ClinicalTrials.gg
RecruitingNCT04616560Updated Oct 28, 2026

Trastuzumab Deruxtecan (DS-8201a) for the Treatment of Recurrent or Refractory Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor

A Phase 2 interventional study of Biospecimen Collection and Computed Tomography in Desmoplastic Small Round Cell Tumor, Osteosarcoma and Recurrent Desmoplastic Small Round Cell Tumor, sponsored by National Cancer Institute (NCI). Recruiting at 32 sites in United States. Open to participants aged 12 Years to 39 Years. Per ClinicalTrials.gov, last updated 2026-10-28.

Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Mar 2021; still recruiting 5 years 8 months later.
Updated Oct 28, 2026Site recruiting status changed2 sites addedGo to Updates ↓
Phase
Phase 2
Study type
Interventional
Enrollment
55
Allocation
Not applicable
Ages
12 Years to 39 Years
Sex
All
01

Study summary

This phase I/II trial studies the effects of trastuzumab deruxtecan (DS-8201a) in treating patients with osteosarcoma, Wilms tumor (WT) or desmoplastic small round cell tumor (DSRCT) that is newly diagnosed or has come back after a period of improvement (recurrent) or that has not responded to previous treatment (refractory). Trastuzumab deruxtecan is in a class of medications called antibody-drug conjugates. It is composed of a monoclonal antibody, called trastuzumab, linked to a chemotherapy drug, called deruxtecan. Trastuzumab attaches to HER2 positive tumor cells in a targeted way and delivers deruxtecan to kill them.

Read the detailed description

PRIMARY OBJECTIVES:

I. To estimate the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of DS-8201a in children at least 2 to less than 12 years old with recurrent or refractory WT or DSRCT. (Phase 1) II. To define and describe the toxicities of DS-8201a in children at least 2 to less than 12 years old with recurrent or refractory WT or DSRCT. (Phase 1) III. To estimate the antitumor activity of DS-8201a in pediatric patients and young adults with recurrent or refractory WT. (Phase 2) IV. To estimate the antitumor activity of DS-8201a in pediatric patients and young adults with recurrent or refractory DSRCT. (Phase 2)

SECONDARY OBJECTIVES:

I. To assess the safety of DS-8201a in patients at least 12 years old with recurrent or refractory WT or DSRCT.

II. To describe the pharmacokinetics of DS-8201a in pediatric patients and young adults with WT or DSRCT.

EXPLORATORY OBJECTIVES:

I. To evaluate whether HER2 expression as measured by immunohistochemistry (IHC) is predictive of response to DS-8201a in pediatric and young adult patients with WT or DSRCT.

II. To evaluate quantitative circulating tumor deoxyribonucleic acid (DNA) (ctDNA) as surrogate markers of response in WT or DSRCT.

III. To estimate the objective response rate (ORR) to DS-8201a by blinded independent central review in pediatric and young adult patients with WT or DSRCT.

IV. To estimate the disease control rate (DCR) for DS-8201 as the proportion of response evaluable patients who achieve complete response (CR), partial response (PR), or stable disease (SD) during study treatment.

V. To estimate the overall survival (OS) for DS-8201 as time from study enrollment to death due to any cause.

VI. To estimate the progression free survival (PFS) defined as the time from study enrollment to progression or death, which ever comes first.

OUTLINE:

Patients receive trastuzumab deruxtecan intravenously (IV) over 30-90 minutes on day 1. Treatment repeats every 21 days for 35 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo computed tomography (CT) and magnetic resonance imaging (MRI), echocardiography or multigated acquisition scan (MUGA), blood sample collection throughout the study.

After completion of study treatment, patients are followed up at 6 months.

02

Conditions studied

  • Desmoplastic Small Round Cell Tumor
  • Osteosarcoma
  • Recurrent Desmoplastic Small Round Cell Tumor
  • Recurrent Kidney Wilms Tumor
  • Recurrent Osteosarcoma
  • Refractory Desmoplastic Small Round Cell Tumor
  • Refractory Wilms Tumor
  • Wilms Tumor
03

In context

Desmoplastic Small Round Cell Tumor

59 studies on the registry are indexed under Desmoplastic Small Round Cell Tumor; 14 are open to participants now.

This study's planned enrollment of 55 is above the median of 44 across 47 interventional studies indexed under Desmoplastic Small Round Cell Tumor.

Browse Desmoplastic Small Round Cell Tumor 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

Ages eligible
12 Years to 39 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

  • Phase 1 (Part A): Patients must be at least 2 years and less than 12 years of age at the time of study enrollment
  • Phase 2: Wilms tumor patients (Part B1): All Wilms tumor patients enrolled must be less than 18 years of age at enrollment

    • Until the completion of the Phase 1 dose confirmation, patients must be at least 12 years of age and less than 18 years of age at the time of study enrollment
    • Following dose confirmation of DS-8201a in children at least 2 to less than 12 years old in the Phase 1 component, Wilms tumor patients at least 2 to less than 18 years of age will be allowed on the Phase 2 component
  • Phase 2: DSRCT patients (Part B2): Until the completion of the Phase 1 component, patients enrolling on the Phase 2 component of the study must be from at least 12 to 39 years of age at the time of study enrollment

    • Following dose confirmation of DS-8201a in children at least 2 to less than 12 years old in the Phase 1 component, DSRCT patients at least 2 to 39 years of age will be allowed on the Phase 2 component
  • Patients must have had histologic verification of Wilms tumor or desmoplastic small round cell tumor at original diagnosis or relapse
  • Solid tumors: Patients must have according to (RECIST) 1.1. Patients with clinically inactive may be included in the study. Patients with treated brain metastases that are no longer symptomatic and who require no treatment with or anticonvulsants may be included in the study if they have recovered from the acute toxic effect of radiotherapy. Lastly, patient must have unresectable lesions or lesions with no intention to surgically remove the lesions in the 6 months following enrollment
  • Wilms tumor: WT patients must have either disease or a very high risk relapse, defined as ANY of the following:

    • Relapse after initial treatment with 4 or more chemotherapy agents (e.g. Regimens vincristine, dactinomycin, doxorubicin, cyclophosphamide, etoposide and radiation [M], vincristine, dactinomycin and doxorubicin, vincristine, and irinotecan [MVI], doxorubicin, vincristine, cyclophosphamide, carboplatin, etoposide and radiation [UH-1], doxorubicin, vincristine, cyclophosphamide, carboplatin, etoposide, vincristine, and irinotecan [UH-2], vincristine, irinotecan, cyclophosphamide, carboplatin, etoposide and doxorubicin [UH-3], and etoposide, carboplatin, cyclophosphamide, and doxorubicin [HR-1])
    • Relapse with high risk histology (anaplasia, blastemal predominant)
    • Multiple relapses
  • Desmoplastic small round cell tumor: DSRCT patients with relapsed or refractory disease are eligible
  • Patient's current disease state must be one for which they have received at least standard initial therapy, defined as systemic therapy combined with either radiation or surgery for local control of the primary tumor at diagnosis. Prior therapy after relapse is not required
  • Patients must have a performance status corresponding to (ECOG) scores of 0 or 1. Use for patients older than 16 years of age and Lansky for patients 16 years of age and younger
  • Patients must have recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately

    • Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive: For agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned Research Coordinator prior to enrollment

      • >= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)
    • Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced or [ANC] counts): >= 7 days after the last dose of agent
    • Antibodies: >= 4 weeks (28 days) must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1
    • Corticosteroids
    • Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
    • Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
    • Stem cell Infusions (with or without total body irradiation [TBI]):

      • Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of (GVHD)
      • Autologous stem cell infusion including boost infusion: >= 30 days
    • Cellular therapy: >= 30 days after the completion the infusion of any type of cellular therapy (e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.)
    • Radiation therapy (XRT)/external beam irradiation including protons: >= 4 weeks (28 days) including palliative radiation therapy to the chest. >= 14 days after palliative local XRT to areas other than the chest or for whole brain radiotherapy
    • Radiopharmaceutical therapy (e.g., radiolabeled antibody, samarium): >= 42 days after systemically administered radiopharmaceutical therapy
    • Patients must not have received prior therapies including antibody drug conjugates (e.g. TDM-1 or DS-8201a), HER2 directed cellular therapies, HER2 receptor therapy (e.g. trastuzumab, pertuzumab, margetuximab, zanidatamab, zenocutuzumab) or small molecule antagonists of HER2 (e.g lapatinib, tucatinib, or neratinib). Prior exposure to antibody drug conjugates which do not target HER2 as well as prior treatment with topoisomerase 1 inhibitors (e.g. irinotecan, topotecan) are permitted
  • Patients must be at least 14 days from the date of last surgery
  • Peripheral absolute neutrophil count (ANC) >= 1000/uL, (for patients with solid tumors without known bone marrow involvement)
  • Platelet count >= 100,000/uL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) (for patients with solid tumors without known bone marrow involvement)
  • >= 8.0 g/dL at baseline (Red Blood Cell transfusion is not allowed within 1 week prior to enrollment) (for patients with solid tumors without known bone marrow involvement)
  • For patients less than or equal to 17 years old, "Bedside" Schwartz formula (2009)
  • Estimated (GFR) (eGFR) ≥ 70 mL/min/1.73 m\^2 (> 70mL/min/1.73 m\^2 for patients > 17 years old)

