Soft Tissue SarcomaPhase 2NCT03719430
APX005M and Doxorubicin in Advanced Sarcoma
A Phase 2 study testing Doxorubicin and APX005M in people with Soft Tissue Sarcoma, run by Alexander Z. Wei, MD at 3 sites in United States.
Trial summary
This phase II clinical trial will evaluate the safety and efficacy of adding APX005M (a CD40 agonistic monoclonal antibody) to doxorubicin for the treatment of patients with advanced soft tissue sarcoma. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Soft Tissue Sarcoma
- What is being tested?
- Doxorubicin and APX005M
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 3 sitesUnited States
- Study dates
- 20 Mar 2019to 11 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Histologically confirmed advanced soft tissue sarcoma for which doxorubicin treatment is considered appropriate. Patients with well-differentiated liposarcoma who have histologic evidence of a dedifferentiated component are eligible. Kaposi sarcoma and gastrointestinal stromal tumor (GIST) are not eligible. Protocol Amendment 4 restricts further enrollment to participants with the following sarcoma subtypes. A total of 10 patients will be enrolled with each of the following sarcoma subtypes for the entire study, inclusive of patients enrolled prior to Amendment 4:
- Dedifferentiated liposarcoma
- Leiomyosarcoma
- Myxofibrosarcoma/undifferentiated pleomorphic sarcoma
- Disease must be locally advanced and unresectable or metastatic (that is, considered not amenable to curative surgery or radiation).
- Patients must have measurable disease by RECIST criteria version 1.1.
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- Patients must demonstrate an ECOG performance status of 0 or 1 and be considered an appropriate candidate for anthracycline chemotherapy. There is no limit on prior lines of systemic therapy received. Treatment naïve patients may be enrolled.
- Acceptable laboratory parameters:
- Absolute neutrophil count (ANC) ≥ 1,500/mm3
- Hemoglobin ≥ 9 g/dL
- Platelets ≥ 100,000/mm3
- Creatinine ≤ 1.5 times upper limit of normal OR Calculated creatinine clearance > 45 mL/min
- Total bilirubin ≤ upper limit of normal
- AST/ALT ≤ 1.5 times upper limit of normal
- Patients must have normal left ventricular systolic function, as demonstrated by a transthoracic echocardiogram or MUGA scan at screening, showing a normal left ventricular ejection fraction as defined by the laboratory performing the test.
- Women of child-bearing potential and all men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must agree to use adequate contraception prior to the study, for the duration of study participation, and for 4 months after completion of study drug administration.
- Ability to understand and willingness to sign a written informed consent document.
- After the safety lead-in phase is complete, the next consecutive 10 patients enrolled on the study must have a site of tumor tissue which is amenable to image-guided biopsy by interventional radiology with at most minimal risk to the patient. These 10 patients will be required to undergo tumor biopsy at screening and while on-treatment.
Who cannot join
- Patients must not have received treatment with any chemotherapy, immunotherapy, radiotherapy or an investigational agent for malignancy within the 21 days preceding registration. Patients may not have received treatment with a small molecule targeted anti-cancer agent within 14 days preceding study registration, provided this represents at least 7 half-lives for that agent. Furthermore, toxic effects from any prior therapy (except alopecia) must have resolved to ≤ grade 1 by NCI CTCAE v 5.0 or to the patient's baseline by registration.
- Patients may not be receiving any other investigational agent for any purpose.
- Patients may not have received prior treatment with:
- any anthracycline chemotherapy
- CD40 agonist
- Patients may not have received prior radiotherapy of the mediastinal or pericardial area or whole pelvis radiation.
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- Patients may not have active, known or suspected autoimmune disease with the exceptions of well-controlled: asthma or allergic rhinitis, vitiligo, type 1 diabetes mellitus, psoriasis, or hypothyroidism.
- Patients may not be receiving chronic systemic steroid therapy in excess of physiologic/ replacement doses (prednisone ≤ 10 mg/day is acceptable), or on any other form of immunosuppressive medication for 14 days prior to registration.
