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Promising drug targets rare cancer in small trial

NCT ID NCT07104331

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial is testing a drug called nab-sirolimus in 41 people with a rare cancer called epithelioid hemangioendothelioma (EHE) that is growing or causing symptoms. The drug is given through an IV twice per cycle. The main goal is to see if it can shrink tumors. Researchers will also track how long the benefit lasts and how long people live without the cancer getting worse.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
nab-sirolimus (a form of the drug sirolimus, given as an IV infusion)
What this could lead to
If it works, this could point toward a new treatment option for people with EHE, a rare blood vessel cancer, by slowing or shrinking tumors.
What could go wrong
This is a small, early-phase trial with only 41 participants, so results may not apply to everyone. The drug may cause side effects like mouth sores or low blood counts, and it may not shrink tumors enough to be considered effective.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 41 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2026

Expected to finish

Aug 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 * 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). Exclusion Criteria: * 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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    6 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Brigham and Women's Hospital

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02115, United States

  • Dana-Farber Cancer Institute

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • Sloan Kettering Institute for Cancer Research

    RECRUITING

    New York, New York, 10065, United States

  • Stanford University

    RECRUITING

    Stanford, California, 94305, United States

  • University of Colorado

    NOT_YET_RECRUITING

    Aurora, Colorado, 80203, United States

  • Washington University St. Louis

    RECRUITING

    St Louis, Missouri, 63130, United States