Actively Recruiting

Phase 1
Phase 2
Age: 18Years - 99Years
All Genders
ID07416188

Phase 1/Phase 2 Open Label Trial of a Novel Indenoisoquinolone C-MYC/TOPOISOMERASE 1 Inhibitor (LMP744) in Recurrent Glioblastoma

Led by National Institute of Neurological Disorders and Stroke (NINDS) · Updated on 2026-06-08

40

Participants Needed

1

Research Sites

104 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are studying the effects of a drug called LMP744 in adults with recurrent glioblastoma, a common and often fatal brain cancer that returns after initial treatments like surgery, radiation, and chemotherapy. This trial aims to see if LMP744 can kill glioblastoma tumor cells by comparing tissue samples taken before and after treatment. The study is an open-label trial involving surgery and drug treatment to explore the drug's impact on the tumor and patient quality of life. Participants will first undergo a biopsy to confirm recurrent glioblastoma and provide baseline tissue samples. They will receive LMP744 through a central line inserted into a vein in the chest for 5 days. Following this, a second surgery will remove as much tumor as possible for further tissue analysis. After recovery, participants will receive monthly 5-day cycles of LMP744 for up to 12 months, with ongoing monitoring including blood tests, heart function tests, and imaging scans. Throughout the trial, participants will stay in the clinic during initial treatments and surgeries, then recover at home with follow-up visits including telehealth every 3 months after stopping the drug. Researchers will assess tumor response using imaging criteria, overall survival, progression-free survival, molecular changes in tumor tissue, and quality of life surveys. Safety will be monitored by tracking adverse events during the study, which may last several years.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years - 99Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants 18 years of age or older
  • Tissue-based diagnosis of recurrent glioblastoma, IDH-wildtype confirmed by a neuropathologist
  • Karnofsky Performance Status (KPS) greater than 60
  • Willing to use an effective birth control method during the study and for 6 months after
  • Agreeable to undergo craniotomy for brain biopsy and/or tumor resection
  • Willing and able to appoint a durable power of attorney
  • Able to provide informed consent or have a legally authorized representative to consent if incapacitated
Not Eligible

You will not qualify if you...

  • Pregnant or nursing females
  • Significant medical conditions that prevent tolerating LMP744, such as uncontrolled kidney disease or heart failure
  • Social situations limiting study compliance, like chronic homelessness
  • Prior chemotherapy or biologic therapy within 4 weeks (6 weeks for certain drugs) before enrollment
  • Additional active malignancy requiring treatment within 1 year
  • Unable to have an MRI scan of the brain
  • Active autoimmune disease needing systemic treatment within 2 years
  • Serious cardiac disease including multiple heart attacks or reduced heart function
  • Chronic low potassium levels
  • Known HIV infection or positive HIV test at screening
  • Active Hepatitis B or C infection
  • Active infection requiring systemic antibacterial, antiviral, or antifungal treatment within 7 days before study drug
  • Prior receipt of autologous or allogeneic T cells
  • Solid organ or tissue transplant recipients

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person) for initial biopsy

Treatment

Duration - Initial 5 days of infusion plus 3-8 weeks recuperation, followed by up to 12 cycles of 5 days on treatment and 23 days off

Participants receive an initial 5-day cycle of LMP744 drug infusion followed by biopsy or surgical resection to collect tissue for molecular analysis, then after a recuperation period, resume LMP744 treatment in cycles.

1 admission visit plus 5 consecutive days of infusion, 1 surgical visit, then repeated treatment cycles with visits every 28 days up to 12 cycles

Follow-up

Duration - Up to 5 years

Participants are followed until death to monitor clinical outcomes, survival, and quality of life after completing treatment cycles.

Visits scheduled after each treatment cycle and at study completion for assessments

Trial Site Locations

Total: 1 location

1

National Institutes of Health Clinical Center

Bethesda, Maryland, United States, 20892

Actively Recruiting

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Research Team

S

Sadhana Jackson, M.D.

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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