BTX-A51 in Patients With Liposarcoma or CIC-rearranged Sarcoma

Launched by MICHAEL WAGNER, MD · May 9, 2024

Keywords

ClinConnect Summary

This clinical trial is investigating a new drug called BTX-A51 to see if it is safe and well-tolerated for patients with liposarcoma, a type of cancer that affects fatty tissues. The study is specifically looking at patients who have recurrent or metastatic liposarcoma, particularly those with certain genetic features. To participate, patients must be at least 18 years old and have a confirmed diagnosis of the disease, with measurable tumors. Additionally, they should have recovered from any previous cancer treatments and meet specific health criteria.

Participants in the trial will receive the study drug and will be monitored closely for any side effects. They will also be required to use contraception during the study due to potential risks to developing fetuses. The trial is currently open for enrollment, and those interested should discuss with their healthcare team to see if they meet the eligibility criteria. This study could provide valuable information about a new treatment option for liposarcoma patients.

Gender

ALL

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), or myxoid liposarcoma, or CIC-rearranged sarcoma.
• • 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.