Proof-of-Concept Study of ACT001 in Adult Patients With Recurrent Glioblastoma Harbouring STAT3-High Signature

NCT06894225 | Suspended Phase 2

• 1 other identifier: 2024-3595
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Recurrent Glioblastoma

Outcome Measures

Primary Outcomes (1)

• 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.

  1.5 years

Secondary Outcomes (1)

• Progression-free survival (PFS) & Overall survival (OS)

  1.5 years

Other Outcomes (3)

• Toxicity evaluation

  1.5 years

• Drug levels

  1.5 years

• Treatment response and resistance

  1.5 years

Study Arms (1)

ACT001

EXPERIMENTAL

Drug: ACT001

Interventions

ACT001 DRUG

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

ACT001

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically confirmed diagnosis of GBM according to 2021 WHO classification
• Availability of tumor tissue representative of GBM from definitive surgery or biopsy and tested to harbour STAT3-High Signature
• Previous treatment with at least radiation therapy
• Documented recurrence of malignant glioma by diagnostic biopsy, resection or MRI performed within 21 days of study enrolment per RANO criteria.
• There is no limit on number of previous recurrences or lines of treatment
• At least 12 weeks after the end of prior radiation therapy is required unless there is either: i) histopathologic confirmation of recurrent tumor, or ii) n new enhancement on MRI outside of the radiation treatment field
• An interval of at least 4 weeks after the last administration of any investigational agent or any other treatment prior to first dose of STAT3 inhibitor
• Age 21 years or older on the day of signing informed consent
• Karnofsky performance status (KPS) of 70 or higher
• Patient has adequate bone marrow, renal, and hepatic function ≤ 21 days prior to study enrolment (Step 2) as follows:
• Absolute neutrophil count (ANC) ≥1,500/mm3
• Platelets ≥ 100,000/mm3
• Hemoglobin (Hgb) ≥ 9.0 g/dL (Note: The use of transfusion or other intervention to achieve Hgb ≥ 9.0 g/dL is acceptable.)
• Renal function: calculated creatinine clearance ≥ 30 mL/min by the Cockcroft-Gault formula
• Hepatic function: Total bilirubin, Aspartate Aminotransferase (AST), and Alanine Aminotransferase (ALT) ≤ 1.5 times upper limit of normal (ULN). Patients with Gilbert's syndrome documented in medical history may be enrolled if bilirubin is \< 3 times ULN.

You may not qualify if:

• Presence of extracranial metastatic or leptomeningeal disease
• Previous or current treatment with a JAK or STAT3 inhibitor
• Previous or current treatment with bevacizumab/VEGF inhibitor
• Patient is a lactating or pregnant female.
• Symptomatic intra-tumoural haemorrhage
• Severe, active co-morbidity, defined as follows:
• Patients with clinically defined Acquired Immune-Deficiency Syndrome (AIDS)-defining illness.
• Active connective tissue disorders, such as lupus or scleroderma, that in the opinion of the Investigator may put the patient at high risk of toxicity
• Any other major medical illnesses or psychiatric impairments that in the Investigator's opinion will prevent administration or completion of protocol therapy

Sponsors & Collaborators

• National Neuroscience Institute: lead

Study Sites (1)

National Neuroscience Institute

Singapore, Singapore

Location

MeSH Terms

Conditions

Glioblastoma

Interventions

ACT001

Condition Hierarchy (Ancestors)

Astrocytoma; Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 27, 2024
• First Posted: March 25, 2025
• Study Start: March 22, 2025
• Primary Completion: May 1, 2026
• Study Completion: May 1, 2026
• Last Updated: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

SG (1)