A Study of Ifinatamab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01D/LIGHTBEAM-U01)
15. september 2026 oppdatert av: Merck Sharp & Dohme LLC
LIGHTBEAM-U01 Substudy 01D: A Phase 1b/2 Substudy to Evaluate the Safety and Efficacy of Ifinatamab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors
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
Studieoversikt
Status
Rekruttering
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
This study will have 2 parts: Part 1 will evaluate the safety and tolerability and determine the recommended dose for expansion (RDE) of I-DXd, followed by Part 2 an efficacy expansion.
Studietype
Intervensjonell
Registrering (Antatt)
134
Fase
- Fase 2
- Fase 1
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: Toll Free Number
- Telefonnummer: 1-888-577-8839
- E-post: Trialsites@msd.com
Studiesteder
-
-
Oost-Vlaanderen
-
Ghent, Oost-Vlaanderen, Belgia, 9000
- Rekruttering
- UZ Gent ( Site 4428)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +32 9 332 16 53
-
-
-
-
Capital Region
-
Copenhagen, Capital Region, Danmark, DK-2100
- Rekruttering
- Rigshospitalet ( Site 4467)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +4535455002
-
-
-
-
California
-
Los Angeles, California, Forente stater, 90027
- Rekruttering
- Children's Hospital Los Angeles ( Site 4006)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 323-361-2121
-
-
Colorado
-
Aurora, Colorado, Forente stater, 80045
- Rekruttering
- Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 4016)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 720-777-6740
-
-
Iowa
-
Iowa City, Iowa, Forente stater, 52242
- Rekruttering
- University of Iowa Hospitals ( Site 4017)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 319-356-2296
-
-
Massachusetts
-
Boston, Massachusetts, Forente stater, 02215
- Rekruttering
- Dana Farber Cancer Center ( Site 4013)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 617-632-4580
-
-
Michigan
-
Grand Rapids, Michigan, Forente stater, 49503
- Rekruttering
- Corewell Health ( Site 4001)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 616-486-0746
-
-
New Jersey
-
New Brunswick, New Jersey, Forente stater, 08901
- Rekruttering
- Rutgers Cancer Institute of New Jersey ( Site 4008)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 732-235-2465
-
-
New York
-
New York, New York, Forente stater, 10065
- Rekruttering
- Memorial Sloan Kettering Cancer Center ( Site 4010)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 888-492-8401
-
Valhalla, New York, Forente stater, 10595
- Rekruttering
- New York Medical College ( Site 4023)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 914-614-4270
-
-
North Dakota
-
Fargo, North Dakota, Forente stater, 58102
- Rekruttering
- Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 4003)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 701-234-2000
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, Forente stater, 19104
- Rekruttering
- Children's Hospital of Philadelphia (CHOP) ( Site 4021)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 267-425-5544
-
-
South Dakota
-
Sioux Falls, South Dakota, Forente stater, 57105
- Rekruttering
- Sanford Children's Hospital ( Site 4015)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 605-312-1000
-
-
Texas
-
Houston, Texas, Forente stater, 77030
- Rekruttering
- University of Texas M.D. Anderson Cancer Center ( Site 4007)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 713-792-5410
-
-
Utah
-
Salt Lake City, Utah, Forente stater, 84113
- Rekruttering
- Intermountain - Primary Children's Hospital ( Site 4014)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 801-662-4700
-
-
-
-
Aquitaine
-
Bordeaux, Aquitaine, Frankrike, 33076
- Rekruttering
- Bordeaux University Hospital - Pellegrin ( Site 4105)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +33 5 56 79 56 79
-
-
Loire-Atlantique
-
Nantes, Loire-Atlantique, Frankrike, 44093
- Rekruttering
- Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes ( Site 4104)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +33240087805
-
-
Rhone
-
Lyon, Rhone, Frankrike, 69373
- Rekruttering
- CENTRE LEON BERARD ( Site 4100)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +33469166572
-
-
-
-
-
Haifa, Israel, 3109601
- Rekruttering
- Rambam Health Care Campus ( Site 4674)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +97247776755
-
Ramat Gan, Israel, 5265601
- Rekruttering
- Sheba Medical Center ( Site 4675)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +97235302996
-
-
-
-
-
Barcelona, Spania, 08035
- Rekruttering
- Hospital Universitari Vall d Hebron ( Site 4716)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +34934893093
-
-
Barcelona
-
Esplugues de Llobregat, Barcelona, Spania, 8950
- Rekruttering
- Hospital Sant Joan de Déu ( Site 4717)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +34671600093
-
-
Madrid, Comunidad de
-
Madrid, Madrid, Comunidad de, Spania, 28009
- Rekruttering
- Hospital Niño Jesús ( Site 4715)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +34915035900
-
-
-
-
Västra Götaland County
-
Gothenburg, Västra Götaland County, Sverige, 416 85
- Rekruttering
- Sahlgrenska Universitetssjukhuset ( Site 4634)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: +46313435865
-
-
-
-
-
Seoul, Sør -Korea, 03080
- Rekruttering
- Seoul National University Hospital-Pediatrics ( Site 4972)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 82220723304
-
Seoul, Sør -Korea, 05505
- Rekruttering
- Asan Medical Center-Pediatrics - Pedicatric Oncology ( Site 4973)
-
Ta kontakt med:
- Study Coordinator
- Telefonnummer: 82230105994
-
-
Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
Tar imot friske frivillige
Nei
Beskrivelse
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
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: Ifinatamab Deruxtecan
Participants receive ifinatamab deruxtecan via intravenous (IV) infusion on day 1 of each 3-week cycle until discontinuation or progression
|
IV infusion
Andre navn:
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience a Dose-limiting Toxicity (DLT)
Tidsramme: 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.