    • For patients older than 17 years of age the Cockroft-Gault equation should be utilized to calculate ≥ 70 ml/min:
    • OR for any age group:

      • A 24 hour urine creatinine clearance ≥ 70 mL/min/1.73 m\^2 (> 70mL/min for patients ≥ 17 years old) OR
      • A directly measured GFR ≥ 70 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
  • (sum of conjugated + unconjugated or total) =\< 1.5 x (ULN) for age. For patients with documented Gilbert's syndrome (unconjugated hyperbilirubinemia) bilirubin must be \< 3 x ULN for age (patients with solid tumors)
  • (AST) =\< 3 x ULN
  • Serum albumin >= 2.5 g/dL (patients with solid tumors)
  • (INR)/prothrombin time (PT) =\< 1.5 x ULN. Exception for patients receiving coumarin-derivative anticoagulants or other similar anticoagulant therapy, who must have INR/PT within the therapeutic range as deemed appropriate by the investigator
  • Shortening fraction of >= 27% by echocardiogram, or of >= 50% by either an echocardiogram (ECHO) or multigated acquisition (MUGA) scan within 28 days before Step 1 enrollment
  • interval (QTc) prolongation to \< 480 ms based on average triplicate 12-lead (ECG)
  • Pulse oximetry > 93% on room air
  • Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled as evidenced by no increase in seizure frequency in the prior 7 days
  • Nervous system disorders ( [CTCAE] version [v]5) resulting from prior chemotherapy, surgery, and/or radiation must be =\< grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible
  • -infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months of enrollment are eligible
  • All patients and/or their parents or legally authorized representatives must sign a written . Assent, when appropriate, will be obtained according to institutional guidelines

Exclusion criteria

Exclusion Criteria:

  • Pregnant, planning to become pregnant, or breast-feeding women will not be entered on this study. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of may not participate unless they have agreed to use two effective methods of , including a medically accepted barrier or contraceptive method (e.g., male or female condom) for the duration of the study and upon completion of the study and for at least 7 months for females and 4 months for males after the last dose of study drug. Abstinence is an acceptable method of birth control

    • Methods considered as highly effective methods of contraception include:

      • Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:

        • Oral
        • Intravaginal
        • Transdermal
      • Progestogen-only hormonal contraception associated with inhibition of ovulation:

        • Oral
        • Injectable
        • Implantable
      • Intrauterine device (IUD)
      • Intrauterine hormone-releasing system (IUS)
      • Bilateral tubal occlusion
      • Vasectomized partner
      • Complete sexual abstinence defined as refraining from heterosexual intercourse. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not an acceptable method of contraception
  • Non- defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or defined as 12 months of spontaneous amenorrhea (in questionable cases, a blood sample with simultaneous follicle-stimulating hormone [FSH] > 40 mIU/mL and estradiol \< 40 pg/mL [\< 147 pmol/L] is confirmatory). Females on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use one of the contraception methods outlined for women of child-bearing potential if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of post-menopausal status prior to study enrollment. For most forms of HRT, at least 2-4 weeks will elapse between the cessation of therapy and the blood draw; this interval depends on the type and dosage of HRT. Following confirmation of their post-menopausal status, they can resume use of HRT during the study without use of a contraceptive method
  • Male patients must not freeze or donate sperm starting at enrollment and throughout the study period, and at least 4 months after the final study drug administration. Preservation of sperm should be considered prior to enrolment in this study
  • Female patients must not donate, or retrieve for their own use, ova from the time of enrollment and throughout the study treatment period, and for at least 7 months after the final study drug administration
  • Patients receiving who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
  • Patients who are currently receiving another drug are not eligible
  • Patients who are currently receiving other anti-cancer agents are not eligible
  • Patients who are receiving cyclosporine, tacrolimus or other agents to prevent post bone marrow transplant are not eligible for this trial
  • Patients who are receiving chloroquine or hydroxychloroquine within 14 days are not eligible for this trial
  • Patients who received a live, attenuated vaccine (messenger ribonucleic acid [mRNA] and replication deficient adenoviral vaccines are not considered attenuated ) within 30 days prior to enrollment are not eligible for this trial

