A Phase 1/2 interventional study of LMP744 and Conventional Surgery or Biopsy in Recurrent Glioblastoma, Glioblastoma IDH (Isocitrate Dehydrogenase) Wildtype and Relapsed Cancer, sponsored by National Institute of Neurological Disorders and Stroke (NINDS). Recruiting at 1 site in United States. Open to participants aged 18 Years to 99 Years. Per ClinicalTrials.gov, last updated 2026-10-16.
Sponsored by National Institute of Neurological Disorders and Stroke (NINDS) · Phase 1/2, Interventional, and Treatment
Background:
Glioblastoma is a common brain cancer in adults. Treatment includes surgery, radiation, and chemotherapy. But this cancer can return after treatment and is often fatal. Researchers want to know if a study drug (LMP744) can kill glioblastoma tumor cells.
Objective:
To test LMP744 in people with glioblastoma.
Eligibility:
People aged 18 years or older with glioblastoma that returned after treatment.
Design:
Participants will be screened. They will have a surgery to remove a small sample of tumor tissue (biopsy) from the brain. This will be done under protocol 03-N-0164. They will stay in the clinic for 1 night. They will also have imaging scans and tests of their heart function.
Participants will have a central line installed: A flexible tube will be inserted into a vein in the chest. It will be attached to a "port" under the skin. This port will be used to draw blood and give medicines without having to insert new needles into a vein.
LMP744 will be given through the central line for 5 days in a row. Participants will remain in the clinic for this time.
Participants will then have a second surgery to remove as much of their tumor as possible. They will remain in the clinic until they recover from the surgery. Then they will recover at home after surgery.
Participants will return to the clinic to receive the study drug for 5 days in a row through the central line, once a month for up to 12 months. Blood tests, heart function tests, and periodic imaging scans will be repeated during these visits.
Participants will continue to have telehealth visits every 3 months after they stop taking the drug.
Study Description:
Patients will undergo pathological confirmation of recurrent glioblastoma via stereotactic or open biopsy (Visit 1, Day 1) to obtain baseline reference tissue. Following confirmation of recurrent glioblastoma by histopathological tissue analysis by a pathologist, study participants with confirmed histopathologically recurrent glioblastoma will be readmitted (Visit 2, Day 1). Patients will receive an initial cycle of 190 mg/m\^2/day LMP744 infused over 1 hour for 5 consecutive days (Visit 2, Days 1-5). Participants will then undergo surgical intervention for brain biopsy and/or resection, as clinically appropriate based on neurosurgical assessment within 7 calendar days of the 5th dose of LMP744, but no earlier than 24 hours after the 5th dose of LMP744 (Visit 2, between Days 6 and 12 inclusive). Tissue, CSF, and plasma will be collected during the post-treatment surgical intervention for molecular analysis. Patients will then be discharged from the hospital. Following a 3-8-week period of recuperation, study participants will then resume LMP744 (5 days on; 23 days off; up to 12 cycles) and be followed until death.
A comparative pharmacodynamic analysis will be conducted on the pre- and post-treatment tissue to evaluate the biological response to LMP744 and correlate pharmacodynamic changes with clinical outcomes.
Objectives:
Primary Objective:
-To evaluate objective response rate (ORR) in patients with recurrent glioblastoma treated with up to 12 cycles of LMP744.
Secondary Objectives:
Exploratory Objective:
Safety Outcomes:
To descriptively monitor, document, and report adverse events (AEs) and serious adverse events (SAEs) in participants with recurrent glioblastoma treated with LMP744.
Endpoints:
Primary Endpoint:
-Stable disease or Partial response (>=50% disease reduction) or complete response (100% disease reduction) based on RANO 2.0 criteria
Secondary Endpoints:
PFS will be assessed based on the RANO 2.0 criteria for glioblastoma assessment as follows:
Imaging Criteria:
Clinical Features:
2,132 studies on the registry are indexed under Glioblastoma; 464 are open to participants now.
This study's planned enrollment of 40 is above the median of 36 across 1,753 interventional studies indexed under Glioblastoma.
Browse Glioblastoma studies →National Institute of Neurological Disorders and Stroke (NINDS) is the lead sponsor of 601 studies on the registry; 56 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 8 (44%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participant must meet all the following inclusion criteria to be deemed eligible for this study:
Willing to use effective method
--The effects of LMP744 on developing human fetuses are unknown. Therefore, females of and their male partners must be willing to use an effective method of contraception during the clinical study (hormonal, barrier, surgical, or abstinence) before study enrollment and for 6 months after the last dose of the study drug. If the female becomes pregnant or suspects she is pregnant while participating in this study, she must inform her treating physician immediately.
Agreeable to undergo surgical intervention for brain and/or resection.
EXCLUSION CRITERIA:
An individual who meets any of the following criteria will be excluded from participation in this study:
Pregnant and/or nursing females
--As LMP744 is a novel agent with the potential for teratogenic or abortifacient effects, pregnant and/or nursing females will be excluded from receiving drug
Cardiac disease
(HIV)
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All patients will receive LMP744 as the interventional drug along with surgery for a biopsy and a second surgery for either resection or an additional biopsy to compare the tissue pre vs. post-drug treatment.
Drug: LMP744 · Procedure: Conventional Surgery or Biopsy
Indenoisoquinolone C-MYC/TOPOISOMERASE 1 Inhibitor
Conventional Surgery or Biopsy
Partial response (>=50% disease reduction) or complete response (100% disease reduction) based on RANO 2.0 criteria
As ORR is associated with survival experience, this endpoint allows for objective evaluation of efficacy of LMP744 in this proof-of-concept phase 2 trial
Time frame: 5 years
Progression-Free Survival (PFS): Time from the start of treatment with LMP744 until disease progression based on RANO 2.0 criteria
PFS provides a measure of treatment efficacy in delaying tumor progression. While the study is not powered to demonstrate superiority of LMP744 to concurrent controls, the study will compare LMP744 to historical controls to generate information for planning of a future phase 3 trial.
Time frame: 5 years
Overall Survival (OS): Time from the start of treatment with LMP744 until death from any cause
OS is the gold standard for assessing the ultimate benefit of a therapy on survival. We will compare OS between LMP744 and historical controls to provide information for planning of a future phase 3 trial
Time frame: 5 years
Changes in transcriptomic and proteomic profiles before and after LMP744 treatment
Evaluating changes in these markers will provide insight into the pharmacodynamic effects of LMP744 at the molecular level. The selected markers are involved in DNA damage response, cell cycle regulation, and survival pathways, which are critical in understanding the tumor's response to the drug.
Time frame: 5 years
Change in self-reported quality of life (QOL) as measured by the SF-36 survey at baseline, after each treatment cycle, and at study completion.
The SF-36 is a validated tool for assessing the quality of life in cancer patients and captures the broad impact of treatment on physical, emotional, and social well-being. Tracking changes in QOL over time will provide insight into the patient-reported outcomes related to LMP744 treatment. This endpoint is essential for understanding the broader implications of treatment beyond clinical and molecular measures, giving a more comprehensive assessment of treatment benefit or burden.
Time frame: 5 years
Plan to share: Yes — We plan to share IPD as requested by publications and CRADA partner.
Supporting information: Study protocol, Clinical study report
Time frame: Study protocol will be shared as requested. CSR will be shared at the time the CSR is due.
Access criteria: De-identified IPD will be shared with the CRADA partner through the EDC.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: LMP-744
National Institute of Neurological Disorders and Stroke (NINDS) →