Epithelioid Hemangioendothelioma (EHE)Phase 2NCT07104331
SARC046: A Phase II Trial of Nab-Sirolimus in Patients With Progressing or Symptomatic Epithelioid Hemangioendothelioma
A Phase 2 study testing nab-Sirolimus in people with Epithelioid Hemangioendothelioma (EHE), run by Sarcoma Alliance for Research through Collaboration at 6 sites in United States.
Trial summary
This is a non-randomized, open label, single arm Phase II trial with a two-stage design with histologically-confirmed metastatic and/or recurrent epithelioid hemangioendothelioma requiring systemic treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Epithelioid Hemangioendothelioma (EHE)
- What is being tested?
- nab-Sirolimus
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 6 sitesUnited States
- Study dates
- 24 Feb 2026to 30 Aug 2029
- Phase
- Phase 2
- 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
- Participants must have histologically or cytologically confirmed EHE that is either progressing or clinically symptomatic, not a candidate for curative intent surgery, and requires systemic therapy in the opinion of the investigator.
- Participants must have measurable disease by RECIST v1.1, 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 12 (Measurement of Effect) for the evaluation of measurable disease.
- Age ≥18 years
- ECOG performance status ≤2
- Participants must meet the following organ and marrow function as defined below:
- Platelets >75,000μl
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- ANC >1500μl
- Hgb >9g/dl
- Creatinine <1.5 x ULN or measured CrCl of >30ml/m2/1.73 m2
- Total bilirubin <2 x ULN
- AST/ALT <3 x ULN
- 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). Toxicities that are permanent, like hearing loss from platinum agents, may be allowed if agreed to by the medical monitor.
- The effects of nab-sirolimus on the developing human fetus are unknown. For this reason, women of child-bearing potential and 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 also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of nab-sirolimus administration.
- 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. (Providing consents in as many languages as possible is encouraged).
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. HIV+ patients are allowed provided the patient has a CD4 count greater than 200 and is HIV undetectable on antiviral medications, and there are no AIDS-defining illnesses within 12 months. The antiviral medication regimen must not contain a strong CYP3A4 inhibitor (eg protease inhibitors).
- 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. Subjects 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
- Co-existing malignancies or use of any other concurrent investigational agents or anticancer agents, excluding adjuvant 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 nab-sirolimus, breastfeeding should be discontinued if the mother is treated with nab-sirolimus.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Not yet recruiting
University of Colorado
Aurora, Colorado, 80203
- Not yet recruiting
Brigham and Women's Hospital
Boston, Massachusetts, 02115
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Dana-Farber Cancer Institute, 877-338-7425, sarc046@sarctrials.org
Michael Wagner, MD
- Recruiting
Sloan Kettering Institute for Cancer Research
New York, New York, 10065
Jennifer Wu, 646-888-6787, wum2@mskcc.org
- Recruiting
Washington University St. Louis
St Louis, Missouri, 63130
Cheryl Callahan, 314-286-2584, callahanc@wustl.edu
- Recruiting
Stanford University
Stanford, California, 94305
Maria Ahern, 650-725-6413, mahern@stanford.edu
About this study
This is a non-randomized, open label, single arm Phase II trial with a two-stage design with histologically-confirmed metastatic and/or recurrent epithelioid hemangioendothelioma requiring systemic treatment. nab-Sirolimus 100 mg/m2 will be administered as an intravenous infusion over 30 minutes on Days 1 and 8 of each 21-day cycle. The primary objective is to determine ORR by RECIST v1.1 of nab-sirolimus in patients with EHE who require systemic treatment.
- Conditions
- Epithelioid Hemangioendothelioma (EHE)
- Being tested
- nab-Sirolimus
- Medical subject headings
- Hemangioendothelioma, Epithelioid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nab-SirolimusDrug
nab-Sirolimus 100 mg/m2 will be administered as an intravenous infusion over 30 minutes on Days 1 and 8 of each 21-day cycle.
| Study group | Role | What they receive |
|---|---|---|
| non-randomized, open label, single arm | Experimental | Not described Receives: Drug: nab-Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tumor response rate
To determine ORR by RECIST v1.1 of nab-sirolimus in patients with EHE who require systemic treatment.