    • Note: Participants, if enrolled, should not receive live vaccines during the study and up to 90 days after the last dose of study intervention. It is recommended that patients receive a yearly influenza killed vaccination and additional killed vaccinations based on local or national recommendations. Consider vaccination against viral pathogens that cause pneumonias according to local or national guidelines
  • Patients who have received a prior solid organ transplantation are not eligible
  • Patients with a medical history of myocardial infarction within 180 days before enrollment, symptomatic congestive heart failure (CHF) ( Class II to IV) or troponin levels consistent with myocardial infarction as defined according to the manufacturer 28 days prior to enrollment are not eligible
  • Additionally, patients with a history of any of the following congenital heart disease are not eligible:

    • Single ventricle heart defects (hypoplastic left heart syndrome, unbalanced atrioventricular septal defects, double inlet left ventricle, tricuspid atresia, the presence of superior cavopulmonary anastomosis or Fontan palliation);
    • Unpalliated defects with significant hemodynamic alterations or palliated lesions with residual hemodynamic alterations (ductal-dependent or shunt-dependent physiology, large unrestrictive ventricular septal defect, transposition of the great arteries, greater than moderate atrioventricular valve insufficiency, moderate or greater aortic valve stenosis, moderate or greater aortic valve insufficiency, large atrial septal defects with significant right ventricular volume overload, large patent ductus arteriosus)
  • Patients who have a pleural effusion, ascites or pericardial effusion that requires drainage, peritoneal shunt, or cell-free and concentrated ascites reinfusion therapy (CART) are not eligible. (Drainage and concentrated ascites reinfusion therapy are not allowed within 2 weeks prior to enrollment)
  • Patients who have spinal cord compression or clinically active central nervous system , defined as untreated and symptomatic, or requiring therapy with corticosteroids or anticonvulsants to control associated symptoms are not eligible
  • Patients with a known history of severe to DS-8201a, any contained in the DS-8201a drug formulation, or -targeted monoclonal antibodies (trastuzumab, pertuzumab, margetuximab) are not eligible
  • Patients who have an uncontrolled infection or non-healing surgical site are not eligible
  • Patients with a known history of substance abuse or any other medical conditions (i.e. psychological conditions) that may, in the opinion of the investigator, interfere with the patient's participation in the clinical study or evaluation of the clinical study results are not eligible
  • Patients who have pulmonary compromise, ex hypoxia, resulting from intercurrent pulmonary illnesses including, but not limited to, any underlying pulmonary disorder (i.e. pulmonary emboli within three months of the study enrollment, severe asthma, severe (COPD), restrictive lung disease, pleural effusion etc.), or prior pneumonectomy are not eligible
  • Patients who have a history of (non-infectious) ILD (interstitial lung disease)/pneumonitis that required steroids, has current ILD/pneumonitis, or for whom suspected ILD/pneumonitis cannot be ruled out by imaging at screening are not eligible. Patients who have history of genetic disorders of the lung (i.e. cystic fibrosis are not eligible)
  • Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study are not eligible
  • Patients with known or C with detectable viral load are not eligible
  • Patients with any , connective tissue or inflammatory disorders (e.g., rheumatoid arthritis, Sjogren's, sarcoidosis etc.) where there is documented, or a suspicion of pulmonary involvement at the time of enrollment or genetic diseases involving the lung are not eligible
  • Patients with an active primary immunodeficiency are not eligible
Terms in these criteria, in plain language(38)

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.

Measurable disease

The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.

The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.

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.

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.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

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.

ECOG performance status

A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.

0
Fully active, no restrictions.
1
Limited in strenuous activity, but walking and able to do light work, such as housework or office work.
2
Walking and able to look after yourself, but unable to work; up and about more than half of waking hours.
3
Able to look after yourself only in part; in bed or a chair more than half of waking hours.
4
Unable to look after yourself at all; in bed or a chair all day.

Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.

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.

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.

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.

GVHD (graft-versus-host disease)

After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.

HER2

HER2 is a protein, made from the ERBB2 gene, that drives the growth of some cancers. A study may look for a level of the protein, extra copies of the gene, or a particular change in it.

In breast cancer, protein tests are read as HER2-positive, HER2-low or HER2-negative; other cancers use their own rules. Check which test result the study requires.

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).

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.

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.

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.

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.

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.

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.

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.

CTCAE grade

The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.

1
Mild.
2
Moderate.
3
Severe.
4
Life-threatening.
5
Death due to the event.

“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.

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.

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.

Reproductive potential

Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.

Criteria about it usually ask 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.

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.

Postmenopausal

Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).

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.

Live vaccines

Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.

NYHA class (heart failure)

The New York Heart Association’s four classes of how much heart failure limits physical activity.

I
No limits: ordinary activity causes no undue tiredness or breathlessness.
II
Slight limits: comfortable at rest, ordinary activity causes symptoms.
III
Marked limits: comfortable at rest, less than ordinary activity causes symptoms.
IV
Symptoms even at rest; any activity makes them worse.

Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.

Metastases

Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Excipients

The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.

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.

COPD

Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.

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.

Autoimmune disease

A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.

Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
55 participants (estimated)

Study arms

  • Experimental
    Treatment (trastuzumab deruxtecan)

    Patients receive trastuzumab deruxtecan IV over 30-90 minutes on day 1. Treatment repeats every 21 days for 35 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo CT and MRI, echocardiography or MUGA, blood sample collection throughout the study.

    Procedure: Biospecimen Collection · Procedure: Computed Tomography · Procedure: Echocardiography Test · Procedure: Magnetic Resonance Imaging · Procedure: Multigated Acquisition Scan · Biological: Trastuzumab Deruxtecan

Interventions

  • ProcedureBiospecimen Collection

    Undergo blood sample collection

    Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

  • ProcedureComputed Tomography

    Undergo CT

    Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography

  • ProcedureEchocardiography Test

    Undergo echocardiography

    Also known as: EC, Echocardiography

  • ProcedureMagnetic Resonance Imaging

    Undergo MRI

    Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI

  • ProcedureMultigated Acquisition Scan

    Undergo MUGA

    Also known as: Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool Scan, MUGA, MUGA Scan, Multi-Gated Acquisition Scan, Radionuclide Ventriculogram Scan, Radionuclide Ventriculography, RNV Scan, RNVG, SYMA Scanning, Synchronized Multigated Acquisition Scanning

  • Given IV

    Also known as: DS-8201, DS-8201a, Enhertu, Fam-trastuzumab Deruxtecan-nxki, T-DXd, WHO 10516

06

What researchers measure

Primary outcomes

  1. Maximum tolerated dose and/or recommended phase 2 dose of DS-8201a in children at least 2 to less than 12 years old with recurrent or refractory Wilms tumor (WT) or desmoplastic small round cell tumor (DSRCT) (Phase 1)

    The recommended dose will be determined by evaluation of safety, tolerability, and available pharmacokinetic and response data.

    Time frame: During the first cycle of therapy (cycle length = 21 days)

  2. Incidence of adverse events (AEs) in children at least 2 to less than 12 years old with recurrent or refractory WT or DSRCT (Phase 1)

    Toxicity tables will be constructed to summarize the observed incidence by type of toxicity and grade. A patient will be counted only once for a given toxicity for the highest grade of that toxicity reported for that patient. Toxicity information recorded will include the type, severity, time of onset, time of resolution, and the probable association with the study regimen.

    Time frame: Up to 30 days after last dose of study treatment

  3. Time to progression

    Median (95% confidence interval \[CI\]) time-to-progression estimated by Kaplan-Meier curves by dose level.

    Time frame: Up to 1 year

Secondary outcomes

  1. Incidence of AEs of DS-8201A in children at least 12 years old with recurrent or refractory WT or DSRCT

    Toxicity tables will be constructed to summarize the observed incidence by type of toxicity and grade. A patient will be counted only once for a given toxicity for the highest grade of that toxicity reported for that patient. Toxicity information recorded will include the type, severity, time of onset, time of resolution, and the probable association with the study regimen.

