Study of LAM561 Acid in Pediatric Patients With Malignant Glioma and Other Advanced Solid Tumors (NCT04299191) | Clinical Trial Compass
RecruitingPhase 1/2
Study of LAM561 Acid in Pediatric Patients With Malignant Glioma and Other Advanced Solid Tumors
United States28 participantsStarted 2020-09-01
Plain-language summary
An open label, non-randomized study in pediatric patients with advanced high-grade gliomas and other solid tumors.
The study will be performed in two phases: a dose escalation phase in up to 18 patients following a standard "3+3" design to establish dose-limiting toxicity (DLT) and a "safe" dose of LAM561 followed by an expanded safety cohort of up to 10 patients treated at the Maximum Tolerated Dose (MTD). If the MTD is well tolerated in the expanded safety cohort, that dose becomes the Recommended Phase 2 Dose (RP2D).
Glioma patients and other solid tumor patients (including non-glial brain tumors) will be treated as a single cohort. Patients with either tumor type 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.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Age \<18 years
✓. 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.
✓. Timing of therapy:
✓. 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.
✓. Able to swallow and ingest oral medication or have a NG or G-tube for drug administration
✓. 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
✓. Adequate hematologic, renal, liver function as demonstrated by laboratory values:
What they're measuring
1
Safety and Tolerability of LAM561
Timeframe: Between 1 to 6 cycles (each cycle is 3 weeks)
2
To identify the Recommended Phase 2 Dose (RP2D) of LAM561 in pediatric patients
Timeframe: Between 1 to 6 cycles (each cycle is 3 weeks)
✕. Known hypersensitivity to any component of the study drug
✕. Use of any other investigational drug within five half-lives of that drug prior to the first dose of LAM561
✕. 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
✕. Any surgery within 14 days prior to the first dose of LAM561 (excluding shunt or line insertion)
✕. 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
✕. 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
✕. 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)