A Study of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01C/LIGHTBEAM-U01)
Sponsor: Merck Sharp & Dohme LLC
ClinicalTrials ID:NCT06941272
This clinical trial evaluates the safety and efficacy of patritumab deruxtecan in pediatric patients with relapsed or refractory hepatoblastoma or rhabdomyosarcoma. The study consists of two parts: a safety lead-in to establish a tolerable dose, followed by an efficacy assessment. Participants will receive patritumab deruxtecan via IV infusion.
Patient Parameters
| Parameter | Options |
|---|
Program Overview
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
Description
This study will have 2 parts: a safety lead-in to demonstrate a tolerable safety profile and confirm a preliminary recommended phase 2 dose (RP2D) (Part 1) followed by an efficacy evaluation (Part 2).
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
- 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 (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
Locations
| FACILITY | ZIP | CITY | STATE |
|---|---|---|---|
| Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 3016) | 80045 | Aurora | Colorado |
| University of Iowa Health Care Holden Comprehensive Cancer Center ( Site 3017) | 52242 | Iowa City | Iowa |
| Corewell Health ( Site 3001) | 49503 | Grand Rapids | Michigan |
| Children's Mercy Hospital ( Site 3024) | 64108 | Kansas City | Missouri |
| Rutgers Cancer Institute of New Jersey ( Site 3008) | 08901 | New Brunswick | New Jersey |
| New York Medical College ( Site 3023) | 10595 | Valhalla | New York |
| Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 3003) | 58122 | Fargo | North Dakota |
| Children's Hospital of Philadelphia (CHOP) ( Site 3021) | 19104 | Philadelphia | Pennsylvania |
| Sanford Children's Hospital ( Site 3015) | 57117 | Sioux Falls | South Dakota |
| University of Texas-MD Anderson Cancer Center ( Site 3007) | 77030 | Houston | Texas |
Get This Program In Your Inbox
You can sign up and apply your patients for this program.