    Time frame: Up to 6 months

  2. Response rates

    Will be estimated by the uniform minimum variance unbiased estimator (Jung \& Kim, 2004) with one-sided 97% CI (Koyama and Chen, 2008).

    Time frame: Up to 1 year

  3. Duration of response

    The duration of overall response is measured from the time measurement criteria are met for complete response or partial response (whichever is first recorded) until the first date that recurrent or progressive disease is objectively documented (taking as reference for progressive disease the smallest measurements recorded since the treatment started).

    Time frame: Up to 1 year

Other outcomes

  1. Progression-free survival (Phase 2)

    Frequency (%) of evaluable patients who are event-free after 24 weeks (4-months) of therapy.

    Time frame: From study enrollment until the first occurrence of an event, defined as relapse, disease progression or death from any cause, assessed up to 1 year

  2. Overall survival of DS-8201A

    Median (95% CI) time-to-death estimated by Kaplan-Meier curves by dose level.

    Time frame: Up to 1 year

  3. Disease control rate

    Will be estimated by the proportion of response-evaluable patients with complete response (CR), partial response (PR), or 24-weeks stable disease with exact, 95% confidence interval. Will be estimated overall and stratified by disease group.

    Time frame: Up to 1 year

  4. Objective response rate

    Will be estimated separately for each Part B cohort. Will be estimated by the proportion with response (CR+PR) with a 95% exact Binomial confidence interval. Estimates will also be stratified by disease cohort.

    Time frame: Up to 1 year

07

Study locations

21 of 32 sites recruiting
08

References and documents

Publications

  • Reed DR, Janeway KA, Minard CG, Hall D, Crompton BD, Lazar AJ, Wang WL, Zylber R, Contreras A, Carrero G, Voss SD, Reid JM, Safgren SL, Militano O, Gorlick R, Pickett CA, Fox E, Weigel BJ. PEPN1924, A Phase II Study of Trastuzumab Deruxtecan in Patients With Recurrent Human Epidermal Growth Factor Receptor 2+ Osteosarcoma: A Children's Oncology Group Pediatric Early-Phase Clinical Trial Network Study. JCO Oncol Adv. 2025;2:e2500094. doi: 10.1200/oa-25-00094. Epub 2025 Oct 14. PubMed 42131734 ↗

Individual participant data

Plan to share: Yes — NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

Data sharing details ↗ · grants.nih.gov

09

Timeline

3 registry updates since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Mar 2021 (actual). Primary completion is estimated for Dec 2027, 81 months after the start.

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

Registered
Nov 4, 2020
First posted
Nov 5, 2020
StartActual
Mar 8, 2021
Primary completionEst.
Dec 31, 2027
CompletionEst.
Dec 31, 2027
Last update
Oct 28, 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.
Sites
2 sites added. Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center is now Recruiting
Show 2 added (2 United States)
  • New York Medical College · Valhalla, United States
  • Seattle Children's Hospital · Seattle, United States
across 3 updates, Sep 29, 2026 – Oct 28, 2026
Site contacts
Changed at 1 site. See the sites
Show the site
  • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center · Houston, United StatesContact added b•••@bcm.edu · •••1354Contact added (no phone or email listed)
Oct 28, 2026
Show all 3 updates
  1. Oct 28, 2026
    Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center is now Recruiting
    Site contacts Changed at 1 site. See the sites
    Show the site
    • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center · Houston, United StatesContact added b•••@bcm.edu · •••1354Contact added (no phone or email listed)
  2. Oct 13, 2026
    1 site added
    Show site
    • Seattle Children's Hospital · Seattle, United States
  3. Sep 29, 2026
    1 site added
    Show site
    • New York Medical College · Valhalla, United States

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
NCT04616560
Responsible party
Sponsor
First posted
Nov 5, 2020
Start date
Mar 8, 2021
Primary completion
Dec 31, 2027 (estimated)
Completion
Dec 31, 2027 (estimated)
Last update
Oct 28, 2026

Study contacts

Damon R Reed
principal investigator · Pediatric Early Phase Clinical Trial Network

Oversight

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

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Studies of the drug tested here: Trastuzumab deruxtecan

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