Nab-Sirolimus for EHE

Not yet recruiting at 5 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sarcoma Alliance for Research through Collaboration

Must not be taking: Protease inhibitors

Disqualifiers: Active infection, CHF, Cirrhosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 2

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called nab-Sirolimus to determine its effectiveness for people with epithelioid hemangioendothelioma (EHE), a rare cancer that has spread or recurred and cannot be surgically removed. The goal is to assess how well this drug shrinks tumors in patients requiring systemic treatment. Individuals diagnosed with worsening or symptomatic EHE, who are not candidates for surgery, might be suitable for this trial. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, if you are on antiviral medications, they must not contain a strong CYP3A4 inhibitor. It's best to discuss your current medications with the trial team.

Is there any evidence suggesting that nab-Sirolimus is likely to be safe for humans?

Research shows that nab-Sirolimus benefits the treatment of advanced malignant PEComa, a type of cancer. This treatment has undergone testing in people, providing some safety information. Past studies have used nab-Sirolimus in both early and later trials for various cancers, indicating it is generally well-tolerated. However, animal studies suggest it could harm unborn babies, so pregnant women should exercise caution. Although more research is needed for specific conditions like EHE, current data offers some reassurance about its use in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for epithelioid hemangioendothelioma (EHE), which often include surgery, radiation, or conventional chemotherapy, nab-Sirolimus offers a novel approach by utilizing a nanoparticle albumin-bound formulation of sirolimus. This unique delivery system enhances the drug's absorption and distribution, potentially improving its effectiveness against the tumor. Researchers are excited about nab-Sirolimus because it targets the mTOR pathway, which is crucial in cancer cell growth and proliferation, offering a more targeted and potentially less toxic treatment option for patients with EHE.

What evidence suggests that nab-Sirolimus might be an effective treatment for EHE?

Studies have shown that sirolimus, a drug that blocks certain cell growth signals, can be effective against epithelioid hemangioendothelioma (EHE), a rare cancer affecting blood vessels. Research suggests that sirolimus can help manage EHE, even though it is not officially approved for this use. Nab-sirolimus, the treatment tested in this trial, is a version of sirolimus attached to a protein called albumin, which may help it reach tumors more effectively. Early research indicates that nab-sirolimus might lead to better results for EHE patients due to its targeted approach. ^{4 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for patients with a rare vascular cancer called Epithelioid Hemangioendothelioma (EHE) that's spreading or causing symptoms, and who need systemic treatment. Specific eligibility details are not provided but typically include factors like age, health status, and previous treatments.

Inclusion Criteria

I have a tumor that can be measured by scans or x-rays.

My blood counts and kidney/liver functions are within required ranges.

Ability to understand and the willingness to sign a written informed consent document.

Exclusion Criteria

Pregnant women are excluded from this study.

I haven't had major surgery or an open biopsy in the last 28 days.

I have not had a heart attack in the last year.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

nab-Sirolimus 100 mg/m2 administered as an intravenous infusion over 30 minutes on Days 1 and 8 of each 21-day cycle

24 months

2 visits per cycle (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

What Are the Treatments Tested in This Trial?

Interventions

• nab-Sirolimus

Trial Overview The trial is testing nab-Sirolimus as an intravenous infusion to treat EHE. Patients receive the drug on Days 1 and 8 of each 21-day cycle. The main goal is to see how well the tumor responds to this treatment according to standard criteria (RECIST v1.1).

How Is the Trial Designed?

Group I: non-randomized, open label, single arm Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Sarcoma Alliance for Research through Collaboration

Lead Sponsor

Trials

26

Recruited

2,000+

Citations

1. withpower.com withpower.com/trial/phase-2-epithelioid-hemangioendothelioma-ehe-11-2025-07fcb

Nab-Sirolimus for EHE · Info for Participants This trial is for patients with a rare vascular cancer called Epithelioid Hemangioendothelioma ... nab-Sirolimus as an intravenous infusion to treat EHE ...

2. ascopubs.org ascopubs.org/doi/10.1200/JCO.2019.37.15_suppl.11065

Activity of sirolimus in advanced epithelioid ... Activity of sirolimus in advanced epithelioid hemangioendothelioma (EHE): A retrospective analysis within the Italian Rare Tumor Network (RTR).

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11853918/

Nab-Paclitaxel Based Chemotherapy in the Treatment of ... To evaluate the clinical efficacy of nab-paclitaxel based chemotherapy in the treatment of advanced epithelioid hemangioendothelioma (EHE).

4. frontiersin.org frontiersin.org/journals/oncology/articles/10.3389/fonc.2024.1367237/full

The patient perspective on sirolimus for epithelioid ... Retrospective evidence supports the activity of mTOR inhibitors (e.g. sirolimus), although available only off-label. EHE patients and advocates ...

5. synapse.patsnap.com synapse.patsnap.com/drug/9526c845891e450aaa32435360869b83

Sirolimus Albumin-Bound - Drug Targets, Indications, Patents A Randomized, Double-blind, Multicenter Phase III Clinical Study to Evaluate the Efficacy and Safety of Sirolimus for Injection (Albumin-bound) Combined ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11095855/

Phase II Trial of nab-Sirolimus in Patients With Advanced ... These results highlight the long-term clinical benefit of nab-sirolimus in patients with advanced malignant PEComa, with a DOR of >3 years.

7. researchgate.net researchgate.net/publication/335052621_Abstract_348_ABI-009_nab-Sirolimus_improves_tumor_accumulation_and_antitumor_activity_over_oral_mTOR_inhibitors

Abstract 348: ABI-009 (nab-Sirolimus) improves tumor ... ABI-009 is currently in phase 1 and 2 clinical studies for the treatment of multiple cancer types, including bladder cancer, soft-tissue sarcomas, ...

8. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2021/213312lbl.pdf

FYARRO (SIROLIMUS ALBUMIN-BOUND NANOPARTICLES ... Based on animal studies and the mechanism of action, FYARRO can cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology (12.1)].