ClinicalTrials.gg
RecruitingNCT04222413Updated Nov 9, 2026

Metarrestin (ML-246) in Subjects With Metastatic Solid Tumors

A Phase 1 interventional study of Metarrestin in Advanced Solid Tumors, Metastatic Pancreatic Cancer and Pediatric Solid Tumor, sponsored by National Cancer Institute (NCI). Recruiting at 2 sites in United States. Open to participants aged 12 Years to 120 Years. Per ClinicalTrials.gov, last updated 2026-11-09.

Sponsored by National Cancer Institute (NCI) · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Started Oct 2020; still recruiting 6 years later.
Updated Oct 30, 2026Eligibility revisedGo to Updates ↓
Phase
Phase 1
Study type
Interventional
Enrollment
116
Allocation
Non-randomized
Ages
12 Years to 120 Years
Sex
All
01

Study summary

Background:

Metastasis is the spread of cancer from one organ to a nonadjacent organ. It causes 90% of cancer deaths. No treatment specifically prevents or reduces metastasis. Researchers hope a new drug can help. It stops cancer cells from growing and spreading further and possibly shrink cancer lesions in distant organs.

Objective:

To find a safe dose of metarrestin and to see if this dose shrinks tumors.

Eligibility:

Adults age 18 and older with pancreatic cancer, breast cancer, or a solid tumor that has not been cured by standard therapies. Also, children age 12-17 with a solid tumor (other than a muscle tumor) with no standard therapy options.

Design:

Participants will be screened with:

  • blood tests
  • physical exam
  • documentation of disease confirmation or tumor biopsy
  • electrocardiogram to evaluate the heart
  • review of their medicines and their ability to do their normal activities

Participants will take metarrestin by mouth until they cannot tolerate it or stop to benefit from it. They will keep a medicine diary.

Participants will visit the Clinical Center. During the first month there are two brief hospital stays required with visits weekly or every other week thereafter. They will repeat some of the screening tests. They will fill out questionnaires. They will have tests of their cognitive function. They will have an electroencephalogram to record brain activity. They will have a computed tomography (CT) scan or magnetic resonance imaging (MRI). A CT is a series of X-rays of the body. An MRI uses magnets and radio waves to take pictures of the body.

Adult participants may have tumor biopsies.

Participants will have a follow-up visit 30 days after treatment ends. Then they will have follow-up phone calls or emails every 6 months for the rest of their life or until the study ends.

...

Read the detailed description

Background:

Metarrestin is a first-in-class investigational agent targeting the peri-nucleolar compartment (PNC), a marker of genome organization associated with metastasis.

Preclinical studies have shown that metarrestin effectively suppresses metastasis and extends overall survival in different cancer models.

Multi-species allometric scaling and good laboratory practice (GLP) toxicology and toxicokinetic studies indicate that metarrestin administered at a calculated safe maximum recommended starting dose (MRSD) to human subjects is predicted to afford intratumoral exposure levels within the therapeutic range observed preclinically.

Objectives:

Phase IA: To determine the maximum tolerated dose (MTD) of metarrestin.

Phase IB: To determine the Objective Response Rate (ORR) according to Evaluation Criteria (RECIST 1.1) in patients treated with metarrestin at the MTD.

Eligibility:

Adult subjects with any advanced solid tumors (Cohort IA), or pancreatic, colorectal, or breast tumors (Cohort IB1).

OR

Pediatric subjects age 12 and older with solid tumors other than rhabdomyosarcoma (RMS) including embryonal, alveolar, spindle cell/sclerosing, and pleomorphic subtypes of RMS (Cohort IB2).

Patients must have progressed on prior standard chemotherapeutic therapy.

Design:

This is first-in-human Phase I trial to investigate the safety and clinical activity of metarrestin in subjects with metastatic solid tumors.

During Phase IA MTD of metarrestin will be estimated in adult patients with solid tumors.

During Phase IB adult patients with breast, colorectal, or pancreatic cancer, and pediatric patients with solid organ cancer will be treated at dose level of estimated MTD.

Patients will receive treatment in cycles consisting of 28 (+7/- 2) days.

Metarrestin will be administered PO until progression or unacceptable toxicity.

02

Conditions studied

  • Advanced Solid Tumors
  • Metastatic Pancreatic Cancer
  • Pediatric Solid Tumor
  • Advanced Breast Cancer
  • Malignant Peripheral Nerve Sheath Tumor
  • Colorectal Neoplasms

Keywords

  • First-In-Class Investigational Agent
  • Peri-Nucleolar Compartment (PNC)
  • effective therapies against metastasis
  • Oral Administration
  • Maximum Recommended Starting Dose
03

In context

Pancreatic Neoplasms

3,896 studies on the registry are indexed under Pancreatic Neoplasms; 1,007 are open to participants now.

