GliomaPhase 1/2NCT01792310
A Phase 1/2A Study of LAM561 in Adult Patients With Advanced Solid Tumours
A Phase 1/2 study testing LAM561 in people with Glioma and Other Solid Tumours, run by Laminar Pharmaceuticals at 5 sites in 2 countries.
Trial summary
This is a phase 1/2A, open label, non-randomized study in patients with advanced solid tumours including malignant glioma Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glioma, Other Solid Tumours
- What is being tested?
- LAM561
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 54 participants
- Where
- 5 sitesin 2 countries
- Study dates
- May 2013to Sep 2016
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males or females providing written, informed consent
- Histologically- or cytologically-confirmed advanced solid malignancy that is refractory to standard-of-care treatment, or for which there is no standard therapy. If this is glioma:Grade III / Grade IV malignant glioma recurring or progressing after first or second line standard-of-care treatment and true progressive disease, confirmed according to the RANO criteria 4.
- Life-expectancy of at least 12 weeks
- Eastern cooperative oncology group (ECOG) performance status of 0-2
- Safety laboratory tests and ECGs within specified limits.
- Using adequate contraception, where applicable
- Presence of lesions suitable for biopsy (mandatory for non-glioma patients enrolled in the expanded safety cohort and highly desirable for non-glioma patients enrolled in the dose escalation phase)
Who cannot join
- Anti cancer therapy within 4 weeks (6 weeks for mitomycin and nitrosureas and 2 weeks for palliative radiotherapy)
- NCI Common terminology criteria for adverse events (CTCAE) >Grade 1 toxicities from prior chemotherapy or radiotherapy that could impact on safety outcome assessment
- Recent >Grade 1 intracranial or intratumoural haemorrhage either by CT or MRI scan. Patients with resolving haemorrhage changes, punctuate haemorrhage or haemosiderin may enter the study
- Significant or uncontrolled cardiovascular disease, unstable angina or myocardial infarction within the preceding 6 months
- Known impairment of GI function that could alter the absorption of study drug
- History of uncontrolled hyperlipidemia and/or the need for concurrent lipid lowering therapy
Show all 9Show fewer
- Concurrent severe and/or uncontrolled other medical disease that could compromise participation in the study
- Taking warfarin, phenytoin or sulphonylureas (glibenclamide, glimepiride, glipizide, glyburide or nateglanide)
- Pregnant or breast feeding Other protocol specific criteria may apply
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
Spain3 sites
Vall D'Hebron Institute of Oncology
Barcelona
Instituto Oncológico IMQ, Clínica IMQ Zorrotzaurre
Bilbao
Onkologikoa
San Sebastián
United Kingdom2 sites
Sir Bobby Robson Cancer Trials Research Centre, The Northern Centre for Cancer Care, Freeman Hospital
Newcastle, Newcastle Upon Tyne, NE7 7DN
The Royal Marsden Hospital Drug Development Unit
Sutton, Surrey, SM25PT
About this study
Official title: A Phase 1/2A Dose Escalation Study of LAM561 in Adult Patients With Advanced Solid Tumours Including Malignant Glioma
This is a phase 1/2A, open label, non-randomized study in patients with advanced solid tumours including malignant glioma
Read the 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
- Conditions
- Glioma, Other Solid Tumours
- Being tested
- LAM561
- Keywords
- Glioma, Solid Tumours, LAM561
- Medical subject headings
- Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LAM561Drug
Patients will receive treatment cycles of 21 days, until any criterion for discontinuation (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion) is met.
Patients are expected to receive between one and 6 cycles of treatment. The treatment period may be extended if clinical benefit is shown.
In the event of significant GI toxicity, the treatment schedule may be modified from continuous dosing to an intermittent regime In the case of toxicity, the dose of LAM561 may be reduced or delayed by up to 14 days at the discretion of the Investigator. A maximum of two dose reductions will be permitted per patient. Treatment "holidays" of no more than 14 days are also permitted for reasons other than toxicity. Intra-patient dose escalation may be permitted in certain specific circumstances.
| Study group | Role | What they receive |
|---|---|---|
| Dose Cohort 1 | Experimental | Intervention: 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. Receives: Drug: LAM561 |
| Dose Cohort 2 | Experimental | Intervention: LAM561. 500 mg twice daily Receives: Drug: LAM561 |
| Dose Cohort 3 | Experimental | Intervention: LAM561.
1g twice daily Receives: Drug: LAM561 |
| LAM561 Dose Cohort 4 | Experimental | Intervention: LAM561. 2g twice daily Receives: Drug: LAM561 |
| LAM561 Dose Cohort 5 | Experimental | Intervention: LAM561. 4g twice daily Receives: Drug: LAM561 |
| LAM561Dose Cohort 6 | Experimental | Intervention: LAM561. 4g three times daily Receives: Drug: LAM561 |
| LAM561 Dose Cohort 7 | Experimental | Intervention: LAM561. 8g twice daily Receives: Drug: LAM561 |
| LAM561 Dose Expansion cohort. Glioma | Experimental | Intervention: LAM561 at the MTD: 4g three times daily. Up to 10 patients with malignant glioma. Receives: Drug: LAM561 |
| LAM561 Dose Expansion cohort. Non-glioma | Experimental | Intervention: LAM561 at the MTD: 4g three times daily. Up to 10 patients with other advanced solid tumours that are suitable for biopsy. Receives: Drug: LAM561 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with adverse events
All adverse events will be recorded including clinically significant physical examinations and vital signs, laboratory safety tests and 12-lead electrocardiograms
Measured: From the first dose of study drug until 30 days after the last dose of study drug
Secondary outcomes (5)
Concentration of LAM561 in blood measured by LC-MS/MS
Full profiles on Day 1 and Day 21 (dose escalation phase only), trough measurements on Day 8, 15 and 21
Measured: 21 days
Concentration of biomarkers in blood or tumour tissue
Effect on glial fibrillary acidic protein in glioma patients and effect on sphingomyelin and dihydrofolate reductase in patients with other solid tumours
Measured: First 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)
Concentration of micro RNA in blood
Blood samples for future analysis of micro RNA
Measured: First 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)
Radiological disease progression
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).
Measured: Every 6 weeks until any criterion for discontinuation is met (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion)
Clinical disease progression
Measured: until any criterion for discontinuation is met (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2013 (estimated)
- Study startMay 2013
- Primary completionSep 2016
- Study completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for GliomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 54 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laminar Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Specialized Medical Services (SMS)-Oncology BV, Royal Marsden NHS Foundation Trust, Northern Institute for Cancer Research, Newcastle, Vall d'Hebron Institute of Oncology, Instituto Oncológico IMQ, Clínica IMQ Zorrotzaurre. Bilbao, Onkologikoa, San Sebastián.
- Responsible party
- Sponsor
- Investigators
- Professor Johann de Bono, MB ChB FRCP MSc PhD, Study chair, The Institute of Cancer Research, 15 Cotswold Road, Belmont, Sutton, Surrey, United Kingdom SM2 5NGProf. Ruth Plummer, BMBCh, MRCP, Cert Me, Principal investigator, Northern Institute for Cancer Research, NewcastleDr Jordi Rodon, Principal investigator, Vall d'Hebron Institute of OncologyDr Juanita Lopez, Principal investigator, The Institute of Cancer Research, 15 Cotswold Road, Belmont, Sutton, Surrey, United Kingdom SM2 5NGDr Ricardo Fernandez Rodriguez, Principal investigator, Instituto Oncológico IMQ, Clínica IMQ Zorrotzaurre. BilbaoDr Ander Urruticoechea Ribate, Principal investigator, Onkologikoa, San Sebastián.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01792310
- Sponsor's protocol ID
- MIN-001-1203
- Other IDs
- EudraCT 2012-001527-13
- Status last verified
- Feb 2023
- First submitted
- 24 Dec 2012
- Last updated
- 21 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
Publications
Lopez J, Lai-Kwon J, Molife R, Welsh L, Tunariu N, Roda D, Fernandez-Garcia P, Llado V, McNicholl AG, Rossello CA, Taylor RJ, Azaro A, Rodon J, Sludden J, Veal GJ, Plummer R, Urruticoechea A, Lahuerta A, Mujika K, Escriba PV. A Phase 1/2A trial of idroxioleic acid: first-in-class sphingolipid regulator and glioma cell autophagy inducer with antitumor activity in refractory glioma. Br J Cancer. 2023 Sep;129(5):811-818. doi: 10.1038/s41416-023-02356-1. Epub 2023 Jul 24.
PubMed 37488446
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2A Dose Escalation Study of LAM561 in Adult Patients With Advanced Solid Tumours Including Malignant Glioma
- ClinicalTrials.gov ID
- NCT01792310
- Acronym / sponsor ID
- MIN-001-1203
- Conditions
- Glioma, Other Solid Tumours
- Keywords
- Glioma, Solid Tumours, LAM561
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 54 participants (actual)
- Lead sponsor
- Laminar PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Specialized Medical Services (SMS)-Oncology BV, Royal Marsden NHS Foundation Trust, Northern Institute for Cancer Research, Newcastle, Vall d'Hebron Institute of Oncology, Instituto Oncológico IMQ, Clínica IMQ Zorrotzaurre. Bilbao, Onkologikoa, San Sebastián.
