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

NCT06414434 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 24-156
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 2

Brief Summary

This study is testing two different doses of BTX-A51 to determine if it is safe and tolerable in participants with liposarcoma with MDM2 amplification, myxoid liposarcoma, and CIC-rearranged sarcoma. The name of the study drug used in this research study is:

• BTX-A51 (a type of kinase inhibitor)

Conditions

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

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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

  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.

  All AEs will be recorded from the time the subject signs informed consent until 30 days after the last dose of study BTX-A51.

Secondary Outcomes (3)

• Overall Response Rate (ORR)

  ORR expected to be observed up to 3 years

• 1-year Progression-Free Survival (PFS) Rate

  1 year

• 1-year Overall Survival (OS)

  1 year

Study Arms (2)

BTX-A51 21mg

EXPERIMENTAL

Participants will be enrolled and will complete study procedures as follows: * Baseline visit with tumor biopsy. * Tumor biopsy at the end of Cycle 1. * Radiologic imaging every 2 cycles. * Cycle 1 through End of Treatment: --Day 1 of 28 day cycle: Predetermined dose of BTX-A51 3x weekly. * End of Treatment visit with radiologic imaging. * Follow-up: every 3 months for 1 year.

Drug: BTX-A51

BTX-A51 30mg

EXPERIMENTAL

Participants will be enrolled and will complete study procedures as follows: * Baseline visit with tumor biopsy. * Tumor biopsy at the end of Cycle 1. * Radiologic imaging every 2 cycles. * Cycle 1 through End of Treatment: --Day 1 of 28 day cycle: Predetermined dose of BTX-A51 3x weekly. * End of Treatment visit with radiologic imaging. * Follow-up: every 3 months for 1 year.

Drug: BTX-A51

Interventions

BTX-A51 DRUG

Multi-kinase inhibitor, 1.0 mg, 2.0 mg, and 7.0 mg immediate-release capsules, taken orally per protocol.

Also known as: C32H41ClN6O6S2, (1r,4r)-N 1-(5-chloro-4-(5-(cyclopropylmethyl)-1-methyl-l H pyrazol-3-yl)pyrimidin-2-yl)cyclohexane-l ,4-diamine bis(4-methylbenzenesulfonate),

BTX-A51 21mg; BTX-A51 30mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Michael Wagner, MD: lead
• Edgewood Oncology Inc.: collaborator

Study Sites (2)

Brigham and Women&#39;s Hospital

Boston, Massachusetts, 02215, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

MeSH Terms

Conditions

Liposarcoma; Liposarcoma, Myxoid

Condition Hierarchy (Ancestors)

Neoplasms, Adipose Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma

Study Officials

• Michael Wagner, MD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor Investigator

Study Record Dates

• First Submitted: May 7, 2024
• First Posted: May 16, 2024
• Study Start: September 30, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2027
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data can be shared no earlier than 1 year following the date of publication
• Access Criteria: Contact the Belfer Office for Dana-Farber Innovations (BODFI) at innovation@dfci.harvard.edu

Locations

US (2)