LiposarcomaPhase 1NCT06414434
BTX-A51 in Patients With Liposarcoma or CIC-rearranged Sarcoma
A Phase 1 study testing BTX-A51 in people with Liposarcoma and Recurrent Liposarcoma, run by Michael Wagner, MD at 2 sites in United States.
Trial 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) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Liposarcoma, Recurrent Liposarcoma, Metastatic Liposarcoma
- What is being tested?
- BTX-A51
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 2 sitesUnited States
- Study dates
- 30 Sep 2024to 1 Jun 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Brigham and Women's Hospital
Boston, Massachusetts, 02215
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
About this study
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
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)
Read the 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
- Being tested
- BTX-A51
- Keywords
- Liposarcoma, Recurrent Liposarcoma, Metastatic Liposarcoma, Unresectable Liposarcoma, MDM2 Gene Amplification, Myxoid Liposarcoma, CIC-Rearranged Sarcoma
- Medical subject headings
- Liposarcoma, Liposarcoma, Myxoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BTX-A51Drug
Also called 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),
Multi-kinase inhibitor, 1.0 mg, 2.0 mg, and 7.0 mg immediate-release capsules, taken orally per protocol.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: BTX-A51 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
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.
Measured: ORR expected to be observed up to 3 years
1-year Progression-Free Survival (PFS) Rate
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.
Measured: 1 year
1-year Overall Survival (OS)
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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2024
- Study start30 Sep 2024
- Primary completion15 Jan 2027 (estimated)
- Study completion1 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LiposarcomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Michael Wagner, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Edgewood Oncology Inc.
- Responsible party
- Sponsor-investigator, Michael Wagner, MD, Dana-Farber Cancer Institute
- Investigators
- Michael Wagner, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06414434
- Sponsor's protocol ID
- 24-156
- Status last verified
- Jun 2026
- First submitted
- 7 May 2024
- Last updated
- 1 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06414434
- Acronym / sponsor ID
- 24-156
- Conditions
- Liposarcoma, Recurrent Liposarcoma, Metastatic Liposarcoma, Unresectable Liposarcoma, MDM2 Gene Amplification, Myxoid Liposarcoma
- Keywords
- Liposarcoma, Recurrent Liposarcoma, Metastatic Liposarcoma, Unresectable Liposarcoma, MDM2 Gene Amplification, Myxoid Liposarcoma, CIC-Rearranged Sarcoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Michael Wagner, MDUniversity, hospital or other
- Collaborators
- Edgewood Oncology Inc.
- Study start
- 30 Sep 2024 (actual)
- Primary completion
- 15 Jan 2027 (estimated)
- Study completion
- 1 Jun 2027 (estimated)
- First posted
- 16 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BTX-A51 21mg | Experimental | Drug: BTX-A51Participants 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. |
| BTX-A51 30mg | Experimental | Drug: BTX-A51Participants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- 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:
- 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.
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BTX-A51Also: 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),Multi-kinase inhibitor, 1.0 mg, 2.0 mg, and 7.0 mg immediate-release capsules, taken orally per protocol. | Drug | BTX-A51 21mg, BTX-A51 30mg |
Primary outcome
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
Time frame: All AEs will be recorded from the time the subject signs informed consent until 30 days after the last dose of study BTX-A51.
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.
3 secondary outcomes
Overall Response Rate (ORR)
Time frame: ORR expected to be observed up to 3 years
1-year Progression-Free Survival (PFS) Rate
Time frame: 1 year
1-year Overall Survival (OS)
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2026.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Wagner, MD principal investigator
Dana-Farber Cancer Institute
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BTX-A51 in people with Liposarcoma and Recurrent Liposarcoma, run by Michael Wagner, MD at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 1 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: All AEs will be recorded from the time the subject signs informed consent until 30 days after the last dose of study BTX-A51.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2026.