Actively Recruiting
LIGHTBEAM-U01 Substudy 01C: Evaluating Safety and Efficacy of Patritumab Deruxtecan in Children With Relapsed or Refractory Solid Tumors
Led by Merck Sharp & Dohme LLC · Updated on 2026-05-12
50
Participants Needed
60
Research Sites
N/A
Total Duration
On this page
Sponsors
M
Merck Sharp & Dohme LLC
Lead Sponsor
D
Daiichi Sankyo
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are investigating new treatments for children with hepatoblastoma, a common liver cancer in very young children, and rhabdomyosarcoma (RMS), a cancer starting in muscle cells often found in children's head, neck, bladder, arms, or legs. This study focuses on cases where the cancer has returned after treatment (relapsed) or did not respond to treatment (refractory). The research evaluates the safety, how the study drug moves through the body, and whether the cancer shrinks or disappears when treated with HER3-DXd, an antibody-drug conjugate designed to deliver treatment directly to cancer cells. The study consists of two parts: Part 1 is a safety lead-in to find a dose that is tolerable and confirm a recommended dose for further study. Part 2 evaluates how effective the treatment is. Participants receive patritumab deruxtecan (HER3-DXd) through an intravenous infusion on the first day of each 3-week cycle. Treatment continues until the cancer progresses or the participant stops treatment. Children involved in the study will be closely monitored with tests at set times for up to about five years. Researchers will track side effects, how the drug behaves in the body, and cancer response using measurements like tumor size and survival rates. Participants may have blood tests, physical exams, and other assessments regularly to ensure safety and gather information about the treatment's effects over time.
CONDITIONS
Brief Title
A Study of Patritumab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01C/LIGHTBEAM-U01)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Child aged between 1 month and 17 years
- Histologically confirmed diagnosis of rhabdomyosarcoma (RMS) or hepatoblastoma
- Cancer has progressed after at least one prior systemic treatment
- No satisfactory alternative treatment options available
- Recovery from previous anticancer treatment side effects to Grade 1 or baseline, with exceptions for certain endocrine, neuropathy, and alopecia conditions
- Hepatitis B surface antigen positive participants eligible if on antiviral therapy with undetectable viral load
- Participants with history of hepatitis C eligible if viral load is undetectable
You will not qualify if you...
- History of noninfectious interstitial lung disease or pneumonitis requiring steroids, or current/presumed undiagnosed ILD/pneumonitis
- Severe respiratory problems from lung illness
- History of solid organ or allogeneic stem cell transplant
- Clinically significant corneal disease
- Active central nervous system metastases or carcinomatous meningitis, except stable treated brain metastases
- Uncontrolled or significant cardiovascular disorders
- History of congenital cardiac syndrome
- HIV infection
- Active additional malignancy requiring treatment within past year
- Active infection needing systemic therapy
- Concurrent active hepatitis B and hepatitis C infection
- Not fully recovered from major surgery or ongoing surgical complications
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 21-day cycles until discontinuation or progression
Participants receive patritumab deruxtecan via IV infusion on Day 1 of each 3-week cycle until discontinuation or disease progression.
1 visit every 3 weeks (in-person)
Duration - Up to approximately 5 years
Participants are monitored for safety and efficacy outcomes up to approximately 5 years after treatment.
Visits at designated timepoints up to 5 years
Trial Site Locations
Total: 60 locations
1
Childrens Hospital Los Angeles ( Site 3006)
Los Angeles, California, United States, 90027
Actively Recruiting
2
Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 3016)
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Yale New Haven Hospital ( Site 3012)
New Haven, Connecticut, United States, 06510
Actively Recruiting
4
Johns Hopkins All Children's Hospital ( Site 3025)
St. Petersburg, Florida, United States, 33701
Actively Recruiting
5
University of Iowa Health Care Holden Comprehensive Cancer Center ( Site 3017)
Iowa City, Iowa, United States, 52242
Actively Recruiting
6
Dana-Farber Cancer Institute ( Site 3013)
Boston, Massachusetts, United States, 02215
Actively Recruiting
7
Corewell Health ( Site 3001)
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
8
Children's Mercy Hospital ( Site 3024)
Kansas City, Missouri, United States, 64108
Actively Recruiting
9
Rutgers Cancer Institute of New Jersey ( Site 3008)
New Brunswick, New Jersey, United States, 08901
Actively Recruiting
10
Memorial Sloan Kettering Cancer Center ( Site 3010)
New York, New York, United States, 10065
Actively Recruiting
11
New York Medical College ( Site 3023)
Valhalla, New York, United States, 10595
Actively Recruiting
12
Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 3003)
Fargo, North Dakota, United States, 58102
Actively Recruiting
13
Oregon Health and Science University ( Site 3004)
Portland, Oregon, United States, 97239
Actively Recruiting
14
