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BTX-A51 for Liposarcoma or CIC-rearranged Sarcoma

This study is investigating BTX-A51, a drug that works by blocking certain proteins (CK1α and CDK9) to treat specific types of cancer. It's for people with metastatic (spread to other parts of the body) or recurrent (returned after treatment) liposarcoma (a type of cancer that begins in fat cells) with MDM2 gene amplification, myxoid liposarcoma, or CIC-rearranged sarcoma. The main goal is to see if BTX-A51 is safe and tolerable at two different doses. Researchers will monitor any side effects, lab changes, or reasons participants might need to stop or adjust their dose. About 24 people are expected to join this study. BTX-A51 is not yet approved by the FDA for these conditions.

Study design
This is a pilot study with two groups, aiming to enroll about 24 participants. It is testing two different doses of BTX-A51.
What's involved
You would undergo screening, CT, MRI, or PET scans, blood tests, and tumor biopsies. You would receive BTX-A51 as long as there are no serious side effects and your disease doesn't worsen.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 1 year after receiving their last dose of BTX-A51.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06414434

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

Active, Not Recruiting
PHASE1Ages 18+InterventionalTreatment
Michael Wagner, MD
~24 participants
Updated 2026-07-01 on ClinicalTrials.gov
What's tested:BTX-A51

At a glance

Recruiting sites
0 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with adverse events, with laboratory abnormalities, with dose modifications, delays, interruptions, or premature discontinuation of BTX-A51 due to an adverse event
Measured over All AEs will be recorded from the time the subject signs informed consent until 30 days after the last dose of study BTX-A51.

Conditions

Liposarcoma
Recurrent Liposarcoma
Metastatic Liposarcoma
Unresectable Liposarcoma
MDM2 Gene Amplification
Myxoid Liposarcoma
CIC-Rearranged Sarcoma

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CIC-Rearranged SarcomaLiposarcoma

Where it's being run

2 sites across 1 states
Massachusetts2

Study leadership

  • Michael Wagner, MD · PRINCIPAL_INVESTIGATOR · Dana-Farber Cancer Institute

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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
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

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.

What this trial measures

  • Number of participants with adverse events, with laboratory abnormalities, with dose modifications, delays, interruptions, or premature discontinuation of BTX-A51 due to an adverse eventAll AEs will be recorded from the time the subject signs informed consent until 30 days after the last dose of study BTX-A51.

    Safety and tolerability will be monitored through continuous reporting of treatment-emergent and treatment-related adverse events, laboratory abnormalities, and incidence of subjects experiencing dose modifications, delays, interruptions, or premature discontinuation of BTX-A51 due to an adverse event. Toxicities are to be assessed according to the CTCAEv5.