NCT07416188 · National Institute of Neurological Disorders and Stroke (NINDS)
Novel Indenoisoquinolone CMYC/TOPOISOMERASE 1 Inhibitor (LMP744) in Recurrent Glioblastoma
What this study is about
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.
View original scientific description
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.
Interventions
DRUG
LMP744
Indenoisoquinolone C-MYC/TOPOISOMERASE 1 Inhibitor
PROCEDURE
Conventional Surgery or Biopsy
Conventional Surgery or Biopsy
Primary outcome measures
Partial response (>=50% disease reduction) or complete response (100% disease reduction) based on RANO 2.0 criteria
Time frame: 5 years
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
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
- Participant must meet all the following inclusion criteria to be deemed eligible for this study:
- Participants \>= 18 years of age
- Tissue-based diagnosis of recurrent glioblastoma, IDH-wildtype by a neuropathologist
- Karnofsky Performance Status (KPS) \>60
- Willing to use effective birth control method --The effects of LMP744 on developing human fetuses are unknown. Therefore, females of childbearing potential 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 craniotomy for brain biopsy and/or resection --Initial diagnostic biopsy under 03-N-0164 to confirm recurrent disease and obtain pre-treatment tissue. Only participants who were not expected to able to achieve a gross total resection of tumor will be included in the study.
- Willing and able to appoint a durable power of attorney
- Able to provide informed consent or have a legally authorized representative (LAR) to provide consent, if incapacitated.
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
- Significant medical co-morbidities that would compromise the participant s ability to tolerate LMP744 and which cannot reasonably be controlled (per the investigator s judgment, such as poorly controlled chronic kidney disease and/or poorly controlled congestive heart failure)
- Social situations that would limit compliance with study requirements, such as chronic homelessness
- Prior chemotherapy or biologic therapy completed within 4 weeks (6 weeks for nitrosoureas and mitomycin C) or a duration of 5 half-lives (whichever is shorter)
- Additional malignancy diagnosed or requiring active treatment within 1 year of screening
- Unable to undergo an MRI scan of the brain
- Active autoimmune disease that requires systemic treatment within 2 years of screening
- Cardiac disease
- \>=2 coronary revascularization procedures
- Cardiac Troponin T or I \>= 2x the institutional upper limit of normal at screening
- Ejection fraction \<45% on screening echocardiogram
- Chronic hypokalemia (K\<2.5 mmol/L)
- Human Immunodeficiency Virus (HIV)
- Known history of HIV
- Positive HIV 1/2 at screening.
- Active Hepatitis B or Hepatitis C infection at screening
- Active infection requiring systemic antibacterial, antiviral or antifungal therapy \<7 days prior to initiation of study drug
- Recipient of autologous or allogeneic T cells
- Solid organ or tissue transplant recipients
Where
- Bethesda, Maryland
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 Jul 17, 2026 · Source of record for eligibility and locations