Symptoms and General Pathology

All Conditions

Neoplasms

Rare Diseases

Recurrence

Liposarcoma

Disease Attributes

Neoplasms, Adipose Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Sarcoma

Soft Tissue Sarcoma

All Drugs and Chemicals

Vasoconstrictor Agents

Respiratory System Agents

Botulinum Toxins, Type A

abobotulinumtoxinA

Epinephrine

Pyrazole

Racepinephrine

Epinephryl borate

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.

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

BTX-A51

Michael Wagner, MD

This study is testing if the recommended dose of BTX-A51 is safe and tolerable in participants with liposarcoma.

The name of the study drug used in this research study is:

-BTX-A51 (a type of kinase inhibitor)

This is a single arm, pilot study assessing the safety and preliminary exploration of BTX-A51 in participants with metastatic and/or recurrent liposarcomas characterized by Murine Double Minute Clone 2 (MDM2) amplifications. 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.

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 12 people will take part in this research study.

Edgewood Oncology is supporting this research study by providing the study drug.

BTX-A51 in Patients With Liposarcoma

A Pilot Study of BTX-A51 in Patients With Metastatic and/or Recurrent Liposarcomas Characterized by MDM2 Amplifications

The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

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.

The overall response rate (ORR) was defined as the proportion of participants achieving complete response (CR) or partial response (PR) based on RECISTv1.1 criteria.

1-year PFS is a probability estimated using progression-free survival based on the Kaplan-Meier method is defined as the duration between randomization and documented disease progression (PD) or death, or is censored at time of last disease assessment.

1-year OS is a probability estimated using the Kaplan-Meier method; OS is defined as the time from study entry to death, or censored at date last known alive.

Edgewood Oncology Inc.

Michael Wagner

Sponsor Investigator

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Skin and Connective Tissue Diseases

Digestive System Diseases

Gland and Hormone Related Diseases

Respiratory Tract (Lung and Bronchial) Diseases

Uterine Cervical Neoplasms

Carcinoma

Cholangiocarcinoma

Breast Neoplasms

Adenocarcinoma

Pancreatic Neoplasms

Melanoma

Carcinoma, Non-Small-Cell Lung

Carcinoma, Transitional Cell

Stomach Neoplasms

Lung Neoplasms

Ovarian Neoplasms

Carcinoma, Ovarian Epithelial

Adenocarcinoma of Lung

Adrenocortical Carcinoma

Biliary Tract Neoplasms

Neoplasms, Glandular and Epithelial

Respiratory Tract Neoplasms

Thoracic Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Biliary Tract Diseases

Endocrine Gland Neoplasms

Endocrine System Diseases

Pancreatic Diseases

Adrenal Cortex Neoplasms

Adrenal Gland Neoplasms

Adrenal Cortex Diseases

Adrenal Gland Diseases

Pancreatic Cancer

Ovarian Cancer

Transitional Cell Carcinoma

Bile Duct Cancer

Stomach Cancer

Ovarian Epithelial Cancer

Biliary Tract Cancer

Germ Cells Tumors

Lung Adenocarcinoma

Testicular Germ Cell Tumor

260 mg once dailly orally on Days 1 to 3 and Days 15 to 17 of each 28-day cycle

260 mg once daily orally on Days 1 to 3 and Days 15 to 17 of each 28-day cycle

RAIN-32

Phase 2, multicenter, single-arm, open-label basket study designed to evaluate the safety and efficacy of milademetan in patients with advanced or metastatic solid tumors refractory or intolerant to standard-of-care therapy that exhibit wild-type (WT) TP53 and MDM2 copy number (CN) ≥ 8 using prespecified biomarker criteria.

Approximately 65 patients will be enrolled to receive milademetan.

Patients will receive the study drug until reaching unequivocal disease progression (per Response Evaluation Criteria in Solid Tumors \[RECIST\] version \[v\]1.1), as determined by the Investigator; experiencing unmanageable toxicity; or until other treatment discontinuation criteria are met. Patients may be treated beyond tumor progression if they are experiencing clinical benefit based on the assessment of the Investigator in discussion with the Medical Monitor.

All patients will be followed for documentation of disease progression and survival information (i.e., date and cause of death). Long-term follow-up will continue every 12 weeks (± 7 days) until the endpoint of death, the patient is lost to follow-up, or for 24 months following the final dose of the study drug, whichever comes first.

Milademetan in Advanced/Metastatic Solid Tumors

A Phase 2 Basket Study of Milademetan in Advanced/Metastatic Solid Tumors (MANTRA-2)

DOR defined as the time from the date of first documentation of CR or PR according to RECIST v1.1 to the date of disease progression or death due to any cause according to Investigator assessment

PFS defined as the time from the date of the first dose of the study drug to the earliest date of the first objective documentation of radiographic disease progression or death due to any cause according to Investigator assessment

GMI defined as the ratio of Time to Progression (TTP) with the nth line of therapy (TTPn; here defined as milademetan) to the most recent prior line of therapy (TTPn-1)

DCR defined as the percentage of patients with confirmed CR, PR, or stable disease (SD) for ≥ 16 weeks

OS as measured from the date of the first dose of the study drug until the date of death due to any cause

Rain Oncology Inc

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
BTX-A51 in Patients With Liposarcoma	NCT06414434	Liposarcoma Recurrent Lipos... Metastatic Lipo... Unresectable Li... MDM2 Gene Ampli...	BTX-A51	18 Years -	Dana-Farber Cancer Institute
Milademetan in Advanced/Metastatic Solid Tumors	NCT05012397	Solid Tumors Head and Neck C... Cholangiocarcin... Sarcoma Lung Adenocarci... Bladder Urothel... Stomach Adenoca... Breast Cancer I... Ovarian Carcino... Cervical Cancer Non Small Cell ... Gastric Cancer Biliary Tract C... Melanoma Pancreas Cancer MDM2 Gene Ampli... Testicular Germ... Adrenocortical ...	RAIN-32	18 Years -	Rain Oncology Inc

Spots Global Cancer Trial Database for mdm2 gene amplification