NCT06941272 · Merck Sharp & Dohme LLC
A Study of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01C/LIGHTBEAM-U01)
What this study is about
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, treatment group$1, 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
View original scientific description
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
Interventions
BIOLOGICAL
Patritumab Deruxtecan
IV Infusion
Primary outcome measures
Part 1: Percentage of Participants Who Experience Dose-limiting Toxicities (DLTs)
Time frame: 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.
Part 1: Percentage of Participants Who Experience an Adverse Event (AE)
Time frame: 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.
Part 1: Percentage of Participants Who Discontinue Study Treatment Due to an AE
Time frame: 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.
Part 1: Area Under the Curve (AUC) of total anti-HER3 antibody liquid chromatography-mass spectrometry (LC-MS) in plasma
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of AUC.
Part 1: AUC of anti-HER3 antibody-conjugated DXd (anti-HER3-ac-DXd) in plasma
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of AUC.
Part 1: AUC of DXd in plasma
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of AUC.
Part 1: Maximum Concentration (Cmax) of anti-HER3 antibody LC-MS in plasma
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of Cmax.
Part 1: Cmax of anti-HER3-ac-DXd in plasma
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of Cmax.
Part 1: Cmax of DXd in plasma
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of Cmax.
Part 1: Concentration Immediately Before the Next Dose is Administered (Ctrough) of anti-HER3 antibody LC-MS in plasma
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of Ctrough.
Part 1: Ctrough of anti-HER3-ac-DXd
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of Ctrough.
Part 1: Ctrough of DXd in plasma
Time frame: At designated timepoints (up to approximately 5 years)
Blood samples will be collected at specified intervals for the determination of Ctrough.
Part 1 and Part 2: Objective Response Rate (ORR)
Time frame: 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) per Response Evaluation Criteria In Solid Tumors Version 1.1 (RECIST 1.1). The percentage of participants who experience CR or PR as assessed by the investigator will be presented.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- 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
Where
- Los Angeles, California
- Aurora, Colorado
- New Haven, Connecticut
- St. Petersburg, Florida
- Iowa City, Iowa
- Boston, Massachusetts
- Grand Rapids, Michigan
- Kansas City, Missouri
- New Brunswick, New Jersey
- New York, New York
- Valhalla, New York
- Fargo, North Dakota
And 5 more locations — see the full list below.
Collaborators
Daiichi Sankyo
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 23, 2026 · Source of record for eligibility and locations