Recruiting

Phase 1

Phase 2

LMP744Novel Indenoisoquinolone CMYC/TOPOISOMERASE 1 Inhibitor (LMP744) in Recurrent Glioblastoma

Sponsor:

National Institute of Neurological Disorders and Stroke (NINDS)

Code:

NCT07416188

Conditions

Recurrent Glioblastoma

Glioblastoma IDH (Isocitrate Dehydrogenase) Wildtype

Relapsed Cancer

Recurrent Tumor

Glioblastoma Multiforme

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

LMP744

Conventional Surgery or Biopsy

Study Details

Brief 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.

Conditions

Recurrent Glioblastoma

Glioblastoma IDH (Isocitrate Dehydrogenase) Wildtype

Relapsed Cancer

Recurrent Tumor

Glioblastoma Multiforme

Study ID

NCT07416188

Start date

Jul 15, 2026

Status verified date

Oct 14, 2026

Completion date

Dec 31, 2032

Anticipated

Primary completion date

Dec 31, 2030

Anticipated

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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 surgical intervention for brain biopsy and/or resection.

Initial diagnostic biopsy under 03-N-0164 will be performed to confirm recurrent disease and obtain pre-treatment tissue.
The surgical intervention may include stereotactic/needle biopsy, open biopsy, and/or resection, as determined by neurosurgical assessment. Only participants for whom gross total resection of tumor is not expected to be achievable 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 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

Study Design

Enrollment

40 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Arm A

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.

Interventions

LMP744

Indenoisoquinolone C-MYC/TOPOISOMERASE 1 Inhibitor

Conventional Surgery or Biopsy

Primary outcome measure

Partial response (>=50% disease reduction) or complete response (100% disease reduction) based on RANO 2.0 criteria [ Time Frame: 5 years ]

Central Contacts and Locations

Central contacts

Sadhana Jackson, M.D.

(301) 594-7037 sadhana.jackson@nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

NIH Clinical Center Office of Patient Recruitment (OPR)

800-411-1222 ccopr@nih.gov

Surgical Neurology Branch

(301) 496-2921 snbrecruiting@nih.gov

More Information

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Last update posted

Oct 16, 2026

Last verified

Oct 14, 2026

Keywords

Recurrent Glioblastoma
Glioma
Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histological Type
Glioblastoma Multiforme (GBM)
Phase I
Phase II
Brain Cancer

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-25. This information was provided to ClinicalTrials.gov by National Institute of Neurological Disorders and Stroke (NINDS) on 2026-10-16. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.