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NCT07416188From ClinicalTrials.govRecruiting

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

  • Recurrent Glioblastoma
  • Glioblastoma IDH (Isocitrate Dehydrogenase) Wildtype
  • Relapsed Cancer
  • Recurrent Tumor
  • Glioblastoma Multiforme
  • Recurring Glioblastoma
  • Brain and Central Nervous System Tumors
  • Glioma

At a glance

Phase
Phase 1
Study type
Interventional
Sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Enrolment target
40
Started
15 July 2026
Main results due
31 December 2030
Study sites
1
Registry updated
28 September 2026

Can you take part?

  • Ages 18 years to 99 years.
  • Open to any sex.
  • You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full eligibility criteria
* 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

What this study is about

In the sponsor’s own words, from the registry.

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 sites(1)

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, United States

    Recruiting

Showing 1 of 1 sites. 1 of the 1 site on this study is recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT07416188. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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Novel Indenoisoquinolone CMYC/TOPOISOMERASE 1 Inhibitor (LMP744) in Recurrent Glioblastoma | Trialion