A Study of Ifinatamab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01D/LIGHTBEAM-U01)
15 septembre 2026 mis à jour par: 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
Aperçu de l'étude
Statut
Recrutement
Les conditions
Intervention / Traitement
Description détaillée
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.
Type d'étude
Interventionnel
Inscription (Estimé)
134
Phase
- Phase 2
- La phase 1
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Coordonnées de l'étude
- Nom: Toll Free Number
- Numéro de téléphone: 1-888-577-8839
- E-mail: Trialsites@msd.com
Lieux d'étude
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Oost-Vlaanderen
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Ghent, Oost-Vlaanderen, Belgique, 9000
- Recrutement
- UZ Gent ( Site 4428)
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Contact:
- Study Coordinator
- Numéro de téléphone: +32 9 332 16 53
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Seoul, Corée du Sud, 03080
- Recrutement
- Seoul National University Hospital-Pediatrics ( Site 4972)
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Contact:
- Study Coordinator
- Numéro de téléphone: 82220723304
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Seoul, Corée du Sud, 05505
- Recrutement
- Asan Medical Center-Pediatrics - Pedicatric Oncology ( Site 4973)
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Contact:
- Study Coordinator
- Numéro de téléphone: 82230105994
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Capital Region
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Copenhagen, Capital Region, Danemark, DK-2100
- Recrutement
- Rigshospitalet ( Site 4467)
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Contact:
- Study Coordinator
- Numéro de téléphone: +4535455002
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Barcelona, Espagne, 08035
- Recrutement
- Hospital Universitari Vall d Hebron ( Site 4716)
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Contact:
- Study Coordinator
- Numéro de téléphone: +34934893093
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Barcelona
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Esplugues de Llobregat, Barcelona, Espagne, 8950
- Recrutement
- Hospital Sant Joan de Déu ( Site 4717)
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Contact:
- Study Coordinator
- Numéro de téléphone: +34671600093
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Madrid, Comunidad de
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Madrid, Madrid, Comunidad de, Espagne, 28009
- Recrutement
- Hospital Niño Jesús ( Site 4715)
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Contact:
- Study Coordinator
- Numéro de téléphone: +34915035900
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Aquitaine
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Bordeaux, Aquitaine, France, 33076
- Recrutement
- Bordeaux University Hospital - Pellegrin ( Site 4105)
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Contact:
- Study Coordinator
- Numéro de téléphone: +33 5 56 79 56 79
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Loire-Atlantique
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Nantes, Loire-Atlantique, France, 44093
- Recrutement
- Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes ( Site 4104)
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Contact:
- Study Coordinator
- Numéro de téléphone: +33240087805
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Rhone
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Lyon, Rhone, France, 69373
- Recrutement
- CENTRE LEON BERARD ( Site 4100)
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Contact:
- Study Coordinator
- Numéro de téléphone: +33469166572
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Haifa, Israël, 3109601
- Recrutement
- Rambam Health Care Campus ( Site 4674)
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Contact:
- Study Coordinator
- Numéro de téléphone: +97247776755
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Ramat Gan, Israël, 5265601
- Recrutement
- Sheba Medical Center ( Site 4675)
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Contact:
- Study Coordinator
- Numéro de téléphone: +97235302996
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Västra Götaland County
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Gothenburg, Västra Götaland County, Suède, 416 85
- Recrutement
- Sahlgrenska Universitetssjukhuset ( Site 4634)
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Contact:
- Study Coordinator
- Numéro de téléphone: +46313435865
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California
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Los Angeles, California, États-Unis, 90027
- Recrutement
- Children's Hospital Los Angeles ( Site 4006)
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Contact:
- Study Coordinator
- Numéro de téléphone: 323-361-2121
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Colorado
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Aurora, Colorado, États-Unis, 80045
- Recrutement
- Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 4016)
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Contact:
- Study Coordinator
- Numéro de téléphone: 720-777-6740
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Iowa
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Iowa City, Iowa, États-Unis, 52242
- Recrutement
- University of Iowa Hospitals ( Site 4017)
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Contact:
- Study Coordinator
- Numéro de téléphone: 319-356-2296
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Massachusetts
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Boston, Massachusetts, États-Unis, 02215
- Recrutement
- Dana Farber Cancer Center ( Site 4013)
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Contact:
- Study Coordinator
- Numéro de téléphone: 617-632-4580
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Michigan
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Grand Rapids, Michigan, États-Unis, 49503
- Recrutement
- Corewell Health ( Site 4001)
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Contact:
- Study Coordinator
- Numéro de téléphone: 616-486-0746
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New Jersey
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New Brunswick, New Jersey, États-Unis, 08901
- Recrutement
- Rutgers Cancer Institute of New Jersey ( Site 4008)
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Contact:
- Study Coordinator
- Numéro de téléphone: 732-235-2465
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New York
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New York, New York, États-Unis, 10065
- Recrutement
- Memorial Sloan Kettering Cancer Center ( Site 4010)
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Contact:
- Study Coordinator
- Numéro de téléphone: 888-492-8401
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Valhalla, New York, États-Unis, 10595
- Recrutement
- New York Medical College ( Site 4023)
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Contact:
- Study Coordinator
- Numéro de téléphone: 914-614-4270
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North Dakota
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Fargo, North Dakota, États-Unis, 58102
- Recrutement
- Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 4003)
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Contact:
- Study Coordinator
- Numéro de téléphone: 701-234-2000
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Pennsylvania
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Philadelphia, Pennsylvania, États-Unis, 19104
- Recrutement
- Children's Hospital of Philadelphia (CHOP) ( Site 4021)
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Contact:
- Study Coordinator
- Numéro de téléphone: 267-425-5544
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South Dakota
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Sioux Falls, South Dakota, États-Unis, 57105
- Recrutement
- Sanford Children's Hospital ( Site 4015)
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Contact:
- Study Coordinator
- Numéro de téléphone: 605-312-1000
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Texas
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Houston, Texas, États-Unis, 77030
- Recrutement
- University of Texas M.D. Anderson Cancer Center ( Site 4007)
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Contact:
- Study Coordinator
- Numéro de téléphone: 713-792-5410
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Utah
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Salt Lake City, Utah, États-Unis, 84113
- Recrutement
- Intermountain - Primary Children's Hospital ( Site 4014)
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Contact:
- Study Coordinator
- Numéro de téléphone: 801-662-4700
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Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
- Enfant
Accepte les volontaires sains
Non
La description
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
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
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Expérimental: Ifinatamab Deruxtecan
Participants receive ifinatamab deruxtecan via intravenous (IV) infusion on day 1 of each 3-week cycle until discontinuation or progression
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IV infusion
Autres noms:
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience a Dose-limiting Toxicity (DLT)
Délai: Cycle 1 (up to approximately 21 days); each cycle is 21 days
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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.
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Cycle 1 (up to approximately 21 days); each cycle is 21 days
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Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience One or More Adverse Events (AEs)
Délai: Up to approximately 5 years
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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.
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Up to approximately 5 years
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Part 1: Number of Participants From ≥1 Month to <12 Years Who Discontinue Study Intervention Due to an AE
Délai: Up to approximately 5 years
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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.
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Up to approximately 5 years
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Part 1: Number of Participants From ≥1 Month to <12 Years Who Receive Dose Modifications Due to AEs
Délai: Up to approximately 5 years
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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.
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Up to approximately 5 years
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Part 1 and Part 2: Objective Response Rate (ORR) for Participants with neuroblastoma (NBL), rhabdomyosarcoma (RMS), and Wilms tumor (WT)
Délai: Up to approximately 5 years
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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.
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Up to approximately 5 years
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Part 1 and Part 2: Disease Control Success at 4 Months (DCS-4) for Participants with osteosarcoma (OST)
Délai: Up to 4 Months
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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.
