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A Phase 1/2A Study of LAM561 in Adult Patients With Advanced Solid Tumours

Phase 1
Completed
Conditions
Glioma
Other Solid Tumours
Interventions
Registration Number
NCT01792310
Lead Sponsor
Laminar Pharmaceuticals
Brief Summary

This is a phase 1/2A, open label, non-randomized study in patients with advanced solid tumours including malignant glioma

Detailed Description

This is an open label, non-randomized study in patients with advanced solid tumours including malignant glioma. The study will be performed in two phases - a dose escalation phase following a standard "3+3" design to establish dose-limiting toxicity (DLT) and a safe dose of LAM561 followed by two expanded safety cohorts (approximately 10 of whom have malignant glioma and approximately 10 of whom have other advanced solid tumours that are suitable for biopsy) treated at the maximum tolerated dose (MTD). If the MTD is well tolerated in the expanded safety cohorts, that dose becomes the recommended phase 2 dose (RP2D). During each dose cohort, at least one week must elapse between the first and subsequent patients receiving treatment with LAM561. Patients may receive palliative localized radiotherapy, if needed (however, this lesion cannot be a target lesion for evaluation of the treatment response).

Safety, pharmacokinetics (PK), pharmacodynamics and efficacy will be evaluated during the study at pre-defined timepoints

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
54
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
LAM561 Dose Cohort 7LAM561Intervention: LAM561. 8g twice daily
Dose Cohort 1LAM561Intervention: LAM561. 7 dose cohorts of up to 6 patients have been performed in the dose escalation phase. The starting dose cohort received 250 mg twice daily.
Dose Cohort 2LAM561Intervention: LAM561. 500 mg twice daily
Dose Cohort 3LAM561Intervention: LAM561. 1g twice daily
LAM561 Dose Cohort 4LAM561Intervention: LAM561. 2g twice daily
LAM561 Dose Cohort 5LAM561Intervention: LAM561. 4g twice daily
LAM561Dose Cohort 6LAM561Intervention: LAM561. 4g three times daily
LAM561 Dose Expansion cohort. GliomaLAM561Intervention: LAM561 at the MTD: 4g three times daily. Up to 10 patients with malignant glioma.
LAM561 Dose Expansion cohort. Non-gliomaLAM561Intervention: LAM561 at the MTD: 4g three times daily. Up to 10 patients with other advanced solid tumours that are suitable for biopsy.
Primary Outcome Measures
NameTimeMethod
Number of patients with adverse eventsFrom the first dose of study drug until 30 days after the last dose of study drug

All adverse events will be recorded including clinically significant physical examinations and vital signs, laboratory safety tests and 12-lead electrocardiograms

Secondary Outcome Measures
NameTimeMethod
Concentration of LAM561 in blood measured by LC-MS/MS21 days

Full profiles on Day 1 and Day 21 (dose escalation phase only), trough measurements on Day 8, 15 and 21

Concentration of micro RNA in bloodFirst 22 days then every 9 cycles until any criterion for discontinuation is met (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion)

Blood samples for future analysis of micro RNA

Radiological disease progressionEvery 6 weeks until any criterion for discontinuation is met (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion)

Measurement by CT or MRI scan. Changes scored according to Response Assessment in Neuro-oncology (RANO) criteria (for glioma patients) or Response evaluation criteria in solid tumours (RECIST v1.1) (for other solid tumour patients).

Concentration of biomarkers in blood or tumour tissueFirst 22 days then every 9 cycles until any criterion for discontinuation is met (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion)

Effect on glial fibrillary acidic protein in glioma patients and effect on sphingomyelin and dihydrofolate reductase in patients with other solid tumours

Clinical disease progressionuntil any criterion for discontinuation is met (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion

Trial Locations

Locations (5)

Vall D'Hebron Institute of Oncology

🇪🇸

Barcelona, Spain

Instituto Oncológico IMQ, Clínica IMQ Zorrotzaurre

🇪🇸

Bilbao, Spain

Onkologikoa

🇪🇸

San Sebastián, Spain

The Royal Marsden Hospital Drug Development Unit

🇬🇧

Sutton, Surrey, United Kingdom

Sir Bobby Robson Cancer Trials Research Centre, The Northern Centre for Cancer Care, Freeman Hospital

🇬🇧

Newcastle, Newcastle Upon Tyne, United Kingdom

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