Novel Indenoisoquinolone CMYC/TOPOISOMERASE 1 Inhibitor (LMP744) in Recurrent Glioblastoma
Phase 1/Phase 2 Open Label Trial of a Novel Indenoisoquinolone C-MYC/TOPOISOMERASE 1 Inhibitor (LMP744) in Recurrent Glioblastoma
National Institute of Neurological Disorders and Stroke (NINDS)
40 participants
May 17, 2026
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria9
- 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 Criteria22
- 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 MIs
- >=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
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Interventions
Indenoisoquinolone C-MYC/TOPOISOMERASE 1 Inhibitor
Conventional Surgery or Biopsy
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07416188