Children's Hospital of Philadelphia (CHOP) ( Site 3021)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
15
Sanford Children's Hospital ( Site 3015)
Sioux Falls, South Dakota, United States, 57117
Actively Recruiting
16
University of Texas-MD Anderson Cancer Center ( Site 3007)
Houston, Texas, United States, 77030
Actively Recruiting
17
Intermountain - Primary Children's Hospital ( Site 3014)
Salt Lake City, Utah, United States, 84113
Actively Recruiting
18
Sydney Children's Hospital-Kids Cancer Centre ( Site 3997)
Sydney, New South Wales, Australia, 2031
Actively Recruiting
19
Queensland Children's Hospital ( Site 3996)
Brisbane, Queensland, Australia, 4101
Actively Recruiting
20
UZ Gent ( Site 3428)
Ghent, Oost-Vlaanderen, Belgium, 9000
Actively Recruiting
21
Hospital de Clinicas de Porto Alegre ( Site 3265)
Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
Actively Recruiting
22
Fundação Pio XII - Hospital de Câncer de Barretos ( Site 3264)
Barretos, São Paulo, Brazil, 14784-400
Actively Recruiting
23
Fundação Faculdade Regional de Medicina de São José do Rio Preto ( Site 3267)
São José do Rio Preto, São Paulo, Brazil, 15090-000
Actively Recruiting
24
The Hospital for Sick Children ( Site 3225)
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
25
Hospital Carlos Van Buren ( Site 3880)
Valparaíso, Valparaiso, Chile, 2341131
Actively Recruiting
26
Hospital Pablo Tobon Uribe ( Site 3923)
Medellín, Antioquia, Colombia, 05034
Actively Recruiting
27
Clinica de la Costa S.A.S. ( Site 3924)
Barranquilla, Atlántico, Colombia, 080020
Actively Recruiting
28
IMAT S.A.S ( Site 3921)
Montería, Departamento de Córdoba, Colombia, 230002
Actively Recruiting
29
Detska nemocnice FN Brno ( Site 3388)
Brno, Brno-mesto, Czechia, 613 00
Actively Recruiting
30
Fakultni nemocnice v Motole-Klinika detske hematologie a onkologie ( Site 3387)
Prague, Praha 5, Czechia, 150 00
Actively Recruiting
31
Rigshospitalet-Department of paediatrics and adolescent medicine, Section of Paed haem-onc ( Site 3467)
Copenhagen, Capital Region, Denmark, DK-2100
Actively Recruiting
32
Bordeaux University Hospital - Pellegrin ( Site 3105)
Bordeaux, Aquitaine, France, 33076
Actively Recruiting
33
Assistance Publique Hôpitaux de Marseille - Hôpital de la Timone ( Site 3102)
Marseille, Bouches-du-Rhone, France, 13005
Actively Recruiting
34
Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes ( Site 3104)
Nantes, Loire-Atlantique, France, 44093
Actively Recruiting
35
Centre Leon-Berard ( Site 3100)
Lyon, Rhone, France, 69373
Actively Recruiting
36
Institut Curie ( Site 3101)
Paris, Île-de-France Region, France, 75248
Actively Recruiting
37
Universitätsklinikum Münster - Albert Schweitzer Campus ( Site 3141)
Münster, North Rhine-Westphalia, Germany, 48149
Actively Recruiting
38
Aghia Sophia Children's Hospital-First Department of Pediatrics, National and Kapodistrian Universi ( Site 3797)
Athens, Attica, Greece, 11527
Actively Recruiting
39
Semmelweis University ( Site 3838)
Budapest, Pest County, Hungary, 1094
Actively Recruiting
40
Rambam Health Care Campus ( Site 3674)
Haifa, Israel, 3109601
Actively Recruiting
41
Sheba Medical Center ( Site 3675)
Ramat Gan, Israel, 5265601
Actively Recruiting
42
Fondazione IRCCS Istituto Nazionale dei Tumori ( Site 3552)
Milan, Italy, 20133
Actively Recruiting
43
Ospedale Pediatrico Bambino Gesù IRCCS ( Site 3553)
Roma, Italy, 00165
Actively Recruiting
44
Ospedale Infantile Regina Margherita ( Site 3551)
Torino, Italy, 10126
Actively Recruiting
45
Prinses Maxima Centrum voor Kinderoncologie ( Site 3510)
Utrecht, Netherlands, 3584 CS
Actively Recruiting
46
Narodny ustav detskych chorob ( Site 3592)
Bratislava, Bratislava Region, Slovakia, 831 01
Actively Recruiting
47
Seoul National University Hospital-Pediatrics ( Site 3972)
Seoul, South Korea, 03080
Actively Recruiting
48
Asan Medical Center-Pediatrics - Pedicatric Oncology ( Site 3973)
Seoul, South Korea, 05505
Actively Recruiting
49
Hospital Sant Joan de Déu ( Site 3717)
Esplugues de Llobregat, Barcelona, Spain, 08950
Actively Recruiting
50
Hospital Universitari Vall d''Hebron ( Site 3716)
Barcelona, Spain, 08035
Actively Recruiting
51
Hospital Infantil Universitario Nino Jesus ( Site 3715)
Madrid, Spain, 28009
Actively Recruiting
52
Sahlgrenska Universitetssjukhuset ( Site 3634)
Gothenburg, Västra Götaland County, Sweden, 416 85
Actively Recruiting
53
National Taiwan University Hospital ( Site 3983)
Taiwan, Taipei, Taiwan, 10002
Actively Recruiting
54
Hacettepe Universitesi Tip Fakultesi Hastanesi ( Site 3961)
Ankara, Turkey (Türkiye), 06230
Actively Recruiting
55
Ankara Bilkent Şehir Hastanesi. ( Site 3962)
Ankara, Turkey (Türkiye), 6800
Actively Recruiting
56
Ege Universitesi Hastanesi ( Site 3963)
Izmir, Turkey (Türkiye), 35100
Actively Recruiting
57
Birmingham Children's Hospital ( Site 3349)
Birmingham, England, United Kingdom, B4 6NH
Actively Recruiting
58
Royal Victoria Infirmary ( Site 3348)
Newcastle upon Tyne, England, United Kingdom, NE1 4PL
Actively Recruiting
59
University Hospital of Wales ( Site 3346)
Cardiff, United Kingdom, CF14 4XW
Actively Recruiting
60
Royal Marsden Hospital ( Site 3347)
Sutton, United Kingdom, SM2 5PT
Actively Recruiting
Research Team
T
Toll Free Number
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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