This study's planned enrollment of 116 is above the median of 47 across 2,932 interventional studies indexed under Pancreatic Neoplasms.

Browse Pancreatic Neoplasms studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.

Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

The criteria were revised on the registry on Oct 30, 2026. See what changed

Ages eligible
12 Years to 120 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Adult (>= 18 years) subjects with:

    • 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 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

    • >= 70% (for patients >= 16 years old), Lansky >= 70% (for patients \<16 years old)
  • Adequate hematological function defined by:

    • (ANC) >= 1.0 x 10(9)/L,
    • transfusion-independent count >= 100 x 10(9)/L,
    • >= 9 g/ dL (patients who have received \<= 2 PRBC transfusions within 48 hours are eligible)
  • Adequate coagulation as defined by:

    --\<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 level \<= 1.5 x , (total bilirubin \<= 2.0 x ULN in case of prior diagnosis of Gilbert syndrome)

    • an level \<= 3xULN
    • an ALT level \<= 3 xULN
  • Adequate renal function defined by:

    --Creatinine OR Measured or calculated (CrCl) ( 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 and individuals who can father children must agree to use adequate (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 document.
  • Subjects must have lesion(s) accessible for (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

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 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.

  • Patients receiving medications known to carry a high to moderate risk to lower seizure threshold within 2 weeks prior to treatment initiation.
  • 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 (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 at rest
    • Fridericia's 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.
  • , , positive patients on antiviral drugs are excluded due to the absence of previous experience with concurrent use of antiviral medications and the 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 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.
  • Significant acute or chronic infections including tuberculosis with presence of clinical symptoms or physical findings.
  • Patients with a history of any seizures within 1 year prior to the study treatment initiation 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. Note: Patients with are eligible providing that patients had no seizures or clinical signs of central nervous system (CNS) disease-related neurotoxicity (ataxia, sensory, cognitive changes) within 1 year prior to the study treatment initiation and baseline EEG shows no changes indicative of increased seizure risk.
  • 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 nausea and vomiting. Note: patients with gastric banding are allowed.
  • Pregnant individuals.
Terms in these criteria, in plain language(28)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Histologically or cytologically confirmed

The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).

Your pathology report is the document that shows this.

RECIST

The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.

“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.

Karnofsky performance status (KPS)

A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.

Studies often ask for 60 or 70 and above. Your doctor gives the score.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

INR, PT and aPTT (clotting tests)

Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.

Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Biopsy and archival tissue

A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.

Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.

CYP3A (CYP3A4) inhibitors and inducers

CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.

Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.

LVEF (left ventricular ejection fraction)

The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.

ECG (electrocardiogram)

A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.

QT and QTc

On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.

The study may name a correction formula and a limit in milliseconds, often around 450–480.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

Brain metastases

Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).

Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
116 participants (estimated)

Study arms

  • Experimental
    1/Arm 1

    Escalating/de-escalation doses of metarrestin

    Drug: Metarrestin

  • Experimental
    2/Arm 2

    MTD of metarrestin

    Drug: Metarrestin

Interventions

  • DrugMetarrestin

    Phase IA: Loading dose on Day 1 of Cycle 1 for Dose Levels 1-7. Loading dose on Days 1 and 3 of Cycle 1 for Dose Levels 8-11. After the loading dose on Day 1 or Days 1 and 3 of Cycle 1, continue on Mondays-Wednesdays-Fridays of every following cycle. Phase IB: PO according to the dose and schedule estimated during Phase IA

06

What researchers measure

Primary outcomes

  1. To identify the maximum tolerated dose (MTD) of metarrestin in subjects with metastatic solid tumors

    MTD of metarrestin

    Time frame: 28 days

  2. To determine the Objective Response Rate (ORR) according to Evaluation Criteria (RECIST 1.1) in patients treated with metarrestin at the MTD

    Fraction of objective responses determined and reported as the ORR

    Time frame: every 2 months

Secondary outcomes

  1. To determine the Objective Response Rate (ORR) according to Evaluation Criteria (RECIST 1.1) in patients treated with metarrestin at the MTD in Cohort IB1

    The ORR rate will be determined by dividing the number of patients with an objective response by the number of evaluable patients who are treated at the MTD for cohort Phase IB1

    Time frame: every 2 months

  2. To determine plasma PK levels of metarrestin in humans

    The pharmacokinetic (PK) profile of metarrestin will be derived from single dose and multiple dose PK measures for each dose level collecting the standard pharmacokinetic parameters

    Time frame: 28 days

  3. To assess progression-free survival (PFS) according to RECIST 1.1

    Median amount of time subject survives without disease progression after treatment

