New hope for kids with brain cancer? LAM561 trial launches

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Age \<18 years 2. Diagnosis: Patients must have a histologically- or cytologically-confirmed advanced solid malignancy that is progressive, recurrent or refractory to standard-of-care treatment, or for which there is no standard therapy. Examples of tumors that lack a standard therapy include, but are not limited to, high-grade glioma, diffuse midline glioma, and diffuse intrinsic pontine glioma. For patients with a radiographic diagnosis of diffuse midline glioma or diffuse intrinsic pontine glioma, histologic or cytologic confirmation of their diagnosis is not required. 3. Timing of therapy: * Patients must be enrolled before treatment begins. Treatment must start within 14 days of study enrollment. * All clinical and laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated in the eligibility section. 4. Patients must have a Lansky or Karnofsky performance status score of ≥ 50%, corresponding to ECOG categories of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients ≤ 16 years of age. Patients who are unable to walk because of paralysis, but who are up in a wheelchair will be considered ambulatory for the purpose of assessing the performance score. 5. Able to swallow and ingest oral medication or have a NG or G-tube for drug administration 6. Able to undergo adequate tumor imaging, via computerized tomography (CT) or magnetic resonance imaging (MRI) scans or any other standardized tumor assessment method based on tumor type (PET, MIBG, etc) to evaluate disease evolution 7. Adequate hematologic, renal, liver function as demonstrated by laboratory values: * ANC ≥ 1,000/ul * Hemoglobin ≥8.0 gm/dl * Platelet count ≥ 100,000/ul * Adequate Liver Function Defined As * Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age, and * SGPT (ALT) \< 2.5 x upper limit of normal (ULN) for age. 8. Adequate Renal Function Defined As Either * Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73m2 * or a serum creatinine less than or equal to the institutional normal for age 9. No history of QTc prolongation, and a normal QTc interval at screening/baseline (QTc ≤450 msec) 10. No evidence of a bleeding diathesis 11. Negative pregnancy test in women of childbearing potential within 7 days of initiating investigational therapy 12. Patient or legal guardian must give written, informed consent or assent (when applicable) - 13. Recent mothers must agree not to breast feed while receiving medications on study. Exclusion Criteria: 1. Age ≥ 18 years 2. Known hypersensitivity to any component of the study drug 3. Use of any other investigational drug within five half-lives of that drug prior to the first dose of LAM561 4. Any National Cancer Institute (NCI) Common Toxicity Criteria for Adverse Events (CTCAE version 4.0) \>Grade 1 toxicities from prior chemotherapy or radiotherapy that could impact on safety outcome assessment 5. Any surgery within 14 days prior to the first dose of LAM561 (excluding shunt or line insertion) 6. Known \>Grade 1 intracranial or intratumoral hemorrhage either by CT or MRI scan within the last 1 month. Patients with resolving hemorrhage changes, punctuate hemorrhage or hemosiderin may enter the study 7. A history of significant or uncontrolled cardiovascular disease, including New York Heart Association Class III-IV heart failure, a left ventricular ejection fraction which is clinically significantly abnormal as measured by 2-dimensional (2-D) echocardiogram or Multi Gated Acquisition(MUGA) scan, unstable angina or myocardial infarction within the preceding 6 months 8. Known impairment of gastrointestinal (GI) function that could alter the absorption of study drug (e.g. active Crohn's disease, malabsorption syndrome or states, unresolved diarrhea, small bowel resection or gastric by-pass surgery) 9. Patients who are unable to take oral medications because of significant uncontrolled vomiting will be excluded. 10. A history of uncontrolled hyperlipidemia and/or the need for concurrent lipid lowering therapy 11. Concurrent severe and/or uncontrolled other medical disease (e.g. uncontrolled diabetes mellitus, active uncontrolled infection) that could compromise participation in the study 12. Need for warfarin, phenytoin or sulphonylureas (glibenclamide, glimepiride, glipizide,glyburide or nateglanide) 13. Any serious and/or unstable pre-existing medical, psychiatric or other condition which in the Investigator's opinion could interfere with subject safety, obtaining written informed consent, or compliance with the study protocol 14. Pregnant female patients are not eligible for this study. Pregnancy tests with a negative result must be obtained in all post-menarchal females. 15. Lactating females must agree they will not breastfeed a child while on this study. 16. Males and females of reproductive potential may not participate unless they agree to use an effective contraceptive method and continue to do so for at least 6 months after the completion of therapy.