A Study of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01C/LIGHTBEAM-U01)
This research study, called LIGHTBEAM-U01, aims to find better treatments for children with certain types of cancer: hepatoblastoma (a liver cancer) or rhabdomyosarcoma (a cancer starting in muscle cells). These are children whose cancer has either returned after treatment (relapsed) or didn't get better with treatment (refractory). The study is testing a new medicine called HER3-DXd (also known as patritumab deruxtecan). This medicine is designed to target cancer cells specifically while causing less harm to healthy cells. Doctors want to understand if HER3-DXd is safe for children, how their bodies handle the medicine, and if it can help shrink or get rid of the cancer. The study is open to children aged 1 month to 17 years.
At a glance
What is this study about?
This research study is focused on helping children who are battling two specific types of cancer: hepatoblastoma and rhabdomyosarcoma. Hepatoblastoma is a liver cancer that typically affects babies and very young children. Rhabdomyosarcoma is a cancer that starts in muscle cells and can appear in various parts of a child's body, such as the head, neck, bladder, arms, or legs. We're particularly interested in children whose cancer has either come back after they've had treatment (this is called 'relapsed' cancer) or whose cancer hasn't responded well to the treatments they've received so far (this is called 'refractory' cancer).
The study is investigating a new medicine called HER3-DXd, which is also known as patritumab deruxtecan. This medicine is a special type of drug called an 'antibody-drug conjugate' or ADC. Think of it like a carefully guided missile: it's designed to find specific proteins on the surface of cancer cells (like a lock on a door) and then attach to them. Once attached, it delivers the treatment directly into the cancer cell to destroy it, hopefully leaving healthy cells largely untouched. The main goals are to check if this new medicine is safe for children, how it acts in their bodies over time, and if it can help to shrink or even make the cancer disappear.
This study will happen in two stages. The first stage will help doctors figure out the safest and most effective dose of the medicine for children. Once that's established, the second stage will look more closely at how well the medicine works in shrinking the cancer. This careful approach ensures that safety is a top priority throughout the research.
Key takeaways
- This study is for children aged 1 month to 17 years with specific cancers: hepatoblastoma or rhabdomyosarcoma.
- It focuses on cancers that have returned ('relapsed') or not responded to previous treatments ('refractory').
- The new medicine, HER3-DXd, is being tested for safety and its ability to shrink tumours.
- Participation involves receiving the medicine and regular hospital visits for monitoring.
- All potential benefits and risks will be discussed openly by the study team.
- Families can decide to withdraw their child from the study at any time.
Who may be eligible?
To be considered for this study, children must be between 1 month and 17 years old and have either a specific type of advanced rhabdomyosarcoma or hepatoblastoma. Their cancer must have either come back or not responded to at least one previous treatment, and there should be no other standard treatment options available for them. It's important that any side effects from their previous cancer treatments have mostly cleared up, though some minor issues like hair loss or certain nerve problems might be acceptable.
There are also some reasons why a child might not be able to join the study. These include having certain lung conditions, a history of organ or stem cell transplants, specific eye problems, or active cancer spread to the brain. Children with serious heart conditions or certain active infections like untreated Hepatitis B or C would also not be able to take part. The research team will carefully review each child's medical history to ensure the study is safe and appropriate for them.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child between 1 month and 17 years old?
- Does your child have hepatoblastoma or rhabdomyosarcoma that has returned or not responded to treatment?
- Are there no other standard treatment options available for your child's cancer at this time?
- Are any side effects from previous cancer treatments mostly gone or very mild?
- Does your child have any serious heart, lung, or eye conditions?
What does participation involve?
If your child takes part in this study, they would receive the study medicine, HER3-DXd, as part of their treatment. This will involve regular visits to the hospital for the medicine and various checks to monitor their health. These checks typically include blood tests, physical examinations, and scans (like X-rays or CT scans) to see how the cancer is responding and to monitor for any side effects. The frequency and duration of these visits will be explained in detail by the study team. You and your child will be closely supported by the medical team throughout the study, and you can ask questions at any time. The total duration of participation can vary depending on individual response and safety, but regular follow-up will be part of the process.
Potential risks and benefits
Locations (60)
- Childrens Hospital Los Angeles ( Site 3006)Verified postcodeLos Angeles, United States· Recruiting
- Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 3016)Verified postcodeAurora, United States· Recruiting
- Yale New Haven Hospital ( Site 3012)Verified postcodeNew Haven, United States· Recruiting
- Johns Hopkins All Children's Hospital ( Site 3025)Verified postcodeSt. Petersburg, United States· Recruiting
- University of Iowa Health Care Holden Comprehensive Cancer Center ( Site 3017)Verified postcodeIowa City, United States· Recruiting
- Dana-Farber Cancer Institute ( Site 3013)Verified postcodeBoston, United States· Recruiting
- Corewell Health ( Site 3001)Verified postcodeGrand Rapids, United States· Recruiting
- Children's Mercy Hospital ( Site 3024)Verified postcodeKansas City, United States· Recruiting
- Rutgers Cancer Institute of New Jersey ( Site 3008)Verified postcodeNew Brunswick, United States· Recruiting
- Memorial Sloan Kettering Cancer Center ( Site 3010)Verified postcodeNew York, United States· Recruiting
- New York Medical College ( Site 3023)Verified postcodeValhalla, United States· Recruiting
- Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 3003)Verified postcodeFargo, United States· Recruiting
Common questions
What is hepatoblastoma?
Hepatoblastoma is a type of cancer that starts in the liver and usually affects babies and very young children.
What is rhabdomyosarcoma (RMS)?
RMS is a cancer that begins in muscle cells, often found in parts of the body like the head and neck, bladder, or limbs.
What does 'relapsed' or 'refractory' cancer mean?
'Relapsed' means the cancer came back after treatment. 'Refractory' means the cancer did not get smaller or go away with treatment.
What is HER3-DXd / patritumab deruxtecan?
It's a new type of medicine designed to specifically find and deliver treatment to cancer cells, aiming to destroy them while protecting healthy cells.
Will my child definitely get this new medicine if they join the study?
Yes, if your child is eligible and joins this study, they will receive the medicine HER3-DXd as the study treatment.
How to find out more
Toll Free Number
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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