RECRUITING

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

Conditions

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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)

Official Title

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

Quick Facts

Study Start:2024-09-30

Study Completion:2027-06-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06414434

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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 * 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.	* 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.

Inclusion Criteria

Exclusion 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
* 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.

* 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.

Contacts and Locations

Study Contact

Michael Wagner, MD

CONTACT

617-632-5204

MICHAEL_WAGNER@DFCI.HARVARD.EDU

Michael Wagner, MD

CONTACT

617-632-3352

MICHAEL_WAGNER@DFCI.HARVARD.EDU

Principal Investigator

Michael Wagner, MD

PRINCIPAL_INVESTIGATOR

Dana-Farber Cancer Institute

Study Locations (Sites)

Brigham and Women's Hospital

Boston, Massachusetts, 02215

United States

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215

United States

Collaborators and Investigators

Sponsor: Michael Wagner, MD

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-09-30

Study Completion Date2027-06-01

Study Record Updates

Study Start Date2024-09-30

Study Completion Date2027-06-01

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

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