NCT04299191 · Laminar Pharmaceuticals
Study of LAM561 Acid in Pediatric Patients With Malignant Glioma and Other Advanced Solid Tumors
What this study is about
An open label, non-randomly assigned study in pediatric patients with advanced high-grade gliomas and other solid tumors.
View original scientific description
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).
Interventions
DRUG
LAM561
Once a patient is allocated a dose of LAM561, they will receive the same dose on a daily basis in treatment cycles of 21 days (3 weeks), which may be repeated without therapy interruption until a criterion for discontinuation (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" criterion) is met. The starting dose will be 2.8 g/m2 twice daily. If tolerated, doses will be escalated to 3.5 g/m2 twice daily and then to a third dose level of 4.2 g/m2 twice daily. These dose levels correspond to 80%, 100%, and 120% of the maximum tolerated dose of LAM561 in adult patients when adjusted for body surface area. A total of 3 dose cohorts are anticipated for the dose escalation phase of the study, with up to 6 patients enrolled at each dose level according to a standard "3+3" design. During each dose cohort, at least 1 week must elapse between the first and subsequent patients receiving treatment with LAM561.
Primary outcome measures
Safety and Tolerability of LAM561
Time frame: Between 1 to 6 cycles (each cycle is 3 weeks)
To determine the safety and tolerability of LAM561 in pediatric patients (under 18 years) when administered orally using a continuous dosing schedule. It will be evaluated through the adverse events \[AEs\], physical examinations and vital signs, laboratory safety tests and 12-lead electrocardiograms \[ECGs\].
To identify the Recommended Phase 2 Dose (RP2D) of LAM561 in pediatric patients
Time frame: Between 1 to 6 cycles (each cycle is 3 weeks)
The RP2D of LAM561 in pediatric patients will be the Maximum Tolerated Dose during the safety cohort and it's well tolerared.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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%, correspon
Where
- Little Rock, Arkansas
- Miami, Florida
- Edison, New Jersey
Collaborators
Hackensack Meridian Health, Dana-Farber Cancer Institute, Laminar Pharma Inc
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Nov 20, 2025 · Source of record for eligibility and locations