A Study of Ifinatamab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01D/LIGHTBEAM-U01)
15 de setembro de 2026 atualizado por: 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
Visão geral do estudo
Status
Recrutamento
Condições
Intervenção / Tratamento
Descrição detalhada
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.
Tipo de estudo
Intervencional
Inscrição (Estimado)
134
Estágio
- Fase 2
- Fase 1
Contactos e Locais
Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.
Contato de estudo
- Nome: Toll Free Number
- Número de telefone: 1-888-577-8839
- E-mail: Trialsites@msd.com
Locais de estudo
-
-
Oost-Vlaanderen
-
Ghent, Oost-Vlaanderen, Bélgica, 9000
- Recrutamento
- UZ Gent ( Site 4428)
-
Contato:
- Study Coordinator
- Número de telefone: +32 9 332 16 53
-
-
-
-
-
Seoul, Coréia do Sul, 03080
- Recrutamento
- Seoul National University Hospital-Pediatrics ( Site 4972)
-
Contato:
- Study Coordinator
- Número de telefone: 82220723304
-
Seoul, Coréia do Sul, 05505
- Recrutamento
- Asan Medical Center-Pediatrics - Pedicatric Oncology ( Site 4973)
-
Contato:
- Study Coordinator
- Número de telefone: 82230105994
-
-
-
-
Capital Region
-
Copenhagen, Capital Region, Dinamarca, DK-2100
- Recrutamento
- Rigshospitalet ( Site 4467)
-
Contato:
- Study Coordinator
- Número de telefone: +4535455002
-
-
-
-
-
Barcelona, Espanha, 08035
- Recrutamento
- Hospital Universitari Vall d Hebron ( Site 4716)
-
Contato:
- Study Coordinator
- Número de telefone: +34934893093
-
-
Barcelona
-
Esplugues de Llobregat, Barcelona, Espanha, 8950
- Recrutamento
- Hospital Sant Joan de Déu ( Site 4717)
-
Contato:
- Study Coordinator
- Número de telefone: +34671600093
-
-
Madrid, Comunidad de
-
Madrid, Madrid, Comunidad de, Espanha, 28009
- Recrutamento
- Hospital Niño Jesús ( Site 4715)
-
Contato:
- Study Coordinator
- Número de telefone: +34915035900
-
-
-
-
California
-
Los Angeles, California, Estados Unidos, 90027
- Recrutamento
- Children's Hospital Los Angeles ( Site 4006)
-
Contato:
- Study Coordinator
- Número de telefone: 323-361-2121
-
-
Colorado
-
Aurora, Colorado, Estados Unidos, 80045
- Recrutamento
- Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 4016)
-
Contato:
- Study Coordinator
- Número de telefone: 720-777-6740
-
-
Iowa
-
Iowa City, Iowa, Estados Unidos, 52242
- Recrutamento
- University of Iowa Hospitals ( Site 4017)
-
Contato:
- Study Coordinator
- Número de telefone: 319-356-2296
-
-
Massachusetts
-
Boston, Massachusetts, Estados Unidos, 02215
- Recrutamento
- Dana Farber Cancer Center ( Site 4013)
-
Contato:
- Study Coordinator
- Número de telefone: 617-632-4580
-
-
Michigan
-
Grand Rapids, Michigan, Estados Unidos, 49503
- Recrutamento
- Corewell Health ( Site 4001)
-
Contato:
- Study Coordinator
- Número de telefone: 616-486-0746
-
-
New Jersey
-
New Brunswick, New Jersey, Estados Unidos, 08901
- Recrutamento
- Rutgers Cancer Institute of New Jersey ( Site 4008)
-
Contato:
- Study Coordinator
- Número de telefone: 732-235-2465
-
-
New York
-
New York, New York, Estados Unidos, 10065
- Recrutamento
- Memorial Sloan Kettering Cancer Center ( Site 4010)
-
Contato:
- Study Coordinator
- Número de telefone: 888-492-8401
-
Valhalla, New York, Estados Unidos, 10595
- Recrutamento
- New York Medical College ( Site 4023)
-
Contato:
- Study Coordinator
- Número de telefone: 914-614-4270
-
-
North Dakota
-
Fargo, North Dakota, Estados Unidos, 58102
- Recrutamento
- Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 4003)
-
Contato:
- Study Coordinator
- Número de telefone: 701-234-2000
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, Estados Unidos, 19104
- Recrutamento
- Children's Hospital of Philadelphia (CHOP) ( Site 4021)
-
Contato:
- Study Coordinator
- Número de telefone: 267-425-5544
-
-
South Dakota
-
Sioux Falls, South Dakota, Estados Unidos, 57105
- Recrutamento
- Sanford Children's Hospital ( Site 4015)
-
Contato:
- Study Coordinator
- Número de telefone: 605-312-1000
-
-
Texas
-
Houston, Texas, Estados Unidos, 77030
- Recrutamento
- University of Texas M.D. Anderson Cancer Center ( Site 4007)
-
Contato:
- Study Coordinator
- Número de telefone: 713-792-5410
-
-
Utah
-
Salt Lake City, Utah, Estados Unidos, 84113
- Recrutamento
- Intermountain - Primary Children's Hospital ( Site 4014)
-
Contato:
- Study Coordinator
- Número de telefone: 801-662-4700
-
-
-
-
Aquitaine
-
Bordeaux, Aquitaine, França, 33076
- Recrutamento
- Bordeaux University Hospital - Pellegrin ( Site 4105)
-
Contato:
- Study Coordinator
- Número de telefone: +33 5 56 79 56 79
-
-
Loire-Atlantique
-
Nantes, Loire-Atlantique, França, 44093
- Recrutamento
- Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes ( Site 4104)
-
Contato:
- Study Coordinator
- Número de telefone: +33240087805
-
-
Rhone
-
Lyon, Rhone, França, 69373
- Recrutamento
- CENTRE LEON BERARD ( Site 4100)
-
Contato:
- Study Coordinator
- Número de telefone: +33469166572
-
-
-
-
-
Haifa, Israel, 3109601
- Recrutamento
- Rambam Health Care Campus ( Site 4674)
-
Contato:
- Study Coordinator
- Número de telefone: +97247776755
-
Ramat Gan, Israel, 5265601
- Recrutamento
- Sheba Medical Center ( Site 4675)
-
Contato:
- Study Coordinator
- Número de telefone: +97235302996
-
-
-
-
Västra Götaland County
-
Gothenburg, Västra Götaland County, Suécia, 416 85
- Recrutamento
- Sahlgrenska Universitetssjukhuset ( Site 4634)
-
Contato:
- Study Coordinator
- Número de telefone: +46313435865
-
-
Critérios de participação
Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.
Critérios de elegibilidade
Idades elegíveis para estudo
- Filho
Aceita Voluntários Saudáveis
Não
Descrição
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
Plano de estudo
Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: Ifinatamab Deruxtecan
Participants receive ifinatamab deruxtecan via intravenous (IV) infusion on day 1 of each 3-week cycle until discontinuation or progression
|
IV infusion
Outros nomes:
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience a Dose-limiting Toxicity (DLT)
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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)
Prazo: 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
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Parte 1 e Parte 2: Sobrevivência geral (OS)
Prazo: Até aproximadamente 5 anos
|
O SO é definido como tempo desde a primeira dose de tratamento de estudo até a morte devido a qualquer causa.
|
Até aproximadamente 5 anos
|
|
Part 1 and Part 2: Duration of Response (DOR) For Participants With NBL, RMS, WT, or OST
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
|
Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Colaboradores
Investigadores
- Diretor de estudo: Medical Director, Merck Sharp & Dohme LLC
Publicações e links úteis
A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.
Datas de registro do estudo
Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.
Datas Principais do Estudo
Início do estudo (Real)
6 de julho de 2026
Conclusão Primária (Estimado)
17 de agosto de 2031
Conclusão do estudo (Estimado)
17 de agosto de 2031
Datas de inscrição no estudo
Enviado pela primeira vez
1 de junho de 2026
Enviado pela primeira vez que atendeu aos critérios de CQ
1 de junho de 2026
Primeira postagem (Real)
5 de junho de 2026
Atualizações de registro de estudo
Última Atualização Postada (Real)
17 de setembro de 2026
Última atualização enviada que atendeu aos critérios de controle de qualidade
15 de setembro de 2026
Última verificação
1 de setembro de 2026
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- 9999-01D
- 2025 (Concessão/Contrato do NIH dos EUA: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- LIGHTBEAM-U01 (Outro identificador: MSD)
- U1111-1322-6561 (Identificador de registro: UTN)
- 2025-522339-32-00 (Identificador de registro: EU CT)
- MK-9999-01D (Outro identificador: MSD)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
SIM
Descrição do plano IPD
https://trialstransparency.msdclinicaltrials.com/pdf/ProcedureAccessClinicalTrialData.pdf
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Sim
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
Não
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .