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NCT06941272PHASE1, PHASE2Recruiting

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

Merck Sharp & Dohme LLC

Start Date

5/26/2025

Completion Date

12/30/2030

Summary

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

Detailed 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

Age Range: No minimum to 17 years

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

Interventions

BIOLOGICAL

Patritumab Deruxtecan

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Conditions

Malignant Neoplasm

Locations

Childrens Hospital Los Angeles ( Site 3006)

Los Angeles, California 90027

United States

Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 3016)

Aurora, Colorado 80045

United States

Yale New Haven Hospital ( Site 3012)

New Haven, Connecticut 06510

United States

Johns Hopkins All Children's Hospital ( Site 3025)

St. Petersburg, Florida 33701

United States

University of Iowa Health Care Holden Comprehensive Cancer Center ( Site 3017)

Iowa City, Iowa 52242

United States

Dana-Farber Cancer Institute ( Site 3013)

Boston, Massachusetts 02215

United States

Corewell Health ( Site 3001)

Grand Rapids, Michigan 49503

United States

Children's Mercy Hospital ( Site 3024)

Kansas City, Missouri 64108

United States

Rutgers Cancer Institute of New Jersey ( Site 3008)

New Brunswick, New Jersey 08901

United States

Memorial Sloan Kettering Cancer Center ( Site 3010)

New York, New York 10065

United States

New York Medical College ( Site 3023)

Valhalla, New York 10595

United States

Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 3003)

Fargo, North Dakota 58102

United States

Oregon Health and Science University ( Site 3004)

Portland, Oregon 97239

United States

Children's Hospital of Philadelphia (CHOP) ( Site 3021)

Philadelphia, Pennsylvania 19104

United States

Sanford Children's Hospital ( Site 3015)

Sioux Falls, South Dakota 57117

United States

University of Texas-MD Anderson Cancer Center ( Site 3007)

Houston, Texas 77030

United States

Intermountain - Primary Children's Hospital ( Site 3014)

Salt Lake City, Utah 84113

United States

Sydney Children's Hospital-Kids Cancer Centre ( Site 3997)

Sydney, New South Wales 2031

Australia

Queensland Children's Hospital ( Site 3996)

Brisbane, Queensland 4101

Australia

Royal Children's Hospital ( Site 3994)

Parkville, Victoria 3052

Australia

UZ Gent ( Site 3428)

Ghent, Oost-Vlaanderen 9000

Belgium

Hospital de Clinicas de Porto Alegre ( Site 3265)

Porto Alegre, Rio Grande do Sul 90035-903

Brazil

Fundação Pio XII - Hospital de Câncer de Barretos ( Site 3264)

Barretos, São Paulo 14784-400

Brazil

Fundação Faculdade Regional de Medicina de São José do Rio Preto ( Site 3267)

São José do Rio Preto, São Paulo 15090-000

Brazil

Alberta Children's Hospital ( Site 3227)

Calgary, Alberta T3B 6A8

Canada

The Hospital for Sick Children ( Site 3225)

Toronto, Ontario M5G 1X8

Canada

Hospital Carlos Van Buren ( Site 3880)

Valparaíso, Valparaiso 2341131

Chile

Hospital Pablo Tobon Uribe ( Site 3923)

Medellín, Antioquia 05034

Colombia

Clinica de la Costa S.A.S. ( Site 3924)

Barranquilla, Atlántico 080020

Colombia

IMAT S.A.S ( Site 3921)

Montería, Departamento de Córdoba 230002

Colombia

Detska nemocnice FN Brno ( Site 3388)

Brno, Brno-mesto 613 00

Czechia

Fakultni nemocnice v Motole-Klinika detske hematologie a onkologie ( Site 3387)

Prague, Praha 5 150 00

Czechia

Rigshospitalet-Department of paediatrics and adolescent medicine, Section of Paed haem-onc ( Site 3467)

Copenhagen, Capital Region DK-2100

Denmark

Bordeaux University Hospital - Pellegrin ( Site 3105)

Bordeaux, Aquitaine 33076

France

Assistance Publique Hôpitaux de Marseille - Hôpital de la Timone ( Site 3102)

Marseille, Bouches-du-Rhone 13005

France

Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes ( Site 3104)

Nantes, Loire-Atlantique 44093

France

Centre Leon-Berard ( Site 3100)

Lyon, Rhone 69373

France

Institut Curie ( Site 3101)

Paris, Île-de-France Region 75248

France

UniversitaetsklInikum Tuebingen ( Site 3142)

Tübingen, Baden-Wurttemberg 72076

Germany

Universitätsklinikum Münster - Albert Schweitzer Campus ( Site 3141)

Münster, North Rhine-Westphalia 48149

Germany

Aghia Sophia Children's Hospital-First Department of Pediatrics, National and Kapodistrian Universi ( Site 3797)

Athens, Attica 11527

Greece

Semmelweis University ( Site 3838)

Budapest, Pest County 1094

Hungary

Rambam Health Care Campus ( Site 3674)

Haifa, 3109601

Israel

Sheba Medical Center ( Site 3675)

Ramat Gan, 5265601

Israel

Fondazione IRCCS Istituto Nazionale dei Tumori ( Site 3552)

Milan, 20133

Italy

Ospedale Pediatrico Bambino Gesù IRCCS ( Site 3553)

Roma, 00165

Italy

Ospedale Infantile Regina Margherita ( Site 3551)

Torino, 10126

Italy

Prinses Maxima Centrum voor Kinderoncologie ( Site 3510)

Utrecht, 3584 CS

Netherlands

Narodny ustav detskych chorob ( Site 3592)

Bratislava, Bratislava Region 831 01

Slovakia

Seoul National University Hospital-Pediatrics ( Site 3972)

Seoul, 03080

South Korea

Asan Medical Center-Pediatrics - Pedicatric Oncology ( Site 3973)

Seoul, 05505

South Korea

Hospital Sant Joan de Déu ( Site 3717)

Esplugues de Llobregat, Barcelona 08950

Spain

Hospital Universitari Vall d''Hebron ( Site 3716)

Barcelona, 08035

Spain

Hospital Infantil Universitario Nino Jesus ( Site 3715)

Madrid, 28009

Spain

Sahlgrenska Universitetssjukhuset ( Site 3634)

Gothenburg, Västra Götaland County 416 85

Sweden

National Taiwan University Hospital ( Site 3983)

Taiwan, Taipei 10002

Taiwan

Hacettepe Universitesi Tip Fakultesi Hastanesi ( Site 3961)

Ankara, 06230

Turkey (Türkiye)

Ankara Bilkent Şehir Hastanesi. ( Site 3962)

Ankara, 6800

Turkey (Türkiye)

Ege Universitesi Hastanesi ( Site 3963)

Izmir, 35100

Turkey (Türkiye)

Birmingham Children's Hospital ( Site 3349)

Birmingham, England B4 6NH

United Kingdom

Royal Victoria Infirmary ( Site 3348)

Newcastle upon Tyne, England NE1 4PL

United Kingdom

University Hospital of Wales ( Site 3346)

Cardiff, CF14 4XW

United Kingdom

Royal Marsden Hospital ( Site 3347)

Sutton, SM2 5PT

United Kingdom