Recurrent Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
Conditions
Interventions
Trade Name: Enhertu
Product Name: Trastuzumab deruxtecan
Product Code: DS-8201a
Pharmaceutical Form: Concentrate for solution for injection
Sponsors
National Institutes of Health, National Cancer Institute, Division of Cancer Treatment and Diagnosis
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? yes Number of subjects for this age range: 40 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: 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. ;Primary 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.;Timepoint(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 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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. ;Timepoint(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 | — |
Countries
Australia, Canada, United States
Contacts
Public ContactClinical Trial Information
Daiichi Sankyo, Inc.
Outcome results
None listed