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Up to 4 Months
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Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Partie 1 et partie 2: survie globale (OS)
Délai: Jusqu'à environ 5 ans
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La SG est définie comme le temps à partir de la première dose de traitement à l'étude à la mort en raison de toute cause.
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Jusqu'à environ 5 ans
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Part 1 and Part 2: Duration of Response (DOR) For Participants With NBL, RMS, WT, or OST
Délai: Up to approximately 5 years
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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.
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Up to approximately 5 years
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Part 1 and Part 2: Disease Control Rate (DCR) For Participants With NBL, RMS, WT, or OST
Délai: Up to approximately 5 years
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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.
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Up to approximately 5 years
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Part 1 and Part 2: Time to Response (TTR) For Participants With NBL, RMS, WT, or OST
Délai: Up to approximately 5 years
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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.
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Up to approximately 5 years
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Part 1 and Part 2: Progression-free Survival (PFS) For Participants With NBL, RMS, WT, or OST
Délai: Up to approximately 5 years
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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.
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Up to approximately 5 years
|
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Part 1 and Part 2: ORR For participants with OST
Délai: 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.
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Up to approximately 5 years
|
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Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Experience an AE
Délai: 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
|
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Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Discontinue Study Treatment Due to an AE
Délai: 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
|
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Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Receive Dose Modification Due to an AE
Délai: 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
|
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Part 1 and Part 2: Maximum Plasma Concentration (Cmax) of I-Dxd
Délai: At designated timepoints (up to approximately 5 years)
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Blood samples will be collected at specified intervals to determine the Cmax of I-Dxd.
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At designated timepoints (up to approximately 5 years)
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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
Délai: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to determine the AUCtau of I-Dxd.
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At designated timepoints (up to approximately 5 years)
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Part 1 and Part 2: Plasma Trough Concentration (Ctrough) of I-Dxd
Délai: 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)
|
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Part 1 and Part 2: Maximum Plasma Concentration (Cmax) of released drug payload (Dxd)
Délai: At designated timepoints (up to approximately 5 years)
|
Blood samples will be collected at specified intervals to determine the Cmax of Dxd.
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At designated timepoints (up to approximately 5 years)
|
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Part 1 and Part 2: Area under the concentration time curve from Time 0 to the End of the Dosing Period (AUCtau) of Dxd
Délai: 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)
|
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Part 1 and Part 2: Plasma Trough Concentration (Ctrough) of Dxd
Délai: 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)
|
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Part 1 and Part 2: Number of Participants with antidrug antibodies (ADA) against I-Dxd
Délai: 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)
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Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Collaborateurs
Les enquêteurs
- Directeur d'études: Medical Director, Merck Sharp & Dohme LLC
Publications et liens utiles
La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.
Liens utiles
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates principales de l'étude
Début de l'étude (Réel)
6 juillet 2026
Achèvement primaire (Estimé)
17 août 2031
Achèvement de l'étude (Estimé)
17 août 2031
Dates d'inscription aux études
Première soumission
1 juin 2026
Première soumission répondant aux critères de contrôle qualité
1 juin 2026
Première publication (Réel)
5 juin 2026
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
17 septembre 2026
Dernière mise à jour soumise répondant aux critères de contrôle qualité
15 septembre 2026
Dernière vérification
1 septembre 2026
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 9999-01D
- 2025 (Subvention/contrat des NIH des États-Unis: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- LIGHTBEAM-U01 (Autre identifiant: MSD)
- U1111-1322-6561 (Identificateur de registre: UTN)
- 2025-522339-32-00 (Identificateur de registre: EU CT)
- MK-9999-01D (Autre identifiant: MSD)
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
OUI
Description du régime IPD
https://trialstransparency.msdclinicaltrials.com/pdf/ProcedureAccessClinicalTrialData.pdf
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Oui
Étudie un produit d'appareil réglementé par la FDA américaine
Non
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .