LAM561 With RT and TMZ for Adults With Glioblastoma

NCT ID: NCT04250922

Last Updated: 2025-11-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

144 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-01

Study Completion Date

2026-11-30

Brief Summary

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The proposed Phase IIB/III randomized, double-blind, placebo-controlled trial in subjects with newly diagnosed primary glioblastoma multiforme (ndGBM) aims to compare the efficacy and safety of LAM561 versus placebo, given with standard of care (SoC) therapy of radiation therapy plus temozolomide (TMZ), followed by an adjuvant treatment of 6 month period of TMZ and then LAM561 or placebo in monotherapy.

Detailed Description

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This is a randomized, double-blind, placebo-controlled, 2 parallel arms (1:1 ratio), adjuvant trial to assess the efficacy of LAM561 versus placebo in patients with newly diagnosed, IDH1 wildtype, GBM. In all arms, patients will receive the SoC and will be randomized to receive either placebo or LAM561 dose.

The study is planned to enrol 140 patients. The primary endpoints of the study are PFS (for CMA) and OS (for FMA) as assessed after observing at least 66 PFS events and at least 90 OS events, respectively. It is expected that the analysis for PFS will be performed 1-2 years earlier than the analysis for OS.

An IDMC (Independent Data Monitoring Committee) meeting took place to assess the trial's futility and safety data from the start of the trial until September 2023. The IDMC had open access to the data without blinding; after analysis IDMC concluded that there were no concerns that would necessitate stopping the study for ethical reasons or safety issues that could adversely affect patients due to taking the medication, so the study is continuing as normal with no changes.

After 45 events for PFS are observed, a formal interim analysis will be performed and the data reviewed by an Independent Data Monitoring Committee (IDMC) or may be activated by the IDMC 12 months after the inclusion of the last patient if follow up is sufficient to identify an overall PFS or OS significant deviation from the literature. After reviewing the interim results, the iDMC will make recommendations regarding: the sample size and the continuation of the trial overall.

Further, the sample size and events will be re-estimated to ensure that the statistical power is maintained given the estimated treatment effect at interim analysis. The events/sample size increase will be based on the considerations of the success probability.

For that purpose, based on the conditional power, the interim results will be classified into the following zones: favourable, unfavourable or promising.

If the interim results fall in the promising zone, then it is planned to increase the total number of events both for PFS and OS by up to 50%, with up to 99 events for PFS and up to 135 events for OS. The total sample size will also be increased to up to 210 patients to ensure the desired number of events within a realistic time. If the interim results are favourable or unfavourable, the study size will remain as initially planned with 66 events for PFS and 90 for OS, collected from 140 patients.

The IDMC committee experts met in February 2024 after 45 PFS events occurred in the trial. Unblinded medical and clinical statistical data from 103 patients were evaluated to assess the efficacy - progression free survival (tumour growth or clinical deterioration) - of LAM561. The IDMC confirmed that, at the current level of evidence, after evaluating the Conditional Power of the unblinded results, no futility has been identified and recommends continuation of the study without modification.

In November 2024, After reaching 66 PFS events as stated by protocol, the independent data monitoring committee (IDMC) analyzed the data and recommended: (1) Continue the trial without modifications until 90 OS events (overall survival), when the final analysis will be carried out, estimated for Q4 2026; (2) that the trial should not be stopped for reasons of safety or futility and (3) opening the study, that is, removing the blind. From that moment, patients, doctors and Laminar as sponsor are able to know if any patient received placebo or LAM561.

Conditions

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Primary Glioblastoma Glioblastoma Multiforme

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a randomized, double-blind, placebo-controlled, 2 parallel arms (1:1), adjuvant trial to assess the efficacy of LAM561 vs placebo in patients with ndGBM. The 2 arms are: Arm A: SoC + placebo for LAM561; Arm B: SoC + 12g/day of LAM561

Treatment consists of 3 phases:

1. Chemoradiotherapy (6 to 7 weeks)

* RT in daily fractions of 2Gy, 5 days per week (60Gy)
* TMZ 75mg/m2 daily (maximum of 49 days)
* Placebo or LAM561 for 4 weeks (from week 3 to week 6, both inclusive) -Washout period (4 weeks)
2. Maintenance (4-week cycles, maximum of 6 cycles)

* Placebo/LAM561 daily in the first 3 weeks of each cycle, followed by 7 days washout
* TMZ 150-200mg/m2 daily in the first 5 days of each cycle
3. Monotherapy: 4-week cycles until disease progression, unacceptable toxicity or lack of clinical benefit Placebo/LAM561 daily in the first 3 weeks of each cycle followed by 7 days washout
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators
At the Screening Visit, the IRT will assign a unique number to the subject. The site will use the IRT to receive drug kit numbers and a unique randomization number.

Subjects will be randomized to Arm A or B in a 1:1 ratio. Drug kit numbers and treatment content will be assigned according to a list generated before the start of the study. At interim, the IRT vendor will transfer the list of attribution to the iSCs. After the final lock, the list of attribution will be transmitted to the company involved in analysis. The IRT vendor will be in charge of the stock management/logistics in each site and shipments. Upon receipt of study drug, the study site will acknowledge receipt in the IRT system.

The investigators, the study site personnel and subject will remain blinded to throughout the course of the study. The IRT will provide access to for a subject in case of medical emergency. If sponsor/clinical team should break the blind, the reason will be documented on the eCRF.

Study Groups

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Arm A: SoC + placebo for LAM561

Chemoradiation Phase: all subjects undergo focal RT, with a treatment given 5 days per week over \~6 weeks (no more than 7 weeks). TMZ will be administered at 75 mg/m2 orally, once daily, continuously throughout the RT for a maximum of 49 days. Subjects in Arm A will receive placebo every day from Day 1 of week 3 to the end of this Phase.

The start of the first cycle during the Maintenance (Adjuvant) Phase will be scheduled \~28 days (and never more than 42 days) after the last day of chemoradiation. During the Maintenance Phase, subjects will receive oral TMZ 150-200 mg/m2 once daily on Days 1-5 of each 28-day cycle for 6 cycles.

Subjects in Arm A will receive placebo every day during the first 3 weeks of each 28-day cycle and until progression. Patients will continue with Placebo after cycle 6 of the monotherapy phase until end of study. Adjuvant treatment will be discontinued upon determination of tumour progression, unacceptable toxicity or refusal to continue study treatment.

Group Type PLACEBO_COMPARATOR

TMZ

Intervention Type DRUG

TMZ will be administered at 75 mg/m2, orally, once daily, continuously from Day 1 of radiotherapy to the last day of radiation for a maximum of 49 days.

During the Maintenance (Adjuvant) Phase, all subjects will receive oral TMZ 150 - 200 mg/m2 once daily on Days 1 - 5 of each 28-day cycle for 6 cycles.

RT

Intervention Type RADIATION

During the Chemoradiation Phase, all subjects will undergo focal RT, with one treatment given daily 5 days per week over approximately 6 weeks (and no more than 7 weeks).

Arm B: SoC + 12 g/day of LAM561

Chemoradiation Phase: all subjects undergo focal RT, with a treatment given 5 days per week over \~6 weeks (no more than 7 weeks). TMZ will be administered at 75 mg/m2 orally, once daily, continuously throughout the RT for a maximum of 49 days. Subjects in Arm B will receive LAM561 every day from Day 1 of week 3 to the end of this Phase.

The start of the first cycle during the Maintenance Phase will be scheduled \~28 days (and never more than 42 days) after the last day of chemoradiation. During the Maintenance Phase, subjects will receive oral TMZ 150-200 mg/m2 once daily on Days 1-5 of each 28-day cycle for 6 cycles.

Subjects in Arm B will receive LAM561 during the Maintenance Phase. Patients will continue to be administered with LAM561 after cycle 6 of the monotherapy phase until end of study. Adjuvant treatment will be discontinued upon determination of tumour progression, unacceptable toxicity or refusal to continue study treatment.

Group Type EXPERIMENTAL

LAM561

Intervention Type DRUG

Subjects in Arm B will receive orally LAM561 during the Chemoradiation Phase.

Subjects in Arm B will receive LAM561 orally during the Maintenance (Adjuvant) Phase. Patients will continue to be administered with LAM561/Placebo after cycle 6 of the monotherapy phase until end of study. Adjuvant treatment will be discontinued upon determination of tumour progression as defined by RANO criteria, unacceptable toxicity, or refusal to continue study treatment.

TMZ

Intervention Type DRUG

TMZ will be administered at 75 mg/m2, orally, once daily, continuously from Day 1 of radiotherapy to the last day of radiation for a maximum of 49 days.

During the Maintenance (Adjuvant) Phase, all subjects will receive oral TMZ 150 - 200 mg/m2 once daily on Days 1 - 5 of each 28-day cycle for 6 cycles.

RT

Intervention Type RADIATION

During the Chemoradiation Phase, all subjects will undergo focal RT, with one treatment given daily 5 days per week over approximately 6 weeks (and no more than 7 weeks).

Interventions

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LAM561

Subjects in Arm B will receive orally LAM561 during the Chemoradiation Phase.

Subjects in Arm B will receive LAM561 orally during the Maintenance (Adjuvant) Phase. Patients will continue to be administered with LAM561/Placebo after cycle 6 of the monotherapy phase until end of study. Adjuvant treatment will be discontinued upon determination of tumour progression as defined by RANO criteria, unacceptable toxicity, or refusal to continue study treatment.

Intervention Type DRUG

TMZ

TMZ will be administered at 75 mg/m2, orally, once daily, continuously from Day 1 of radiotherapy to the last day of radiation for a maximum of 49 days.

During the Maintenance (Adjuvant) Phase, all subjects will receive oral TMZ 150 - 200 mg/m2 once daily on Days 1 - 5 of each 28-day cycle for 6 cycles.

Intervention Type DRUG

RT

During the Chemoradiation Phase, all subjects will undergo focal RT, with one treatment given daily 5 days per week over approximately 6 weeks (and no more than 7 weeks).

Intervention Type RADIATION

Eligibility Criteria

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Inclusion Criteria

1. Written informed consent, signed and dated
2. Subjects who are able to understand and follow instructions during the trial
3. Age ≥18 and ≤75
4. Subjects with newly histologically confirmed intracranial malignant glioma (glioblastoma WHO Grade IV) that is IDH1 wildtype (local assessment) and who are scheduled to receive chemo-radiotherapy with temozolomide
5. Ability to swallow and retain oral medication
6. Centrally obtained MGMT promoter methylation status
7. Subjects who underwent total or partial / incomplete resection and with the appropriate quantity of tumour tissue releasable for eligibility
8. Karnofsky Performance Score (KPS) \> 50 %
9. Female subjects with a childbearing potential must have a negative pregnancy test within one week before inclusion in the trial. Those female and male subjects admitted in the study must use a reliable method of contraception, for female subjects during the study period up until 32 days after last study treatment and for male subjects up until 92 days after last study administration.

Women must be:
* Either of NOT childbearing potential: postmenopausal (≥ 60 years of age, or \< 60 years of age and amenorrhoea for 12 months in the absence of chemotherapy, tamoxifen, toremifene, or ovarian suppression with follicle-stimulating hormone (FSH) above 40 U/L and oestradiol below 30 ng/L, or if taking tamoxifen or toremifene, and age \< 60 years, then FSH and oestradiol in the postmenopausal range), permanently sterilized (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy), or otherwise incapable of pregnancy
* Or of childbearing potential and practicing a highly effective method of birth control consistent with local regulations regarding the use of birth control methods for subjects participating in clinical studies: e.g., established use of oral, injected or implanted hormonal methods of contraception; placement of an intrauterine device or intrauterine system; male partner sterilization (the vasectomized partner should be the sole partner for that subject).
10. A man who is sexually active and has not had a vasectomy must agree to use a barrier method of birth control e.g., either condom or partner with occlusive cap (diaphragm or cervical/vault caps).
11. Adequate bone marrow function including: Absolute neutrophil count (ANC) ≥1,500/mm\^3 or ≥1.5 x 10\^9/L; Platelets ≥ 100,000/mm3 or ≥100 x10\^9/L; Haemoglobin ≥ 9 g/dL (may have been transfused).
12. Adequate hepatic function defined by a total bilirubin level ≤ 1.5 × the upper limit of normal range (ULN), an aspartate aminotransferase (AST), level ≤ 2.5 × ULN, and an alanine aminotransferase (ALT) level ≤ 2.5 × ULN or, for subjects with documented metastatic disease to the liver, AST and ALT levels ≤ 5 × ULN. Subjects with documented Gilbert disease are allowed if total bilirubin ≤ 3 x ULN
13. Adequate renal function defined by an estimated creatinine clearance ≥ 30 mL/min according to the Cockcroft-Gault formula

Exclusion Criteria

1. Known hypersensitivity to any component of the investigational product.
2. Any other investigational drug within the preceding 30 days. Prior, concomitant, or planned concomitant treatment with anti-neoplastic aim including (but not limited) to NovoTumor Treatment Fields (Novo TTF), bevacizumab, intratumoural or intracavitary anti-neoplastic therapy (e.g Gliadel wafers), or other experimental therapeutics intended to treat the tumour.
3. Subjects who underwent "only biopsy" resection
4. Anticancer therapy within 4 weeks of study entry (6 weeks for mitomycin and nitrosoureas)
5. Other major surgery within the preceding 30 days
6. Allergy, hypersensitivity or other intolerability to temozolomide and its excipients, patients with hypersensitivity to dacarbazine and patients with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption.
7. Unable to undergo MRI
8. Presenting with diffuse midline gliomas or multifocal GBM (distant from the flare or contralateral) or rapid progression between early post-surgery MRI and pre-radiotherapy MRI
9. Uncontrolled or significant cardiovascular disease
10. A history of uncontrolled hyperlipidaemia and/or the need for concurrent lipid lowering therapy
11. Need for warfarin, phenytoin or sulphonylureas (glibenclamide, glimepiride, glipizide, glyburide or nateglanide)
12. Past medical history of uncontrolled clinically significant active or chronic gastrointestinal disorders (for example, Crohn's disease, celiac disease, untreated stomach ulcers, etc) and gastro-inflammatory pathologies
13. Uncontrolled diabetes mellitus, with glycated haemoglobin (HbA1c) levels at the screening visit of ≥7.5%
14. Cardiac disease, defined specifically as either

1. Mean resting corrected QT interval (QTc) \> 470 msec (for women) and \> 450 ms (for men) obtained from 3 consecutive ECGs
2. Any clinically important abnormalities in rhythm, conduction or morphology of resting ECG (example, complete left bundle branch block, third degree heart block)
3. Any factors that increase the risk of QTc prolongation or risk of arrhythmic events such as heart failure, hypokalaemia, potential for Torsades de Pointes, congenital long QT syndrome, family history of long QT syndrome or unexplained sudden death under 40 years of age
15. Previous malignancies within the last three years other than ndGBM, except successfully treated squamous cell carcinoma of the skin, superficial bladder cancer, and in situ carcinoma of the cervix.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Laboratory Corporation of America

INDUSTRY

Sponsor Role collaborator

Northern Institute for Cancer Research, Newcastle

UNKNOWN

Sponsor Role collaborator

Theradis pharma

UNKNOWN

Sponsor Role collaborator

LIPODOM THERAPEUTICS

UNKNOWN

Sponsor Role collaborator

Laminar Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Institut Cancerologie de L'Ouest (ICO)

Angers, , France

Site Status

Centre Eugène Marquis (CEM)

Rennes, , France

Site Status

Gustave Roussy University Hospital

Rennes, , France

Site Status

Institut universitaire du cancer

Toulouse, , France

Site Status

Reaserch Fund of the Hadassah Medical Organization

Jerusalem, , Israel

Site Status

Istituto Oncologico Veneto IRCCS

Padua, Veneto, Italy

Site Status

Istituto Nazionale Neurologico Carlo Besta

Milan, , Italy

Site Status

Istituto Nazionale Tumori "Regina Elena"

Roma, , Italy

Site Status

University of Turin

Turin, , Italy

Site Status

Hospital Universitario Reina Sofía

Córdoba, Andalusia, Spain

Site Status

Hospital Universitario Virgen del Rocío

Seville, Andalusia, Spain

Site Status

Hospital Clinic

Barcelona, Barcelona, Spain

Site Status

Hospital Vall d'Hebron

Barcelona, Barcelona, Spain

Site Status

Hospital Clínico San Carlos

Madrid, Madrid, Spain

Site Status

Hospital Universitari i Politécnic La Fe.

Valencia, Valencia, Spain

Site Status

Hospital del Mar

Barcelona, , Spain

Site Status

Hospital Universitario 12 De Octubre

Madrid, , Spain

Site Status

Hospital Parc Tauli

Sabadell, , Spain

Site Status

Freeman Hospital's Northern Centre of Cancer Care

Newcastle, Newcastle Upon Tyne, United Kingdom

Site Status

University Hospitals Birmingham NHS Foundation Trust - New Queen Elizabeth Hospital

Birmingham, , United Kingdom

Site Status

Cambridge university hospital

Cambridge, , United Kingdom

Site Status

The Royal Marsden Hospital

London, , United Kingdom

Site Status

Countries

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France Israel Italy Spain United Kingdom

Other Identifiers

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MIN-003-1806

Identifier Type: -

Identifier Source: org_study_id

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