Testing BTX-A51 for patients with liposarcoma

A Pilot Study of BTX-A51 in Patients With Metastatic and/or Recurrent Liposarcomas Characterized by MDM2 Amplifications

This study is testing a new drug called BTX-A51 to see if it is safe and helps people with advanced liposarcoma that has certain genetic changes.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Dana-Farber Cancer Institute (other)
• Drugs / interventions: immunotherapy, radiation
• Locations: 2 sites (Boston, Massachusetts and 1 other locations)
• Trial ID: NCT06414434 on ClinicalTrials.gov

What this trial studies

This pilot study is evaluating the safety and tolerability of BTX-A51, a kinase inhibitor, in patients with metastatic or recurrent liposarcoma characterized by MDM2 gene amplifications. The study involves a single-arm approach where participants will receive the drug and be monitored for side effects and disease progression. Participants will undergo various imaging and blood tests to assess their eligibility and response to treatment. The study aims to enroll approximately 12 individuals over the course of the research.

Who should consider this trial

Good fit: Ideal candidates include individuals with histologically-confirmed metastatic or recurrent liposarcoma associated with MDM2 amplifications.

Not a fit: Patients with liposarcoma subtypes not associated with MDM2 amplifications or those with significant comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat liposarcoma.

How similar studies have performed: While this approach is novel, other studies targeting MDM2 amplifications in sarcomas have shown promise, suggesting potential for success.

Eligibility criteria

Inclusion Criteria:

* Study participants must have histologically-confirmed metastatic and/or recurrent liposarcoma (limited to the subtypes of well-differentiated and/or dedifferentiated liposarcoma, which are associated with MDM2 amplifications).
* ECOG performance status ≤2
* Adequate organ and marrow function as defined by the following metrics resulted within 7 days of study enrollment:

  * WBC \>3000/mm3
  * Platelets \>75,000μl
  * ANC \>1500μl
  * Hgb \>9g/dl
  * Creatinine \<1.5 x ULN or measured CrCl of \>60ml/m2/1.73 m2
  * Total bilirubin \<2 x ULN
  * AST/ALT \<3 x ULN
* Participants must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for non- nodal lesions and short axis for nodal lesions) as ≥20 mm (≥2 cm) by chest x-ray or as

  ≥10 mm (≥1 cm) with CT scan, MRI, or calipers by clinical exam. See Section 11 (Measurement of Effect) for the evaluation of measurable disease.
* Patients must have recovered from toxicity related to prior therapy to grade \<=1 (defined by CTCAE v5.0) (except alopecia and neuropathy, or immunotherapy related hypothyroidism)
* As the effect of this study drug on the developing human fetus is not known, women of child-bearing potential and men must agree to use at least 2 methods of contraception (abstinence; hormonal or barrier method of birth control) for the study and at least 2 months after completion.
* Female patient of childbearing potential has a negative serum pregnancy test within 7 days of study enrollment.
* Ability to understand and the willingness to sign a written informed consent document.
* Age ≥18 years
* Patients must have completed all prior anti-cancer treatment for liposarcoma, including radiation, ≥ 14 days prior to registration.

Exclusion Criteria:

* Patient with current evidence of active and uncontrolled infection, NYHA Class III-IV CHF, documented Child's class B-C cirrhosis, or uncontrolled medical disease which in the opinion of the investigator or the sponsor could compromise safety and/or assessment of efficacy.
* Active infection with hepatitis C virus (HCV) or hepatitis B virus (HBV); subjects who are positive for hepatitis B core antibody, hepatitis B surface antigen, or hepatitis C antibody must have a negative PCR result before enrollment; those who are PCR positive will be excluded.
* Major surgical procedure or open surgical biopsy within 28 days of first dose of study drug
* Active central nervous system (CNS) disease involvement, or prior history of NCI CTCAE Grade ≥3 drug-related CNS toxicity. Subject with known CNS metastases that are treated and stable (without evidence of CNS toxicity) and are not requiring systemic steroids are allowed to be enrolled.
* Patient has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial
* Myocardial infarction within 12 months of screening
* Use of any other concurrent investigational agents or anticancer agents, excluding hormonal therapy for breast or prostate cancer
* Pregnant women are excluded from this study because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with BTX- A51, breastfeeding should be discontinued if the mother is treated with BTX-A51.
* Inability to swallow pills or inadequate GI absorption in the opinion of the treating investigator.

Where this trial is running

Boston, Massachusetts and 1 other locations

• Brigham and Women's Hospital — Boston, Massachusetts, United States (RECRUITING)
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States (RECRUITING)

Study contacts

• Principal investigator: Michael Wagner, MD — Dana-Farber Cancer Institute
• Study coordinator: Michael Wagner, MD

Conditions: Liposarcoma, Recurrent Liposarcoma, Metastatic Liposarcoma, Unresectable Liposarcoma, MDM2 Gene Amplification