Testing AST-001 in patients with advanced solid tumors

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Efficacy and Correlation With AKR1C3 Enzyme Expression of AST-001 in Subjects With Advanced Solid Tumors

This study is testing a new treatment called AST-001 to see if it is safe and effective for people with advanced solid tumors, like pancreatic cancer.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 180 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Ascentawits Pharmaceuticals, Ltd (industry)
• Drugs / interventions: chemotherapy, radiation
• Locations: 1 site (Changchun, Jinlin)
• Trial ID: NCT06245330 on ClinicalTrials.gov

What this trial studies

This is a first-in-human, open-label Phase I/II study designed to evaluate the safety and tolerability of AST-001, a new treatment for patients with advanced solid tumors, including pancreatic cancer. The study will assess the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D), as well as the pharmacokinetics (PK) and preliminary efficacy of AST-001 administered as a single agent. Participants will be closely monitored for any adverse effects and treatment responses throughout the trial.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18-70 with metastatic or unresectable solid malignancies that have not responded to standard therapies.

Not a fit: Patients with a history of another primary malignancy within the last two years or those who have undergone major surgery may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced solid tumors who have limited or no effective standard treatments available.

How similar studies have performed: While this approach is novel in the context of AST-001, similar studies evaluating new treatments for advanced solid tumors have shown promise in the past.

Eligibility criteria

* phase I: dose escalation phase

Inclusion Criteria:

1. Patient has ability to understand the risks of the study and is willing to comply with the protocol and has signed a written informed consent.
2. Aged 18-70 years (inclusive), males and females.
3. Histologically or cytologically confirmed solid malignancy that is metastatic or unresectable and for which standard curative do not exist or are no longer effective.
4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
5. Expected life expectancy ≥ 12 weeks
6. Recovered from toxicities of prior therapy to Grade 0 or 1
7. An adequate renal, liver and bone marrow function.

Exclusion Criteria:

1. History of another primary malignancy within 2 years prior to Day 1, except for adequately treated basaloma, in situ cancer, or other cancers whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the current study.
2. Major surgery, other than diagnostic surgery, within 4 weeks prior to Day 1.
3. Treatment with radiation therapy, chemotherapy, biotherapy, targeted therapies or hormones within 4 weeks prior to Day 1.
4. Receiving investigational therapy within 4 weeks prior to Day 1.
5. Concomitant use of repaglinide, medium/strong CYP2C8/CYP2B6/ CYP2C9 inhibitors/inducers.
6. Pleural effusion or ascites which need to be drained every other week or more frequently.
7. HBV infection and HBV-DNA ≥ 2,000 IU/mL
8. Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic therapy.
9. History of human immunodeficiency virus (HIV) infection or syphilis infection.
10. History of cardiac disease fits any of the following conditions：

    1. NYHA III or IV CHF；
    2. QTcF : male \> 450ms，female \> 470ms；
    3. Myocardial infarction, bypass surgery, stent surgery within 6 months prior to Day 1；
    4. Other cardiac disease that the investigator judged unsuitable for inclusion.
11. Females who are pregnant or breast-feeding
12. Concomitant disease or condition that could interfere with the conduct of the study, or that would, in the opinion of the investigator, pose an unacceptable risk to the subject in this study
13. Previously allergic to ethanol, polyoxyethylene (35) castor oil, N, N-dimethylacetamide.
14. Unwillingness or inability to comply with the study protocol for any reason

    * phase II: pancreatic cancer

Inclusion Criteria:

1. Patient has ability to understand the risks of the study and is willing to comply with the protocol and has signed a written informed consent.
2. Aged 18-70 years (inclusive), males and females.
3. Histologically or cytologically confirmed pancreatic cancer that is unresectable or cannot be controlled by local treatment and for which standard curative do not exist or are no longer effective.
4. At least one measurable lesion that meets RECIST 1.1 criteria.
5. Can provide pathological wax blocks or sections (including archived pathological wax blocks or sections) for AKR1C3 expression analysis and be confirmed that AKR1C3 expression is strongly positive.
6. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
7. Expected life expectancy ≥ 12 weeks
8. Recovered from toxicities of prior therapy to Grade 0 or 1
9. An adequate renal, liver and bone marrow function.

Exclusion Criteria:

1. History of another primary malignancy within 2 years prior to Day 1, except for adequately treated basaloma, in situ cancer, or other cancers whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the current study.
2. Major surgery, other than diagnostic surgery, within 4 weeks prior to Day 1.
3. Treatment with radiation therapy, chemotherapy, biotherapy, targeted therapies or hormones within 4 weeks prior to Day 1.
4. Receiving investigational therapy within 4 weeks prior to Day 1.
5. Concomitant use of repaglinide, medium/strong CYP2C8/CYP2B6/ CYP2C9 inhibitors/inducers.
6. Pleural effusion or ascites which need to be drained every other week or more frequently.
7. HBV infection and HBV-DNA ≥ 2,000 IU/mL
8. Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic therapy.
9. History of human immunodeficiency virus (HIV) infection or syphilis infection.
10. History of cardiac disease fits any of the following conditions：

    1. NYHA III or IV CHF；
    2. QTcF : male \> 450ms，female \> 470ms；
    3. Myocardial infarction, bypass surgery, stent surgery within 6 months prior to Day 1；
    4. Other cardiac disease that the investigator judged unsuitable for inclusion.
11. Females who are pregnant or breast-feeding
12. Concomitant disease or condition that could interfere with the conduct of the study, or that would, in the opinion of the investigator, pose an unacceptable risk to the subject in this study
13. Previously allergic to ethanol, polyoxyethylene (35) castor oil, N, N-dimethylacetamide.
14. Unwillingness or inability to comply with the study protocol for any reason

Where this trial is running

Changchun, Jinlin

• Jinlin Cancer Hospital — Changchun, Jinlin, China (RECRUITING)

Study contacts

• Principal investigator: Ying Cheng — Study Principal Investigator
• Study coordinator: Claire Hao

Conditions: Solid Tumor, Pancreatic Cancer