A Phase 2 interventional study of ACT001 in Recurrent Glioblastoma, sponsored by National Neuroscience Institute. Recruiting at 1 site in Singapore. Open to participants aged 21 Years and older. Per ClinicalTrials.gov, last updated 2026-09-04.
Sponsored by National Neuroscience Institute · Phase 2, Interventional, and Treatment
This trial will study the effectiveness of ACT001 in adult patients whose Glioblastoma have recurred with a STAT3-high signature after standard-of-care treatment with at least radiation therapy.
1,920 studies on the registry are indexed under Glioblastoma; 450 are open to participants now.
This study's planned enrollment of 12 is below the median of 36 across 1,618 interventional studies indexed under Glioblastoma.
Browse Glioblastoma studies →National Neuroscience Institute is the lead sponsor of 10 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
There is no limit on number of previous recurrences or lines of treatment
Patient has adequate bone marrow, renal, and hepatic function ≤ 21 days prior to study enrolment (Step 2) as follows:
Exclusion Criteria:
Severe, active co-morbidity, defined as follows:
Drug: ACT001
Patients recruited and have signed informed consent will be initiated on ACT001 400mg twice a day. Treatment course will repeat every 28 days in the absence of disease progression or unacceptable toxicity
Overall Response Rate
ORR as defined as radiographic complete response, or partial response, or stable disease. Patients with stable disease will be considered responders if disease is stable for 24 weeks or more. The regimen will be considered worthy of further study if responses as defined above are observed in at least 3 of the 12 patients.
Time frame: 1.5 years
Progression-free survival (PFS) & Overall survival (OS)
6 months progression-free survival (PFS), defined as proportion of patients who remained alive and progression-free at 6 months. PFS, which is defined as time from study enrolment to progression of disease per RANO criteria, or death, whichever occurs first. For patients who are not documented to have experienced a PFS event at the time of an analysis, PFS will be right-censored on the date of their last adequate assessment of disease. Overall survival (OS), which is defined as time from study enrolment to death from any cause. For patients who are not reported to have died at the time of an analysis, OS will be right-censored at the last date the patient is documented to be alive.
Time frame: 1.5 years
Toxicity evaluation
Study team will assess adverse events, laboratory data and vital signs throughout the study. Analyses of adverse events will include only "treatment-emergent" events, i.e., those that start or worsen on or after the day of the first dose of study drug. Adverse event severity and laboratory evaluation changes will be assessed by utilizing National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0. Adverse events will be summarized by preferred terms within a System and Organ Class according to the most current Medical Dictionary for Regulatory Activities (MedDRA) dictionary. Changes from baseline will be analyzed for each scheduled post-baseline visit and for the final visit for blood chemistry and hematology parameters, as well as urinalysis and vital sign parameters. Shifts in laboratory values from baseline NCI CTCAE grades to maximum and final post-baseline grades will be assessed.
Time frame: 1.5 years
Drug levels
Drug levels in cerebrospinal fluid and tissue sample when available in patients who are agreeable to proceed with Omaya shunt insertion, lumbar puncture or undergoes further surgical resection following treatment with ACT001.
Time frame: 1.5 years
Treatment response and resistance
Tissue and blood exosome biomarkers that are predictive of treatment response and treatment resistance.
Time frame: 1.5 years
Plan to share: No
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