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New drug targets cancer spread in first human trial

NCT ID NCT04222413

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Aug 28, 2026 · Updated 17 times

Summary

This early-phase trial tests a new drug called metarrestin in people with advanced solid tumors that have spread (metastasized). The goal is to find a safe dose and see if it can shrink tumors. The study includes adults with pancreatic, breast, or other solid tumors, and children aged 12-17 with certain solid tumors. Participants take metarrestin by mouth and are closely monitored.

What this could mean

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

Active substance
Metarrestin (ML-246)
What this could lead to
If it works, this could point toward a new treatment that slows or shrinks metastatic solid tumors, potentially improving outcomes for patients with advanced cancer.
What could go wrong
This is a very early Phase 1 trial focused on safety and dosing, so it is unknown if metarrestin will effectively shrink tumors. Side effects may occur, and the drug may not work for all cancer types studied.

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 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 116 people

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

Started

Oct 2020

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

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

Ages

12 to 120 years

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: * Adult (\>= 18 years) subjects with: * histologically or cytologically confirmed solid tumors (Phase IA). OR --histologically or cytologically confirmed pancreatic, colorectal, or breast cancer (Phase IB) OR * Pediatric (\>=12 and \< 18 years) subjects with histologically or cytologically confirmed solid tumors other than rhabdomyosarcoma (RMS) including embryonal, alveolar, spindle cell/sclerosing and pleomorphic subtypes of RMS (Phase IB). * Subjects must have disease that: * is not amenable to potentially curative resection, * spread at least to one other organ system other than primary tumor or recurred after removal of primary tumor * has site measurable per RECIST 1.1 (Phase IB only). * progressed on or after at least one line of standard systemic chemotherapy (Phase IA and IB1) * have no standard therapy option available (Phase IB2) * Patients must have recovered from any acute toxicity related to prior therapy or surgery or disease to a grade 1 or less. * Performance status * Karnofsky \>= 70% (for patients \>= 16 years old), Lansky \>= 70% (for patients \<16 years old) * Adequate hematological function defined by: * absolute neutrophil count (ANC) \>= 1.0 x 10(9)/L, * transfusion-independent platelet count \>= 100 x 10(9)/L, * Hgb \>= 9 g/ dL (patients who have received \<= 2 PRBC transfusions within 48 hours are eligible) * Adequate coagulation as defined by: --INR\<1.5 (or \< 3.0 if subjects are currently taking anticoagulated medications) Note: increase of the upper limit of INR is restricted only to subjects who are receiving anticoagulation for medical reasons (DVT/PE prophylaxis, treatment for a thromboembolic event) and have increased INR because of these medications. Patients who have an elevated INR due to compromised liver function or any other medical conditions remain excluded * Adequate hepatic function defined by: --a total bilirubin level \<= 1.5 x ULN, (total bilirubin \<= 2.0 x ULN in case of prior diagnosis of Gilbert syndrome) * an AST level \<= 3xULN * an ALT level \<= 3 xULN * Adequate renal function defined by: --Creatinine OR Measured or calculated creatinine clearance (CrCl) (eGFR may also be used in place of CrCl)\* ---\< 1.5x institution upper limit of normal OR ---\>= 45 mL/min/1.73 m\^2 for participant with creatinine levels \>= 1.5 X institutional ULN \*Creatinine clearance (CrCl) or eGFR should be calculated per institutional standard. * The effects of the study treatment on the developing human fetus are unknown; thus, individuals of childbearing potential and individuals who can father children must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study therapy and up to 120 days after the last dose of the study drug. * Nursing participants must be willing to discontinue nursing at the time of the study treatment initiation. * Weight: \>= 35 kg (\>= 18 years old) or \>=40 kg (\>= 12 and \< 18 years old). * Ability of subject or parent/guardian to understand and the willingness to sign a written informed consent document. * Subjects must have lesion(s) accessible for biopsy (other than used for measurement of disease) and be willing to undergo mandatory