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)
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.
Description
Study Description: Patients will undergo pathological confirmation of recurrent glioblastoma via stereotactic or open biopsy (Visit 1, Day 1) to obtain baseline reference tissue. Following confirmation of recurrent glioblastoma by histopathological tissue analysis by a pathologist, study participants with confirmed histopathologically recurrent glioblastoma will be readmitted (Visit 2, Day 1). Patients will receive an initial cycle of 190 mg/m\^2/day LMP744 infused over 1 hour for 5 consecutive days (Visit 2, Days 2-6). Participants will then undergo biopsy or surgical resection as clinically…
Eligibility
- Age range
- 18–99 years
- Sex
- All
- Healthy volunteers
- No
* 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)…
Interventions
- DrugLMP744
Indenoisoquinolone C-MYC/TOPOISOMERASE 1 Inhibitor
- ProcedureConventional Surgery or Biopsy
Conventional Surgery or Biopsy
Location
- National Institutes of Health Clinical CenterBethesda, Maryland