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
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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)

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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.

Locations
United States
Massachusetts
Brigham and Women&#39;s Hospital
RECRUITING
Boston
Dana-Farber Cancer Institute
RECRUITING
Boston
Contact Information
Primary
Michael Wagner, MD
MICHAEL_WAGNER@DFCI.HARVARD.EDU
617-632-5204
Backup
Michael Wagner, MD
MICHAEL_WAGNER@DFCI.HARVARD.EDU
617-632-3352
Time Frame
Start Date: 2024-09-30
Estimated Completion Date: 2027-06-01
Participants
Target number of participants: 24
Treatments
Experimental: BTX-A51 21mg
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.
Experimental: BTX-A51 30mg
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.
Sponsors
Leads: Michael Wagner, MD
Collaborators: Edgewood Oncology Inc.

This content was sourced from clinicaltrials.gov