A PHASE I/IIA STUDY OF 2-HYDROXYOLEIC ACID IN PEDIATRIC PATIENTS WITH MALIGNANT GLIOMA AND OTHER ADVANCED SOLID TUMORS

Objectives:

To determine the safety and tolerability of the orally administered investigational medication 2-OHOA (2-hydroxyoleic acid sodium salt) in patients less than 18 years old in phase 1 of the trial, and to identify the recommended dose of the medication in phase 2 of the trial. Additional objectives include describing how the medication acts in the body after one dose and after regular use while being administered daily, as well as evaluating the effects of the investigational medication on various bodily structures (i.e. proteins). Glioma patients and other solid tumor patients (including non-glial brain tumors) will be allowed to enroll on the study as positions are made available. No tumor type will be given priority over another, and there is no minimum number of glioma patients or solid tumor patients that must be enrolled on the trial. 

Summary:

This open label, non-randomized study is the first study of 2-OHOA in pediatric patients. High-grade gliomas (HGG) are relatively rare forms of pediatric brain tumors, and management involves surgical removal to the extent feasible, radiation, and chemotherapy. Despite these interventions, there has been a history of poor prognosis, and outcomes are far below what has been achieved in the treatment of other pediatric cancers. As a result, new therapeutic agents must be investigated to improve clinical outcomes. This investigational medication has demonstrated anti-tumor activity against several human glioma cell lines and a number of other solid tumors, as well as the inhibition of tumor cell growth in mice. It is believed that 2-OHOA may offer significant benefit to patients with glioma. Because pediatric glioma is relatively rare, and due to the effects of this medication demonstrated in non-clinical models of other tumor types, this study also includes pediatric patients with other advanced solid tumors who have failed standard therapy or for whom there is no standard therapy.

In addition to various exclusion criteria, there are some criteria participants must meet in order to be included in this study, including but not limited to: 

• Age: < 18 years
• Diagnosis: Must have a confirmed diagnosis of advanced solid malignancy that is progressive, recurrent, or refractory to standard-of-care treatment, or for which there is no standard therapy
• Must have an approved performance status (per protocol requirements)
• Must be able to swallow oral medications or have a gastric/nasogastric tube for medication administration
• Must be able to undergo adequate tumor imaging (CT, MRI, PET, MIBG, etc.) to evaluate disease evolution
• Must have adequate lab values/organ function (per protocol requirements)
• Must have a negative pregnancy test in women of childbearing potential (within 7 days of starting therapy)
• Must not breastfeed while receiving medications on study.

Clinicaltrials.gov Study Details >