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A Study of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01C/LIGHTBEAM-U01)

RecruitingPhase 1/2

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

Who can join

1 Month – 17 Years · All sexes

Full eligibility criteria
The main inclusion criteria include but are not limited to the following:

* 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

The main exclusion criteria include but are not limited to the following:

* Has a history of interstitial lung disease (ILD)/pneumonitis irrespective of steroid use, or current ILD/pneumonitis, or suspected ILD/pneumonitis, or ILD that cannot be ruled out by imaging
* 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

About the study

Researchers are looking for new ways to treat children with hepatoblastoma or rhabdomyosarcoma (RMS) that has relapsed or is refractory:

* Hepatoblastoma is a common liver cancer in babies and very young children * RMS is a cancer that starts in muscle cells, often in a child's head and neck, bladder, arms, or legs * Relapsed means the cancer came back after treatment * Refractory means the cancer did not respond (get smaller or go away) to treatment

The study treatment HER3-DXd (also known as MK-1022 or patritumab 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 HER3-DXd in children and if they tolerate it * What happens to HER3-DXd in children's bodies over time * If children who receive HER3-DXd have the cancer get smaller or go away

What is being tested

Sponsor: Merck Sharp & Dohme LLC · Participants: 50 · Started: May 26, 2025

Contact the study team

Official record on ClinicalTrials.gov — NCT06941272

Locations in the U.S.

CaliforniaChildrens Hospital Los Angeles ( Site 3006), Los Angeles
ColoradoChildren's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 3016), Aurora
ConnecticutYale New Haven Hospital ( Site 3012), New Haven
FloridaJohns Hopkins All Children's Hospital ( Site 3025), St. Petersburg
IowaUniversity of Iowa Health Care Holden Comprehensive Cancer Center ( Site 3017), Iowa City
MassachusettsDana-Farber Cancer Institute ( Site 3013), Boston
MichiganCorewell Health ( Site 3001), Grand Rapids
MissouriChildren's Mercy Hospital ( Site 3024), Kansas City
New JerseyRutgers Cancer Institute of New Jersey ( Site 3008), New Brunswick
New YorkMemorial Sloan Kettering Cancer Center ( Site 3010), New York
New York Medical College ( Site 3023), Valhalla
North DakotaSanford Fargo Medical Center-Roger Maris Cancer Center ( Site 3003), Fargo
OregonOregon Health and Science University ( Site 3004), Portland
PennsylvaniaChildren's Hospital of Philadelphia (CHOP) ( Site 3021), Philadelphia
South DakotaSanford Children's Hospital ( Site 3015), Sioux Falls
TexasUniversity of Texas-MD Anderson Cancer Center ( Site 3007), Houston
UtahIntermountain - Primary Children's Hospital ( Site 3014), Salt Lake City

Conditions

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.