FDA Issues ODD for Gallium Maltolate for Pediatric GBM

FDA Issues ODD for Gallium Maltolate for Pediatric GBM

Imaging Biometrics announced that gallium maltolate (GaM) has received orphan drug designation (ODD) from the FDA for the treatment of pediatric patients with glioblastoma multiforme (GBM). This designation comes after GaM was previously granted ODD for adult patients with GBM in February 2023. A preclinical study of oral GaM in pediatric GBM patients demonstrated a significant survival benefit, with a median overall survival duration more than doubling compared to untreated controls. The easy administration of GaM through capsules suggests its potential benefit for both pediatric and adult patients.

Currently, a Phase I study (NCT04319276) is underway, evaluating the preliminary efficacy and safety of GaM in patients with relapsed/refractory GBM. The trial will enroll 24 patients in a dose-escalation part using a 3 + 3 Phase I design. Patients will receive GaM at doses of 500 mg every other day, 1,000 mg daily, or 1,500 mg daily.

Eligible patients must be 18 years or older with a previous histological diagnosis of GBM and have undergone standard treatment with radiotherapy and temozolomide (Temodal). They should also have an ECOG performance status of 0-2, measurable disease, adequate bone marrow function, and sufficient hepatic and renal function. Patients taking oral iron supplements or iron chelators must discontinue them at least one week before starting GaM as they may impact its efficacy.

Female patients need to be postmenopausal for at least one year, surgically sterile, agree to use two acceptable methods of contraception, or practice abstinence. Male patients must agree to use effective barrier contraception during the study and for 60 calendar days after the last dose of GaM, or practice abstinence.

The study will exclude patients with other active malignant diseases diagnosed within the last 12 months, those who received chemotherapy or radiotherapy within 14 days before study entry, and individuals with known hypersensitivity to gallium-based medications. Concurrent use of cytotoxic chemotherapy, unstable or severe concurrent medical conditions, and a history of certain respiratory conditions like interstitial lung disease, sarcoidosis, idiopathic pulmonary fibrosis, and others will also be excluded.

The primary endpoint of the study is to determine the maximum-tolerated dose, while secondary endpoints include progression-free survival, overall survival, and dose-limiting toxicity.

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