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    Summary
    EudraCT Number:2025-000543-40
    Sponsor's Protocol Code Number:PEPN1924
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2026-06-05
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2025-000543-40
    A.3Full title of the trial
    A Phase 1/2 Study of DS-8201a (NSC# 807708, IND# 153036) in Children, Adolescents, or Young Adults With Recurrent Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of DS-8201a in Children, Adolescents, or Young Adults With recurrent Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
    A.4.1Sponsor's protocol code numberPEPN1924
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/397/2024
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNational Institutes of Health, National Cancer Institute, Division of Cancer Treatment and Diagnosis
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNational Institutes of Health, National Cancer Institute, Division of Cancer Treatment and Diagnosis
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDaiichi Sankyo, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address211 Mt. Airy Rd.
    B.5.3.2Town/ cityBasking Ridge
    B.5.3.3Post code07920
    B.5.3.4CountryUnited States
    B.5.4Telephone number+19089926400
    B.5.6E-mailCTRinfo_us@daiichisankyo.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Enhertu
    D.2.1.1.2Name of the Marketing Authorisation holderDaiichi Sankyo, Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTrastuzumab deruxtecan
    D.3.2Product code DS-8201a
    D.3.4Pharmaceutical form Concentrate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Recurrent Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
    E.1.1.1Medical condition in easily understood language
    Recurrent Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    In Phase 1, the primary objectives are to estimate the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of DS-8201a and to define and describe the toxicities of DS-8201a in children at least 2 to less than 6 years old and at least 6 to less than 12 years old with recurrent or refractory WT or DSRCT.

    In Phase 2, the primary objectives are to estimate the antitumor activity of DS-8201a in pediatric patients and young adults with recurrent or refractory WT and to estimate the antitumor activity of DS-8201a in pediatric patients and young adults with recurrent or refractory DSRCT.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to assess the safety of DS-8201a in patients at least 12 years old with recurrent or refractory WT, or DSRCT and to describe the pharmacokinetics of DS-8201a in pediatric patients and young adults with WT or DSRCT.
    E.2.3Trial contains a sub-study No
    E.3Principal 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 (Part B1): All WT patients enrolled must be less than 18 years of age at enrollment.
    -Phase 2 (Part B2): Until the completion of the Phase 1 component, all DSRCT 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.
    -Patients must have had histologic verification of WT or DSRCT at original diagnosis or relapse.
    -Patients must have measurable disease according to RECIST 1.1.
    -WT patients must have either refractory disease or a very high-risk relapse.
    -DSRCT patients with relapsed or refractory disease are eligible.
    -Performance status corresponding to ECOG scores of 0 or 1.
    -Adequate bone marrow, liver, cardiac, renal, pulmonary, and neurologic function.
    -Written, informed consent.
    E.4Principal exclusion criteria
    -Women who are pregnant, planning to become pregnant, or breast-feeding.
    -Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment.
    -Patients who are currently receiving another investigational drug.
    -Patients who are currently receiving other anti-cancer agents.
    -Patients who have received a prior solid organ transplantation.
    -Patients with a medical history of myocardial infarction within 180 days before enrollment, symptomatic congestive heart failure (CHF) (New York Heart Association Class II to IV) or troponin levels consistent with myocardial infarction as defined according to the manufacturer 28 days prior to enrollment.
    -Patients who have spinal cord compression or clinically active central nervous system metastases, defined as untreated and symptomatic, or requiring therapy with corticosteroids or anticonvulsants to control associated symptoms.
    -Patients with a known history of severe hypersensitivity to DS-8201a, any excipient contained in the DS-8201a drug formulation, or HER2-targeted monoclonal antibodies (trastuzumab, pertuzumab, margetuximab).
    -Patients who have pulmonary compromise resulting from intercurrent pulmonary illnesses.
    -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.
    E.5 End points
    E.5.1Primary end point(s)
    Co-primary endpoint 1: Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of DS-8201a in children at least 2 to less than 6 years old and at least 6 to less than 12 years old with recurrent or refractory WT or DSRCT

    Co-primary endpoint 2: Toxicities of DS-8201a in children at least 2 to less than 6 years old and at least 6 to less than 12 years old with recurrent or refractory WT or DSRCT.

    Co-primary endpoint 3: Antitumor activity of DS-8201a in pediatric patients and young adults with recurrent or refractory WT.

    Co-primary endpoint 4: Antitumor activity of DS-8201a in pediatric patients and young adults with recurrent or refractory DSRCT.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Co-primary endpoint 1: Day 1 (post dose) to Day 21 (Cycle 1)
    Co-primary endpoint 2: Baseline (Day 0) up to 13 months
    Co-primary endpoint 3: Baseline (Day 0) up to 13 months
    Co-primary endpoint 4: Baseline (Day 0) up to 13 months
    E.5.2Secondary end point(s)
    Co-secondary endpoint 1: Safety of DS-8201a in patients at least 12 years old with recurrent or refractory WT, or DSRCT.

    Co-secondary endpoint 2: Pharmacokinetics of DS-8201a in pediatric patients and young adults with WT or DSRCT.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Co-secondary endpoint 1: Baseline (Day 0) up to 13 months
    Co-secondary endpoint 2: Baseline (Day 0) up to 13 months
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Dose confirmation study
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Australia
    Canada
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 40
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects under-age incapable of giving consent personally
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 55
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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