Study of LAM561 Acid Treatment in Children Under 18 With Malignant Glioma and Other Advanced Solid Tumors

What this Trial Is About

Researchers are studying LAM561 acid in children with malignant glioma and other advanced solid tumors that are progressive, recurrent, or resistant to standard treatments. This open-label, non-randomized clinical trial aims to find the safest dose and evaluate the safety and tolerability of LAM561, as well as identify the recommended dose for further studies. The study includes patients under 18 years with confirmed advanced solid tumors or certain brain tumors, without prioritizing tumor types within enrollment. Participants will receive LAM561 orally in daily doses during treatment cycles lasting 21 days, repeated continuously until disease progression, unacceptable side effects, or other criteria require stopping treatment. The dose may start at 2.8 gm2 twice daily and may be increased in steps up to 4.2 gm2 twice daily, adjusted for body surface area. Treatment interruptions, dose reductions, or intermittent dosing schedules may be used if needed due to side effects. The study has a dose escalation phase followed by an expanded safety group to confirm the recommended dose. During the study, patients will undergo regular safety monitoring, laboratory tests, and tumor imaging to assess treatment effects. Investigators will evaluate how well patients tolerate the drug and monitor for any toxicities. Pharmacokinetic profiles will be characterized during the first two cycles, and anti-tumor effects will be assessed every 12 weeks. Patients may receive 1 to 6 cycles of treatment, with the possibility of extension if benefits are seen. Those benefiting may continue treatment through compassionate use after the study ends.

Study of LAM561 Acid in Pediatric Patients With Malignant Glioma and Other Advanced Solid Tumors