Measured: Approximately 24 months
Secondary outcomes (2)
Progression-free survival
The time from starting treatment until disease progression
Measured: Month 6 and 12
Duration of response
The time from tumor response to disease progression
Measured: Approximately 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2025
- Study start24 Feb 2026
- Primary completion30 Aug 2029 (estimated)
- Study completion30 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epithelioid Hemangioendothelioma (EHE)Studies 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
- Model description
- This is a non-randomized, open label, single arm Phase II trial with a two stage design
- Masking
- None (open label)
- Enrolment
- 41 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarcoma Alliance for Research through Collaboration
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07104331
- Sponsor's protocol ID
- SARC046
- Status last verified
- May 2026
- First submitted
- 29 Jul 2025
- Last updated
- 26 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: There is not a plan to make IPD available.
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SARC046: A Phase II Trial of Nab-Sirolimus in Patients With Progressing or Symptomatic Epithelioid Hemangioendothelioma
- ClinicalTrials.gov ID
- NCT07104331
- Acronym / sponsor ID
- SARC046
- Conditions
- Epithelioid Hemangioendothelioma (EHE)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 41 participants (estimated)
- Lead sponsor
- Sarcoma Alliance for Research through CollaborationUniversity, hospital or other
- Study start
- 24 Feb 2026 (actual)
- Primary completion
- 30 Aug 2029 (estimated)
- Study completion
- 30 Aug 2029 (estimated)
- First posted
- 5 Aug 2025 (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 is a non-randomized, open label, single arm Phase II trial with a two-stage design with histologically-confirmed metastatic and/or recurrent epithelioid hemangioendothelioma requiring systemic treatment. nab-Sirolimus 100 mg/m2 will be administered as an intravenous infusion over 30 minutes on Days 1 and 8 of each 21-day cycle. The primary objective is to determine ORR by RECIST v1.1 of nab-sirolimus in patients with EHE who require systemic treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a non-randomized, open label, single arm Phase II trial with a two stage design
| Arm | Type | Receives |
|---|---|---|
| non-randomized, open label, single arm | Experimental | Drug: nab-Sirolimus |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Participants must have histologically or cytologically confirmed EHE that is either progressing or clinically symptomatic, not a candidate for curative intent surgery, and requires systemic therapy in the opinion of the investigator.
- Participants must have measurable disease by RECIST v1.1, 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 12 (Measurement of Effect) for the evaluation of measurable disease.
- Age ≥18 years
- ECOG performance status ≤2
- Participants must meet the following organ and marrow function as defined below:
Key exclusion criteria (8 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. HIV+ patients are allowed provided the patient has a CD4 count greater than 200 and is HIV undetectable on antiviral medications, and there are no AIDS-defining illnesses within 12 months. The antiviral medication regimen must not contain a strong CYP3A4 inhibitor (eg protease inhibitors).
- 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. Subjects 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 |
|---|---|---|
| nab-Sirolimusnab-Sirolimus 100 mg/m2 will be administered as an intravenous infusion over 30 minutes on Days 1 and 8 of each 21-day cycle. | Drug | non-randomized, open label, single arm |
Primary outcome
Tumor response rate
Time frame: Approximately 24 months
To determine ORR by RECIST v1.1 of nab-sirolimus in patients with EHE who require systemic treatment.
2 secondary outcomes
Progression-free survival
Time frame: Month 6 and 12
Duration of response
Time frame: Approximately 12 months
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 26 May 2026.
- Individual participant dataSharing planned: No. There is not a plan to make IPD available.
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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.
6 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 nab-Sirolimus in people with Epithelioid Hemangioendothelioma (EHE), run by Sarcoma Alliance for Research through Collaboration at 6 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 15 inclusion and 8 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: looking for participants now, as of the sponsor's last update on 26 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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?
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tumor response rate (time frame: Approximately 24 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 26 May 2026.