study biopsies (Cohort IB1 only). * Ability to swallow oral capsules. EXCLUSION CRITERIA: * Anticancer treatment within designated period before treatment initiation including: * minor surgical procedure (such as biliary stenting) within 14 days. Note: if liver function tests after biliary stenting or renal function tests after ureteral stenting return to normal, within 5 days after biliary or ureteral stenting; * major surgical procedure or curative radiation treatment within 28 days; * palliative radiation treatment within 14 days; * chemotherapy or experimental drug treatment with published half-life known to be 72 hours or less within 14 days; * experimental drug treatment with unpublished or half-life greater than 72 hours within 28 days; * chemotherapy regimen containing an alkylating antineoplastic agent (cyclophosphamide, chlorambucil, melphalan, or ifosfamide), alkylating-like (platinumbased chemotherapeutic drugs, platinum analogues), and non-classical alkylating agent (dacarbazine, temozolomide) within 28 days. * Patients receiving any medications or substances that are moderate and strong inhibitors or inducers of CYP3A4 and are not able to safely stop these medications are excluded from this study; patients must stop strong CYP3A4 inhibiting/inducing medications within 5 published half-lives and moderate within 3 published half-lives prior to the treatment initiation. Note: dihydropyridine calcium - channel blockers are permitted for management of underling disease. * Subjects with cardiomyopathy diagnosed within 6 months prior to treatment initiation including but not limited to the following: * hypertrophic cardiomyopathy * arrhythmogenic right ventricular cardiomyopathy * abnormal ejection fraction (echocardiogram \[ECHO\]) \<= 53% (if a range is given then the upper value of the range will be used) * previous moderate or severe impairment of left ventricular systolic function (LVEF \<45%) * severe valvular heart disease * atrial fibrillation with a ventricular rate \>100 bpm on EKG at rest * Fridericia's corrected QT interval (QTcF) \>= 480 msec (adults) or \>= 460 msec (pediatric subjects, aged 12 to \<18 years) or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome. * HIV, HCV, HBV positive patients on antiviral drugs are excluded due to the absence of previous experience with concurrent use of antiviral medications and the investigational drug product to be evaluated in the current study and possible for adverse pharmacokinetic and/or pharmacodynamic interactions. * Previous malignant disease (other than the target malignancy to be investigated in this trial) within the last 3 years. Note: subjects with a history of cervical carcinoma in situ, superficial or non-invasive bladder cancer, or basal cell or squamous cell carcinoma in situ previously treated with curative intent are NOT excluded. * Rapidly progressive disease which, in the opinion of the Investigator, may predispose to inability to tolerate treatment or trial procedures. * Subjects with central nervous system (CNS) metastases or CNS disorders known to increase possible neurotoxicity of metarrestin in case of compromised blood-brain barrier (e.g. recent stroke (\<3 months of treatment initiation), infectious causes). * Significant acute or chronic infections including tuberculosis with presence of clinical symptoms or physical findings. * Patients with a history of any seizures or increased risk of seizures on screening EEGs defined by 1) interictal epileptiform discharges, 2) temporal intermittent rhythmic delta activity (TIRDA), or 3) electrographic or clinical seizures on EEG. * Clinically relevant diseases (for example, inflammatory bowel disease) and / or uncontrolled medical conditions, which, in the opinion of the Investigator, might impair the subject's tolerance or ability to participate in the trial. * Patients with previous gastric bypass, patients receiving nutrition via feeding tubes or parenterally, or patients with malabsorptive conditions (damage to the intestine from infection, inflammation, trauma, or surgery, celiac disease, Crohn's disease, chronic pancreatitis, or cystic fibrosis resulting malabsorption). Patients with refractory nausea and vomiting. Note: patients with gastric banding are allowed. * Pregnant individuals.

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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

    2 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

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

  • University of Kansas

    RECRUITING

    Fairway, Kansas, 66205, United States

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