NCT06414434

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

A Pilot Study of BTX-A51 in Patients With Metastatic and/or Recurrent Liposarcomas Characterized by MDM2 Amplifications, Myxoid Liposarcoma, and CIC-Rearranged Sarcoma

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Michael Wagner, MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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)

Detailed description

This is a two-cohort, pilot study assessing the safety and preliminary exploration of BTX-A51 at two dose levels in participants with metastatic and/or recurrent liposarcomas characterized by Murine Double Minute Clone 2 (MDM2) amplifications (i.e. de-differentiated and /or well-differentiated liposarcomas), myxoid liposarcoma, and CIC-rearranged sarcomas. BTX-A51 works in a different way from currently approved therapies used to treat liposarcoma by blocking proteins called CK1α and CDK9. The U.S. Food and Drug Administration (FDA) has not approved BTX-A51 as a treatment for liposarcoma characterized by MDM2 amplifications, myxoid liposarcoma, or CIC-rearranged sarcomas. The research study procedures include screening for eligibility, Computerized Tomography (CT) scans, Magnetic Resonance Imaging (MRI) scans, or Positron Emission Tomography (PET) scans, blood tests, and tumor biopsies. Participants will receive study treatment for as long as there are no serious side effects, and disease does not get worse. Participants will be followed for 1 year after the last dose of BTX-A51. It is expected that about 24 people will take part in this research study. Edgewood Oncology is supporting this research study by providing the study drug.

Conditions

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

Interventions

Type	Name	Description
DRUG	BTX-A51	Multi-kinase inhibitor, 1.0 mg, 2.0 mg, and 7.0 mg immediate-release capsules, taken orally per protocol.

Timeline

• Start date: 2024-09-30
• Primary completion: 2026-06-01
• Completion: 2027-06-01
• First posted: 2024-05-16
• Last updated: 2025-10-31

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study