    Time frame: at progression

  4. To determine duration of overall response (DOR) rate according to Response Evaluation Criteria (RECIST 1.1) in subjects with metastatic solid tumors

    Kaplan-Meier curves beginning at the date of response will be used and will be determined in all responding patients treated on the Phase IB expansion cohorts

    Time frame: at progression

  5. To determine tolerability of the adult recommended dose in children >= 12 year of age (Cohort IB2)

    The fraction of patients who can tolerate the dose

    Time frame: 28 days after treatment discontinuation

  6. To assess safety and tolerability of metarrestin (Cohort IB1)

    All treatment-related AEs will be captured, and for each grade, the number and type of AE per dose level will be descriptively described in Cohort 1B1

    Time frame: 28 days after treatment discontinuation

  7. To assess safety and tolerability of metarrestin in subjects with metastatic solid tumors

    All treatment-related AEs will be captured, and for each grade, the number and type of AE per dose level will be descriptively described

    Time frame: 28 days after treatment discontinuation

07

Study locations

2 of 2 sites recruiting
  • University of Kansas
    Fairway, Kansas 66205, United States
    Recruiting
  • National Institutes of Health Clinical Center
    Bethesda, Maryland 20892, United States
    Recruiting
    • For more information at the NIH Clinical Center contact National Cancer Institute Referral Office · Contact888-624-1937
08

References and documents

Individual participant data

Plan to share: Yes — All IPD recorded in the medical record will be shared with intramural investigators upon request.

Supporting information: Study protocol, Statistical analysis plan, Informed consent form

Time frame: Clinical data available during the study and indefinitely.

Access criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.

09

Timeline

4 registry updates since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Oct 2020 (actual). Primary completion is estimated for Dec 2027, 86 months after the start.

Of 208 non-industry phase 1 interventional studies under Pancreatic Neoplasms, half reached primary completion within 46 months of starting (the median), counting those still running; a quarter within 33 and three quarters within 74. This study's planned 86 months is above that.

Counting every sponsor, half of 379 studies reached it within 45 months; a quarter within 32 and three quarters within 67.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 7, 2020
First posted
Jan 10, 2020
StartActual
Oct 27, 2020
Primary completionEst.
Dec 31, 2027
CompletionEst.
Dec 31, 2028
Last update
Nov 9, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Eligibility
1 inclusion criterion edited; 1 exclusion criterion edited, 1 added, 1 removed
What changed in the criteria
Inclusion criteria
  • EditedPediatric (>=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).
Exclusion criteria
  • AddedPatients receiving medications known to carry a high to moderate risk to lower seizure threshold within 2 weeks prior to treatment initiation.
  • RemovedSubjects 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).
  • EditedPatients with a history of any seizures within 1 year prior to the study treatment initiation 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. Note: Patients with brain metastasis are eligible providing that patients had no seizures or clinical signs of central nervous system (CNS) disease-related neurotoxicity (ataxia, sensory, cognitive changes) within 1 year prior to the study treatment initiation and baseline EEG shows no changes indicative of increased seizure risk.
Oct 30, 2026
Show all 4 updates
  1. Nov 9, 2026
    Minor edits only
    + 1 other change: description
  2. Nov 2, 2026
    Minor edits only
    + 1 other change: verification date
  3. Oct 30, 2026
    Eligibility 1 inclusion criterion edited; 1 exclusion criterion edited, 1 added, 1 removed
    What changed in the criteria
    Inclusion criteria
    • EditedPediatric (>=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).
    Exclusion criteria
    • AddedPatients receiving medications known to carry a high to moderate risk to lower seizure threshold within 2 weeks prior to treatment initiation.
    • RemovedSubjects 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).
    • EditedPatients with a history of any seizures within 1 year prior to the study treatment initiation 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. Note: Patients with brain metastasis are eligible providing that patients had no seizures or clinical signs of central nervous system (CNS) disease-related neurotoxicity (ataxia, sensory, cognitive changes) within 1 year prior to the study treatment initiation and baseline EEG shows no changes indicative of increased seizure risk.
  4. Oct 16, 2026
    Minor edits only
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT04222413
Responsible party
Sponsor
First posted
Jan 10, 2020
Start date
Oct 27, 2020
Primary completion
Dec 31, 2027 (estimated)
Completion
Dec 31, 2028 (estimated)
Last update
Nov 9, 2026

Study contacts

Murielle Hogu
Contact
murielle.hogu@nih.gov
(240) 858-3335
Udo Rudloff, M.D.
Contact
rudloffu@mail.nih.gov
(240) 760-6238
Udo Rudloff, M.D.
principal investigator · National Cancer Institute (NCI)

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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