- Study start
- May 2013
- Primary completion
- Sep 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 15 Feb 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 9
| Arm | Type | Receives |
|---|---|---|
| Dose Cohort 1 | Experimental | Drug: LAM561Intervention: 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 2 | Experimental | Drug: LAM561Intervention: LAM561. 500 mg twice daily |
| Dose Cohort 3 | Experimental | Drug: LAM561Intervention: LAM561. 1g twice daily |
| LAM561 Dose Cohort 4 | Experimental | Drug: LAM561Intervention: LAM561. 2g twice daily |
| LAM561 Dose Cohort 5 | Experimental | Drug: LAM561Intervention: LAM561. 4g twice daily |
| LAM561Dose Cohort 6 | Experimental | Drug: LAM561Intervention: LAM561. 4g three times daily |
| LAM561 Dose Cohort 7 | Experimental | Drug: LAM561Intervention: LAM561. 8g twice daily |
| LAM561 Dose Expansion cohort. Glioma | Experimental | Drug: LAM561Intervention: LAM561 at the MTD: 4g three times daily. Up to 10 patients with malignant glioma. |
| LAM561 Dose Expansion cohort. Non-glioma | Experimental | Drug: LAM561Intervention: LAM561 at the MTD: 4g three times daily. Up to 10 patients with other advanced solid tumours that are suitable for biopsy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Males or females providing written, informed consent
- Histologically- or cytologically-confirmed advanced solid malignancy that is refractory to standard-of-care treatment, or for which there is no standard therapy. If this is glioma:Grade III / Grade IV malignant glioma recurring or progressing after first or second line standard-of-care treatment and true progressive disease, confirmed according to the RANO criteria 4.
- Life-expectancy of at least 12 weeks
- Eastern cooperative oncology group (ECOG) performance status of 0-2
- Safety laboratory tests and ECGs within specified limits.
Key exclusion criteria (9 listed)
- Anti cancer therapy within 4 weeks (6 weeks for mitomycin and nitrosureas and 2 weeks for palliative radiotherapy)
- NCI Common terminology criteria for adverse events (CTCAE) >Grade 1 toxicities from prior chemotherapy or radiotherapy that could impact on safety outcome assessment
- Recent >Grade 1 intracranial or intratumoural haemorrhage either by CT or MRI scan. Patients with resolving haemorrhage changes, punctuate haemorrhage or haemosiderin may enter the study
- Significant or uncontrolled cardiovascular disease, unstable angina or myocardial infarction within the preceding 6 months
- Known impairment of GI function that could alter the absorption of study drug
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LAM561Patients will receive treatment cycles of 21 days, until any criterion for discontinuation (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion) is met. Patients are expected to receive between one and 6 cycles of treatment. The treatment period may be extended if clinical benefit is shown. In the event of significant GI toxicity, the treatment schedule may be modified from continuous dosing to an intermittent regime In the case of toxicity, the dose of LAM561 may be reduced or delayed by up to 14 days at the discretion of the Investigator. A maximum of two dose reductions will be permitted per patient. Treatment "holidays" of no more than 14 days are also permitted for reasons other than toxicity. Intra-patient dose escalation may be permitted in certain specific circumstances. | Drug | Dose Cohort 1, Dose Cohort 2, Dose Cohort 3, LAM561 Dose Cohort 4, LAM561 Dose Cohort 5, LAM561 Dose Cohort 7, LAM561 Dose Expansion cohort. Glioma, LAM561 Dose Expansion cohort. Non-glioma, LAM561Dose Cohort 6 |
Primary outcome
Number of patients with adverse events
Time frame: From 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
5 secondary outcomes
Concentration of LAM561 in blood measured by LC-MS/MS
Time frame: 21 days
Concentration of biomarkers in blood or tumour tissue
Time frame: First 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)
Concentration of micro RNA in blood
Time frame: First 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)
Radiological disease progression
Time frame: Every 6 weeks until any criterion for discontinuation is met (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion)
Clinical disease progression
Time frame: until any criterion for discontinuation is met (clinical or radiological progression of disease, clinically unacceptable toxicity, or another "general" discontinuation criterion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2023.
- Derived from this study: PubMed 37488446Lopez J, Lai-Kwon J, Molife R, Welsh L, Tunariu N, Roda D, Fernandez-Garcia P, Llado V, McNicholl AG, Rossello CA, Taylor RJ, Azaro A, Rodon J, Sludden J, Veal GJ, Plummer R, Urruticoechea A, Lahuerta A, Mujika K, Escriba PV. A Phase 1/2A trial of idroxioleic acid: first-in-class sphingolipid regulator and glioma cell autophagy inducer with antitumor activity in refractory glioma. Br J Cancer. 2023 Sep;129(5):811-818. doi: 10.1038/s41416-023-02356-1. Epub 2023 Jul 24.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Professor Johann de Bono, MB ChB FRCP MSc PhD study chair
The Institute of Cancer Research, 15 Cotswold Road, Belmont, Sutton, Surrey, United Kingdom SM2 5NG
Prof. Ruth Plummer, BMBCh, MRCP, Cert Me principal investigator
Northern Institute for Cancer Research, Newcastle
Dr Jordi Rodon principal investigator
Vall d'Hebron Institute of Oncology
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing LAM561 in people with Glioma and Other Solid Tumours, run by Laminar Pharmaceuticals at 5 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with adverse events (time frame: From the first dose of study drug until 30 days after the last dose of study drug). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01792310, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Feb 2023.