LIGHTBEAM-U01 Substudy 01C in Paediatric & Young Adult Participants

• Research type

  Research Study

• Full title

  LIGHTBEAM-U01 Substudy 01C: A Phase 1/2 Substudy to Evaluate the Safety and Efficacy of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors

• IRAS ID

  1010764

• Contact name

  - -

• Contact email

  N/A

• Sponsor organisation

  Merck Sharp & Dohme LLC

• Research summary

  Researchers are looking for new ways to treat children with hepatoblastoma or rhabdomyosarcoma (RMS) that has relapsed or is refractory:
  • Relapsed means the cancer came back after treatment
  • Refractory means the cancer did not respond to treatment
  The study treatment HER3-DXd is an antibody–drug conjugate (ADC). An ADC attaches to a protein on cancer cells and delivers treatment to destroy those cells.

  About 33 to 50 children will be in this study.

  All children will receive the study treatment HER3-DXd into a vein as an intravenous (IV) infusion every 3 weeks. Children will receive it until they (or their parents or caregivers) choose to stop it, the researchers decide to stop it, the cancer grows or spreads, or the study ends.
  Researchers will put children in 1 of 4 groups based on their age when they join the study:

  • Group A: 12 years old to less than 18
  • Group B: 6 years old to less than 12
  • Group C: 2 years old to less than 6
  • Group D: 1 month old to less than 2
  
  This study has 2 parts:
  • Part 1: Researchers will start by giving 1 dose level (amount) of HER3-DXd to a few children in Group B. They will adjust the dose level of HER3-DXd, if needed, to find a dose level to use in Part 2 for Group B. Researchers will repeat this in Groups C and D.
  • Part 2: Children in Group A will receive a dose level of HER3-DXd based on studies in adults. Researchers will also give HER3-DXd to more children from Groups B, C, and D as their dose level is found in Part 1.

  During the study, children will have urine, blood, eye, and imaging tests. Children may be in this study for up to about 5 years.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  25/NW/0038

• Date of REC Opinion

  8 May 2025

• REC opinion

  Further Information Favourable Opinion