Testing ATX-559, a new oral drug, in patients with advanced solid tumors
A Phase 1/2, Open-Label, Dose-Escalation and Expansion First-In-Human Study of ATX-559, an Oral Inhibitor of the Helicase DHX9, in Patients With Locally Advanced or Metastatic Solid Tumors and Molecularly Defined Cancers
This study is testing a new oral drug called ATX-559 to see if it is safe and helps people with advanced solid tumors, especially those with certain genetic markers, who have run out of other treatment options.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Accent Therapeutics Industry-sponsored |
| Locations | 4 sites (Oklahoma City, Oklahoma and 3 other locations) |
| Trial ID | NCT06625515 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and tolerability of ATX-559, an oral inhibitor of the DHX9 protein, in patients with advanced solid tumors and molecularly defined cancers. The study will assess the pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of ATX-559 through a first-in-human, Phase 1, open-label, single-arm, dose-escalation design. Patients with specific tumor types, such as BRCA-deficient breast cancer and those with microsatellite instability-high (MSI-H) or deficient mismatch repair (dMMR), will be enrolled to determine the recommended phase 2 dose and explore biomarker responses. The trial will also include patients who have exhausted standard treatment options.
Who should consider this trial
Good fit: Ideal candidates include patients with advanced solid tumors, particularly those with BRCA1 or BRCA2 mutations or MSI-H/dMMR tumors, who have not responded to standard therapies.
Not a fit: Patients with clinically unstable central nervous system tumors or brain metastasis may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for patients with advanced solid tumors that are currently difficult to treat.
How similar studies have performed: Other studies targeting similar molecular pathways have shown promise, suggesting potential for success with this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: * Patients with histologically confirmed solid tumors who have locally recurrent or metastatic disease * Refractory to or relapsed after all standard therapies with proven clinical benefit, unless as deemed by the Investigator, the subject is not a candidate for standard treatment, there is no standard treatment, or the subject refuses standard treatment after expressing an understanding of all available therapies with proven clinical benefit * For the expansion cohorts, participants must have histological confirmation of the specified tumor types: * BRCA1 or BRCA2 deficient, HER2 negative metastatic breast cancer * dMMR or MSI-H with unresectable or metastatic solid tumors * There is no limit to the number of prior treatment regimens * Have measurable or evaluable disease * Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1 Key Exclusion Criteria: * Clinically unstable central nervous system (CNS) tumors or brain metastasis * Any other concurrent anti-cancer treatment * Has undergone a major surgery within 3 weeks of starting study treatment * Medical issue that limits oral ingestion or impairment of gastrointestinal function that is expected to significantly reduce the absorption of ATX-559 * Clinically significant (ie, active) or uncontrolled cardiovascular disease * Unable to transition off strong or moderate CYP2C8 inhibitors or inducers * Pregnancy or intent to breastfeed or conceive a child within the projected duration of treatment Other inclusion and exclusion criteria as defined in the study protocol
Where this trial is running
Oklahoma City, Oklahoma and 3 other locations
- Stephenson Cancer Center at OU Medicine — Oklahoma City, Oklahoma, United States (Recruiting)
- SCRI Oncology Partners — Nashville, Tennessee, United States (Recruiting)
- The University of Texas MD Anderson Cancer Center — Houston, Texas, United States (Recruiting)
- NEXT Oncology — San Antonio, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Jason Sager, MD
- Email: clinicaltrials@accenttx.com
- Phone: (339) 970-7400
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.