaging; A distinctive bony lesion (such as: sphenoid dysplasia, anterolateral bowing of the tibia, or pseudoarthrosis of a long bone); A NF1 heterozygous pathogenic variant with a variant allele fraction of 50% in apparently normal tissue such as white blood cells; A parent with NF1 by the above criteria.
✓. Symptomatic PNs:The PN has to cause or have the potential to cause significant clinical complications, as judged by the investigator, such as (but not limited to) head and neck lesions that could compromise the airway or great vessels, paraspinal lesions that can cause myelopathy brachial or lumbar plexus lesions that could cause nerve compression and loss of function, lesions that could result in major deformity (e.g., orbital lesions) orare significantly disfiguring, lesions of the extremity that cause limb hypertrophy or loss of function, and painful lesions.
✓. All study participants have received sub-total or partial resection of the targeted PN lesion (residue should be \> 20% of pre-operation volume, and measurable, defined as a lesion of ≥3 cm measured in one dimension on ≥3 imaging slices and have a reasonably well-defined contour), at least 4 weeks and up to 3 months after surgery.
✓. Performance status: Subjects \>16 years of age must have a Karnofsky performance level of ≥70, and children ≤16 years old must have a Lansky performance of ≥70 (Appendix G). 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 (Appendix G).
✓. All participants must be able to swallow whole capsules.
✓. Adequate organ and marrow function as follows with no blood transfusions (of red blood cells/ other blood products) within 28 days prior to screening assessment and no growth factors within 7 days prior to screening assessment:
✓. Reproduction:
Exclusion criteria
✕. Participants with confirmed or suspected malignant glioma or MPNST. Participants with low-grade gliomas (LGG) (including optic glioma) not requiring systemic therapy 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.
✕. Subjects with clinically significant cardiovascular disease as defined by the following:
✕. Subjects with the following ophthalmological findings/conditions:
✕. Have received or are receiving an IMP or other systemic PN target treatment (including chemotherapy, immunotherapy, targeted therapy, biologic therapy or monoclonal antibodies) within 4 weeks prior to the first dose of study treatment, or within a period during which the IMP or systemic PN target treatment has not been cleared from the body (e.g. a period of 5 'half-lives'), whichever is longer.
✕. Has received radiotherapy in the 6 weeks prior to the start of study intervention or any prior radiotherapy directed at the target or non-target PN.
✕. Inability to undergo MRI and/or contraindication for MRI examinations. Prosthesis or orthopaedic or dental braces in close proximity to the area of interest that would interfere with volumetric analysis of target PN on MRI.
✕. 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.