|
Cycle 1 (up to approximately 21 days); each cycle is 21 days
|
|
Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience One or More Adverse Events (AEs)
Tidsramme: 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.
|
Up to approximately 5 years
|
|
Part 1: Number of Participants From ≥1 Month to <12 Years Who Discontinue Study Intervention Due to an AE
Tidsramme: 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.
|
Up to approximately 5 years
|
|
Part 1: Number of Participants From ≥1 Month to <12 Years Who Receive Dose Modifications Due to AEs
Tidsramme: 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.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Objective Response Rate (ORR) for Participants with neuroblastoma (NBL), rhabdomyosarcoma (RMS), and Wilms tumor (WT)
Tidsramme: 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.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Disease Control Success at 4 Months (DCS-4) for Participants with osteosarcoma (OST)
Tidsramme: 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.
|
Up to 4 Months
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Del 1 og del 2: Total Survival (OS)
Tidsramme: Opptil omtrent 5 år
|
OS er definert som tid fra første dose studiebehandling til død på grunn av enhver årsak.
|
Opptil omtrent 5 år
|
|
Part 1 and Part 2: Duration of Response (DOR) For Participants With NBL, RMS, WT, or OST
Tidsramme: Up to approximately 5 years
|
For participants who demonstrate a confirmed CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1, DOR is defined as the time from first documented evidence of CR or PR until PD or death.
PD is defined as at least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5 mm.
Note: The appearance of one or more new lesions is also considered PD.
DOR as assessed by the investigator will be presented.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Disease Control Rate (DCR) For Participants With NBL, RMS, WT, or OST
Tidsramme: Up to approximately 5 years
|
DCR is defined, per RECIST 1.1, as the percentage of participants who have a CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions) or Stable Disease (SD).
SD is defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD: at least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5 mm).
Note: The appearance of one or more new lesions is also considered PD.
The time from the first dose until the date of SD must be greater than or equal to 6 weeks.
The DCR as assessed by the investigator will be presented.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Time to Response (TTR) For Participants With NBL, RMS, WT, or OST
Tidsramme: Up to approximately 5 years
|
For participants who demonstrate a confirmed CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1, TTR is defined as the time from the first dose to the first documented evidence of a CR or PR.
The TTR as assessed by the investigator will be presented.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Progression-free Survival (PFS) For Participants With NBL, RMS, WT, or OST
Tidsramme: Up to approximately 5 years
|
PFS is defined as the time from randomization to the first documented PD or death due to any cause, whichever occurs first as assessed by RECIST 1.1.
PD is defined as at least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5 mm.
Note: The appearance of one or more new lesions is also considered PD.
PFS as assessed by the investigator will be presented.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: ORR For participants with OST
Tidsramme: 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 with OST who experience CR or PR as assessed by the investigator will be presented.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Experience an AE
Tidsramme: 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.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Discontinue Study Treatment Due to an AE
Tidsramme: 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.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Receive Dose Modification Due to an AE
Tidsramme: 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.
|
Up to approximately 5 years
|
|
Part 1 and Part 2: Maximum Plasma Concentration (Cmax) of I-Dxd
Tidsramme: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to determine the Cmax of I-Dxd.
|
At designated timepoints (up to approximately 5 years)
|
|
Part 1 and Part 2: Area under the concentration time curve from Time 0 to the End of the Dosing Period (AUCtau) of I-Dxd
Tidsramme: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to determine the AUCtau of I-Dxd.
|
At designated timepoints (up to approximately 5 years)
|
|
Part 1 and Part 2: Plasma Trough Concentration (Ctrough) of I-Dxd
Tidsramme: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to determine the Ctrough of I-Dxd.
|
At designated timepoints (up to approximately 5 years)
|
|
Part 1 and Part 2: Maximum Plasma Concentration (Cmax) of released drug payload (Dxd)
Tidsramme: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to determine the Cmax of Dxd.
|
At designated timepoints (up to approximately 5 years)
|
|
Part 1 and Part 2: Area under the concentration time curve from Time 0 to the End of the Dosing Period (AUCtau) of Dxd
Tidsramme: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to determine the AUCtau of Dxd.
|
At designated timepoints (up to approximately 5 years)
|
|
Part 1 and Part 2: Plasma Trough Concentration (Ctrough) of Dxd
Tidsramme: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to determine the Ctrough of Dxd.
|
At designated timepoints (up to approximately 5 years)
|
|
Part 1 and Part 2: Number of Participants with antidrug antibodies (ADA) against I-Dxd
Tidsramme: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to assess I-Dxd immunogenicity by determining the incidence of ADA of I-Dxd.
|
At designated timepoints (up to approximately 5 years)
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Samarbeidspartnere
Etterforskere
- Studieleder: Medical Director, Merck Sharp & Dohme LLC
Publikasjoner og nyttige lenker
Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.
Hjelpsomme linker
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Faktiske)
6. juli 2026
Primær fullføring (Antatt)
17. august 2031
Studiet fullført (Antatt)
17. august 2031
Datoer for studieregistrering
Først innsendt
1. juni 2026
Først innsendt som oppfylte QC-kriteriene
1. juni 2026
Først lagt ut (Faktiske)
5. juni 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
17. september 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
15. september 2026
Sist bekreftet
1. september 2026
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- 9999-01D
- 2025 (U.S. NIH-stipend/kontrakt: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- LIGHTBEAM-U01 (Annen identifikator: MSD)
- U1111-1322-6561 (Registeridentifikator: UTN)
- 2025-522339-32-00 (Registeridentifikator: EU CT)
- MK-9999-01D (Annen identifikator: MSD)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
JA
IPD-planbeskrivelse
https://trialstransparency.msdclinicaltrials.com/pdf/ProcedureAccessClinicalTrialData.pdf
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .