A Phase II, multi-center, open-label, single-arm study of the efficacy and safety of oral LDE225 in patients with Hh-pathway activated relapsed medulloblastoma - LDE225 in patients with Hh+ relapsed medulloblastoma

ovartis Pharma B.V.0 个研究点目标入组 2 人开始时间: 待定最近更新: 2024年4月29日

适应症

试验速览

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•Patients aged * 4 months
•Patients with histologically confirmed diagnosis of MB, who have experienced relapse or progression after standard-of-care therapy including radiotherapy or patients aged >4 months and * 6 years who are RT naive.
•Patients currently receiving steroids must have been on a stable (or decreasing) dose for at least 5 days before the brain/spine MRI obtained at screening.
•Patients with any number of prior relapses are eligible to enroll provided they have Hh-pathway activated tumors as assessed using the 5-gene Hh signature assay.
•Relapsed MB may be defined by imaging tumor biopsy, or evidence of tumor cells in the CSF.
•At least one measurable lesion.

•Prior treatment with a Smoothened inhibitor
•Patients who have neuromuscular disorders that are associated with elevated CK
•Patients on concomitant treatment with drugs that are recognized to cause rhabdomyolysis that cannot be discontinued at least 2 weeks before first dose of study treatment. If it is essential that the patient stays on a statin to control hyperlipidemia only pravastatin may be used with extra caution.
•Patients receiving treatment with medications that are known to be strong inhibitors or inducers of CYP3A4/5 or are metabolized by CYP2B6 and CYP2C9, that have narrow therapeutic indices that cannot be discontinued at least 2 weeks before first dose of study treatment and for the duration of the study.
•Patients receiving unstable or increasing doses of corticosteroids. If patients are on corticosteroids for endocrine deficiencies or tumor-associated symptoms, dose must have been stabilized (or decreasing) for at least 5 days before the brain/spine MRI obtained at screening.
•Patients receiving treatment with any enzyme-inducing anticonvulsant that cannot be discontinued at least 2 weeks before first dose of study treatment, and for the duration of the study. Patients on non-enzyme-inducing anticonvulsants are eligible.

发起方

ovartis Pharma B.V.