Resumen del Estudio
Este estudio clínico busca nuevas esperanzas para niños diagnosticados con hepatoblastoma (un cáncer de hígado común en la infancia) o rabdomiosarcoma (cáncer muscular) que ha regresado después del tratamiento inicial o que no ha respondido a las terapias estándar. Sabemos lo difícil que es enfrentar un diagnóstico resistente, por lo que la investigación se centra en opciones innovadoras cuando las convencionales se agotan. El tratamiento en investigación es el Patritumab Deruxtecan (HER3-DXd). Funciona como un 'misil inteligente': es un conjugado anticuerpo-fármaco (ADC) diseñado para buscar una proteína específica en la superficie de las células cancerosas y liberar una potente medicación directamente dentro de ellas, buscando destruirlas con mayor precisión. El estudio evaluará principalmente la seguridad de este fármaco en el organismo infantil y su capacidad para reducir el tamaño de los tumores.
Criterios de Elegibilidad
Resumen de Elegibilidad:Inclusión: Niños con Hepatoblastoma o Rabdomiosarcoma confirmado que haya progresado tras al menos un tratamiento previo y sin otras opciones curativas. Exclusión: Historial de enfermedad pulmonar intersticial (neumonitis), trasplante de órganos sólidos o células madre, o metástasis cerebrales activas no controladas.
- Has one of the following histologically confirmed advanced or metastatic solid tumors: Rhabdomyosarcoma (RMS), or Hepatoblastoma
- Has progressed after at least 1 prior systemic treatment for RMS or hepatoblastoma and who has no satisfactory alternative treatment option (ie, is ineligible for other standard treatment regimens)
- Participants who have adverse events (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. Participants with Grade ≤2 alopecia are also eligible
- Hepatitis B surface antigen (HBsAg) positive participants are eligible if they have received hepatitis B virus (HBV) antiviral therapy and have undetectable HBV viral load
- Participants with a history of hepatitis C virus (HCV) infection are eligible if HCV viral load is undetectable
- Has a history of (noninfectious) interstitial lung disease (ILD)/pneumonitis that required steroids or has current ILD/pneumonitis, and/or suspected ILD/pneumonitis that cannot be ruled out by standard diagnostic assessments
- Has clinically severe respiratory compromise resulting from intercurrent pulmonary illness
- Has a history of solid organ transplant
- Has a history of allogeneic stem cell transplant
- Has clinically significant corneal disease
- Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis/leptomeningeal disease; participants with previously treated brain metastases may participate provided they are radiologically stable (ie, without evidence of progression) for at least 4 weeks
- Has uncontrolled or significant cardiovascular disorder
- Has a history of clinically significant congenital cardiac syndrome
- Has a history of human immunodeficiency virus (HIV) infection
- Has a known additional malignancy that is progressing or has required active treatment within the past 1 year
- Has an active infection requiring systemic therapy
- Has concurrent active hepatitis B (HBsAg positive and/or detectable HBV deoxyribonucleic acid \[DNA\]) and HCV defined as anti-HCV antibody (Ab) positive and detectable HCV ribonucleic acid \[RNA\]) infection
- Has not adequately recovered from major surgery or have ongoing surgical complications