- Patients with symptomatic brain metastases may not be enrolled. Those subjects with untreated brain metastases ≤ 1 cm who are asymptomatic and for whom there are no plans for surgery, radiation or corticosteroid use may be considered eligible at the discretion of the principal investigator. Subjects with brain metastases that have been treated and are stable for at least 30 days are eligible if asymptomatic and not receiving corticosteroids. Screening for brain metastases is not required and should not be routinely pursued given their uncommon incidence in sarcoma.
- Patients may not have:
- uncontrolled intercurrent illness including, but not limited to congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmias, uncontrolled diabetes mellitus or uncontrolled psychiatric illness that would limit compliance with study requirements in the opinion of the investigator.
- unstable angina pectoris, angioplasty, cardiac stenting, or myocardial infarction within 6 months of registration.
- any thromboembolic event within 1 month prior to registration
- any active coagulopathy
- any clinically serious, active infection requiring treatment with antibiotics within 14 days prior to registration
- major surgery within 28 days of registration.
- Patients may not have history of another primary cancer, with the exception of:
- curatively treated non-melanomatous skin cancer,
- curatively treated cervical carcinoma in-situ,
- other primary cancers treated with curative intent, no known active disease and no treatment administered within 2 years prior to registration.
- other cancers considered indolent and for which no treatment is anticipated, in the opinion of the principal investigator
- Patients may not be pregnant or nursing.
- Patients may not have known HIV or hepatitis A, B or C infection; however, screening tests for these infections are not required.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
City of Hope
Duarte, California, 91010
Columbia University Irving Medical Center/NYP
New York, New York, 10032
Washington University School of Medicine - Siteman Cancer Center
St Louis, Missouri, 63110
About this study
Official title: A Phase II Trial Evaluating APX005M (a CD40 Agonistic Monoclonal Antibody) in Combination With Standard-of-Care Doxorubicin for the Treatment of Advanced Sarcomas
This phase II clinical trial will evaluate the safety and efficacy of adding APX005M (a CD40 agonistic monoclonal antibody) to doxorubicin for the treatment of patients with advanced soft tissue sarcoma. The investigators believe that doxorubicin, which is currently the standard of care for most advanced sarcomas, could work better when combined with APX005M, which is a type of immunotherapy.
Read the detailed description
Doxorubicin, a chemotherapy, is currently considered standard-of-care treatment for most advanced soft tissue sarcomas. This study will assess the safety and efficacy of combining APX005M, a novel immunomodulatory drug, together with standard of care doxorubicin, for the treatment of patients with advanced soft tissue sarcoma. APX005M is an agonistic monoclonal antibody targeting the CD40 receptor and may have favorable effects on certain types of immune cells in sarcoma tumors, particularly macrophages.
The primary objective is to determine the objective response rate. Secondary objectives include further evaluation of safety and efficacy. A subset of patients will undergo tumor biopsies at baseline and while on study treatment to help understand how the drug combination works and to evaluate how the composition of immune cells in the tumor changes after the treatment.
- Conditions
- Soft Tissue Sarcoma
- Being tested
- Doxorubicin, APX005M
- Keywords
- Leiomyosarcoma, Liposarcoma, Pleomorphic sarcoma, Synovial sarcoma, Malignant peripheral nerve sheath tumor, Spindle cell sarcoma
- Medical subject headings
- Sarcoma, Leiomyosarcoma, Liposarcoma, Sarcoma, Synovial, Neurofibrosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DoxorubicinDrug
Also called ADRIAMYCIN
Doxorubicin is an anthracycline antibiotic with antineoplastic activity 75 mg/m2 IV day 1 (21 day cycles)
APX005MDrug
Also called APX-005M
APX005M is a CD40 agonistic monoclonal antibody 0.3 mg/kg IV day 1 (21 day cycles)
| Study group | Role | What they receive |
|---|---|---|
| Doxorubicin/APX005M | Experimental | Patients will be treated with doxorubicin and APX005M in 21 day cycles. All patients receive the same treatment (there is no "placebo" arm). After completing 8 cycles of study treatment, patients without evidence of disease progression or unacceptable toxicity may continue treatment with APX005M alone. Doxorubicin will not be continued beyond cycle 8 due to the risk for cardiac toxicity from cumulative dosing. Receives: Drug: Doxorubicin; Drug: APX005M |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate
The percentage of patients achieving a partial or complete response as measured by imaging assessments from study treatment
Measured: 6 months
Secondary outcomes (4)
Recommended Dose Combination for APX005M and Doxorubicin and Combination Treatment
A safety-lead in phase will be conducted during which a small number of patients will be treated and monitored closely for certain side effects. If these side effects are seen, the dose of doxorubicin will be adjusted and the study treatment reassessed among another small number of patients. The purpose of the safety lead-in phase is to establish a safe and tolerable dose combination ("the recommended dose") which will be used during the remainder of the study.
Measured: 6 months
Evaluation of Side Effects from APXO05M and Doxorubicin Treatment
Patients on the study will be assessed at regular intervals during clinical visits and through laboratory testing to monitor side effects from the study treatment.
Measured: 18 months
Progression Free Survival
The mean time to either disease progression or death, whichever comes first, for patients on the study
Measured: 18 months
Objective Response Rate (ORR)
Objective response rate in patients with dedifferentiated liposarcoma, leiomyosarcoma, and myxofibrosarcoma/undifferentiated pleomorphic sarcoma. The confirmed ORR per RECIST version 1.1. criteria will be evaluated in the group of patients with these 3 sarcoma subtypes and within each of these subtypes.
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 posted25 Oct 2018
- Study start20 Mar 2019
- Primary completion11 Dec 2025
- Study completion11 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Soft Tissue SarcomaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexander Z. Wei, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Apexigen America, Inc.
- Responsible party
- Sponsor-investigator, Alexander Z. Wei, MD, Columbia University
- Investigators
- Alexander Wei, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03719430
- Sponsor's protocol ID
- AAAS0095
- Status last verified
- Jun 2026
- First submitted
- 22 Oct 2018
- Last updated
- 10 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial Evaluating APX005M (a CD40 Agonistic Monoclonal Antibody) in Combination With Standard-of-Care Doxorubicin for the Treatment of Advanced Sarcomas
- ClinicalTrials.gov ID
- NCT03719430
- Acronym / sponsor ID
- AAAS0095
- Conditions
- Soft Tissue Sarcoma
- Keywords
- Leiomyosarcoma, Liposarcoma, Pleomorphic sarcoma, Synovial sarcoma, Malignant peripheral nerve sheath tumor, Spindle cell sarcoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Alexander Z. Wei, MDUniversity, hospital or other
- Collaborators
- Apexigen America, Inc.
- Study start
- 20 Mar 2019 (actual)
- Primary completion
- 11 Dec 2025 (actual)
- Study completion
- 11 Dec 2025 (actual)
- First posted
- 25 Oct 2018 (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 phase II clinical trial will evaluate the safety and efficacy of adding APX005M (a CD40 agonistic monoclonal antibody) to doxorubicin for the treatment of patients with advanced soft tissue sarcoma. The investigators believe that doxorubicin, which is currently the standard of care for most advanced sarcomas, could work better when combined with APX005M, which is a type of immunotherapy.
Detailed description
Doxorubicin, a chemotherapy, is currently considered standard-of-care treatment for most advanced soft tissue sarcomas. This study will assess the safety and efficacy of combining APX005M, a novel immunomodulatory drug, together with standard of care doxorubicin, for the treatment of patients with advanced soft tissue sarcoma. APX005M is an agonistic monoclonal antibody targeting the CD40 receptor and may have favorable effects on certain types of immune cells in sarcoma tumors, particularly macrophages.
The primary objective is to determine the objective response rate. Secondary objectives include further evaluation of safety and efficacy. A subset of patients will undergo tumor biopsies at baseline and while on study treatment to help understand how the drug combination works and to evaluate how the composition of immune cells in the tumor changes after the treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Doxorubicin/APX005M | Experimental | Drug: Doxorubicin, Drug: APX005MPatients will be treated with doxorubicin and APX005M in 21 day cycles. All patients receive the same treatment (there is no "placebo" arm). After completing 8 cycles of study treatment, patients without evidence of disease progression or unacceptable toxicity may continue treatment with APX005M alone. Doxorubicin will not be continued beyond cycle 8 due to the risk for cardiac toxicity from cumulative dosing. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Histologically confirmed advanced soft tissue sarcoma for which doxorubicin treatment is considered appropriate. Patients with well-differentiated liposarcoma who have histologic evidence of a dedifferentiated component are eligible. Kaposi sarcoma and gastrointestinal stromal tumor (GIST) are not eligible. Protocol Amendment 4 restricts further enrollment to participants with the following sarcoma subtypes. A total of 10 patients will be enrolled with each of the following sarcoma subtypes for the entire study, inclusive of patients enrolled prior to Amendment 4:
- Disease must be locally advanced and unresectable or metastatic (that is, considered not amenable to curative surgery or radiation).
- Patients must have measurable disease by RECIST criteria version 1.1.
- Patients must demonstrate an ECOG performance status of 0 or 1 and be considered an appropriate candidate for anthracycline chemotherapy. There is no limit on prior lines of systemic therapy received. Treatment naïve patients may be enrolled.
- Acceptable laboratory parameters:
Key exclusion criteria (23 listed)
- Patients must not have received treatment with any chemotherapy, immunotherapy, radiotherapy or an investigational agent for malignancy within the 21 days preceding registration. Patients may not have received treatment with a small molecule targeted anti-cancer agent within 14 days preceding study registration, provided this represents at least 7 half-lives for that agent. Furthermore, toxic effects from any prior therapy (except alopecia) must have resolved to ≤ grade 1 by NCI CTCAE v 5.0 or to the patient's baseline by registration.
- Patients may not be receiving any other investigational agent for any purpose.
- Patients may not have received prior treatment with:
- Patients may not have received prior radiotherapy of the mediastinal or pericardial area or whole pelvis radiation.
- Patients may not have active, known or suspected autoimmune disease with the exceptions of well-controlled: asthma or allergic rhinitis, vitiligo, type 1 diabetes mellitus, psoriasis, or hypothyroidism.
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 |
|---|---|---|
| DoxorubicinAlso: ADRIAMYCINDoxorubicin is an anthracycline antibiotic with antineoplastic activity 75 mg/m2 IV day 1 (21 day cycles) | Drug | Doxorubicin/APX005M |
| APX005MAlso: APX-005MAPX005M is a CD40 agonistic monoclonal antibody 0.3 mg/kg IV day 1 (21 day cycles) | Drug | Doxorubicin/APX005M |
Primary outcome
Objective Response Rate
Time frame: 6 months
The percentage of patients achieving a partial or complete response as measured by imaging assessments from study treatment
4 secondary outcomes
Recommended Dose Combination for APX005M and Doxorubicin and Combination Treatment
Time frame: 6 months
Evaluation of Side Effects from APXO05M and Doxorubicin Treatment
Time frame: 18 months
Progression Free Survival
Time frame: 18 months
Objective Response Rate (ORR)
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2026.
- Individual participant dataSharing planned: No
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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.
Alexander Wei, MD principal investigator
Columbia University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Doxorubicin and APX005M in people with Soft Tissue Sarcoma, run by Alexander Z. Wei, MD at 3 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 23 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: finished, as of the sponsor's last update on 10 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Objective Response Rate (time frame: 6 months). 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 10 Jun 2026.