Researchers are looking for new ways to treat children with relapsed or refractory solid tumors:
* Relapsed means the cancer came back after treatment
* Refractory means the cancer did not respond (get smaller or go away) to treatment
* Solid tumors are cancers mostly in body organs and tissues, not in the blood or other body liquids
The study treatment I-DXd (also known as MK-2400 or ifinatamab deruxtecan) is an antibody-drug conjugate (ADC). An ADC attaches to a protein on cancer cells and delivers treatment to destroy those cells. The goals of this study are to learn:
* About the safety of I-DXd and if children younger than 12 years old tolerate it
* How many children who receive I-DXd have the cancer get smaller or go away
Eligibility
Sex
ALL
Min age
1 Month
Max age
17 Years
Healthy volunteers
No
The main inclusion criteria include but are not limited to the following:
* In Part 1, participant has recurrent or relapsed, refractory solid tumors (excluding primary central nervous system (CNS)); and in Part 2, participant has recurrent or relapsed, refractory and histologically confirmed diagnosis of osteosarcoma (OST), neuroblastoma (NBL), rhabdomyosarcoma (RMS), or Wilms tumor (WT). All participants must meet the following criteria: Has documented radiological disease progression after at least 1 line of prior therapy in the locally advanced/metastatic setting and who has no satisfactory alternative treatment option (ie, is ineligible for other standard treatment regimens).
* Is an individual of any sex/gender, ≥1 month to \<12 years of age for Part 1 and ≥1 month to \<18 years for Part 2 at the time of providing the informed consent or assent, as applicable
* Participants who have AEs due to previous anticancer therapies must have recovered to ≤Grade 1 or baseline. Participants with endocrine-related AEs who are adequately treated with hormone replacement or participants who have ≤Grade 2 neuropathy are eligible.
The main exclusion criteria include but are not limited to the following:
* Has clinically significant corneal disease
* Has a history of cerebrovascular accident, transient ischemic attack, or another arterial thromboembolic event within 6 months before screening
* Has uncontrolled or significant cardiovascular disease, including conduction abnormalities, hypertension, ischemic heart disease, heart failure, and peripheral vascular disease
* Has any history of interstitial lung disease (ILD)/pneumonitis, irrespective of steroid use, except for a history of radiation pneumonitis that did not require steroids, current ILD, or Clinical or radiographic suspicion of ILD for which the diagnosis of ILD cannot be ruled out
* Has clinically severe respiratory compromise resulting from intercurrent pulmonary illnesses
* Has an active, known or suspected autoimmune disease.
* Has history of solid organ transplant.
* Has history of allogeneic stem cell transplant (SCT).
* Has known active CNS metastases and/or carcinomatous meningitis/leptomeningeal disease/spinal cord compression. Participants with untreated and asymptomatic brain metastases or previously treated brain metastases may participate provided they are radiologically stable, (i.e, without evidence of progression) for at least 4 weeks
* Has history of human immunodeficiency virus (HIV) infection.
* Has known additional malignancy that is progressing or has required active treatment within the past 1 year.
* Has active infection requiring systemic therapy
* Has known hypersensitivity or contraindication to either the study intervention substance or inactive ingredients in the study intervention product
* Participants who have not adequately recovered from major surgery or have ongoing surgical complications
Primary outcome measure(s)
Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience a Dose-limiting Toxicity (DLT) — Cycle 1 (up to approximately 21 days); each cycle is 21 days A DLT is any of a prespecified list of adverse events (AEs) that occur during Cycle 1 (up to 21 days) if attributed to the study treatment and not attributed to any other clearly identifiable cause. The percentage of participants who experience DLTs will be reported. Each cycle is 21 days.
Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience One or More Adverse Events (AEs) — Up to approximately 5 years An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who experience AEs will be reported.
Part 1: Number of Participants From ≥1 Month to <12 Years Who Discontinue Study Intervention Due to an AE — Up to approximately 5 years An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who discontinue study treatment due to an AE will be reported.
Part 1: Number of Participants From ≥1 Month to <12 Years Who Receive Dose Modifications Due to AEs — Up to approximately 5 years An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who receive dose modification due to an AE will be reported.
Part 1 and Part 2: Objective Response Rate (ORR) for Participants with neuroblastoma (NBL), rhabdomyosarcoma (RMS), and Wilms tumor (WT) — Up to approximately 5 years ORR is defined as the percentage of participants with Complete Response (CR: disappearance of all target lesions) or Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions). The percentage of participants who experience CR or PR as assessed by the investigator will be presented.
Part 1 and Part 2: Disease Control Success at 4 Months (DCS-4) for Participants with osteosarcoma (OST) — Up to 4 Months DCS-4 is defined as no occurrence of disease progression per disease specific criteria as assessed by investigator or death due to any cause by Month 4 following the first administration of study intervention for participants with OST. Participants who discontinue from study for any reason prior to completing the third post baseline (or at least 16 weeks) response assessments will be considered disease control failures.
Trial sites (26)
Facility
City
Region
Status
Children's Hospital Los Angeles ( Site 4006)
Los Angeles
California
Recruiting
Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 4016)
Aurora
Colorado
Recruiting
University of Iowa Hospitals ( Site 4017)
Iowa City
Iowa
Recruiting
Dana Farber Cancer Center ( Site 4013)
Boston
Massachusetts
Recruiting
Corewell Health ( Site 4001)
Grand Rapids
Michigan
Recruiting
Rutgers Cancer Institute of New Jersey ( Site 4008)
New Brunswick
New Jersey
Recruiting
Memorial Sloan Kettering Cancer Center ( Site 4010)
New York
New York
Recruiting
New York Medical College ( Site 4023)
Valhalla
New York
Recruiting
Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 4003)
Fargo
North Dakota
Recruiting
Children's Hospital of Philadelphia (CHOP) ( Site 4021)
Philadelphia
Pennsylvania
Recruiting
Sanford Children's Hospital ( Site 4015)
Sioux Falls
South Dakota
Recruiting
University of Texas M.D. Anderson Cancer Center ( Site 4007)
Houston
Texas
Recruiting
Intermountain - Primary Children's Hospital ( Site 4014)
Salt Lake City
Utah
Recruiting
UZ Gent ( Site 4428)
Ghent
Oost-Vlaanderen
Recruiting
Rigshospitalet ( Site 4467)
Copenhagen
Capital Region
Recruiting
Bordeaux University Hospital - Pellegrin ( Site 4105)
Bordeaux
Aquitaine
Recruiting
Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes ( Site 4104)
Nantes
Loire-Atlantique
Recruiting
CENTRE LEON BERARD ( Site 4100)
Lyon
Rhone
Recruiting
Rambam Health Care Campus ( Site 4674)
Haifa
Israel
Recruiting
Sheba Medical Center ( Site 4675)
Ramat Gan
Israel
Recruiting
Seoul National University Hospital-Pediatrics ( Site 4972)
Seoul
South Korea
Recruiting
Asan Medical Center-Pediatrics - Pedicatric Oncology ( Site 4973)
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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