Clinical trial • Phase I/II • Oncology

PATRITUMAB DERUXTECAN for Hepatoblastoma | Rhabdomyosarcoma

Phase I/II trial of PATRITUMAB DERUXTECAN for Hepatoblastoma | Rhabdomyosarcoma. open-label, adaptive. 45 participants.

Overview

Trial Therapeutic Area
Oncology
Trial Disease
Hepatoblastoma | Rhabdomyosarcoma
Trial Stage
Phase I/II
Drug Modality
ADC | Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
18-02-2025
First CTIS Authorization Date
03-06-2025

Trial design

open-label, adaptive Phase I/II trial in Sweden, Hungary, Netherlands and others.

Open Label
Yes
Adaptive
True, Dose-escalation design in Part 1 to evaluate DLTs and establish a preliminary RP2D; PK characterization and interim safety evaluations are included
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
45

Eligibility

Recruits 45 paediatric patients.

Vulnerable Population
Pediatric participants are included (paediatric trial). The submission contains multiple age-specific assent and consent documents: assent forms for age groups (02-05 yr, 06-09 yr, 10-14 yr, 15-17 yr) and parent/guardian consent forms; 'child become adult' consent forms and adult/custodian consent forms are provided where applicable. Country-specific ICFs and assent forms are present (documents for SWE/Swedish, HUN/Hungarian, NLD/Dutch, GRC/Greek, FRA/French, ESP/Spanish, DEU/German, CZE/Czech, SVK/Slovak, ITA/Italian, DNK/Danish, BEL/English/Dutch/French). Parent/guardian consent is required for minors and assent is obtained according to age group; child-to-adult transition consent materials are included.

Inclusion criteria

  • {"criterion_text":"- Has one of the following histologically confirmed advanced or metastatic solid tumors: Rhabdomyosarcoma (RMS), or Hepatoblastoma"}
  • {"criterion_text":"- 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)"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- Participants with a history of hepatitis C virus (HCV) infection are eligible if HCV viral load is undetectable"}

Exclusion criteria

  • {"criterion_text":"- 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"}
  • {"criterion_text":"- Has a history of clinically significant congenital cardiac syndrome"}
  • {"criterion_text":"- Has a history of human immunodeficiency virus (HIV) infection"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- Has not adequately recovered from major surgery or have ongoing surgical complications"}
  • {"criterion_text":"- Has clinically severe respiratory compromise resulting from intercurrent pulmonary illness"}
  • {"criterion_text":"- Has a history of solid organ transplant"}
  • {"criterion_text":"- Has a history of allogeneic stem cell transplant"}
  • {"criterion_text":"- Has clinically significant corneal disease"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- Has uncontrolled or significant cardiovascular disorder"}
  • {"criterion_text":"- Has a history of clinically significant congenital cardiac syndrome"}
  • {"criterion_text":"- Has a known additional malignancy that is progressing or has required active treatment within the past 1 year"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Part 1: Percentage of Participants Who Experience Dose-limiting Toxicities (DLTs)","definition_or_measurement_approach":"Percentage (proportion) of participants experiencing DLTs during Part 1 (safety/drug-limiting toxicity assessment)."}
  • {"endpoint_text":"- Part 1: Percentage of Participants Who Experience an Adverse Event (AE)","definition_or_measurement_approach":"Percentage of participants with at least one AE (incidence of AEs during Part 1)."}
  • {"endpoint_text":"- Part 1: Percentage of Participants Who Discontinue Study Treatment Due to an AE","definition_or_measurement_approach":"Percentage of participants who discontinue study treatment because of an AE during Part 1."}
  • {"endpoint_text":"- Part 1: Area Under the Curve (AUC) of total anti-HER3 antibody liquid chromatography-mass spectrometry (LC-MS) in plasma","definition_or_measurement_approach":"Pharmacokinetic measure: AUC of total anti-HER3 antibody in plasma measured by LC-MS."}
  • {"endpoint_text":"- Part 1: AUC of anti-HER3 antibody-conjugated DXd (anti-HER3-ac-DXd) in plasma","definition_or_measurement_approach":"PK measure: AUC of antibody-conjugated DXd in plasma."}
  • {"endpoint_text":"- Part 1: AUC of DXd in plasma","definition_or_measurement_approach":"PK measure: AUC of DXd (payload) in plasma."}
  • {"endpoint_text":"- Part 1: Maximum Concentration (Cmax) of anti-HER3 antibody LC-MS in plasma","definition_or_measurement_approach":"PK measure: Cmax of anti-HER3 antibody in plasma measured by LC-MS."}
  • {"endpoint_text":"- Part 1: Cmax of anti-HER3-ac-DXd in plasma","definition_or_measurement_approach":"PK measure: Cmax of antibody-conjugated DXd in plasma."}
  • {"endpoint_text":"- Part 1: Cmax of DXd in plasma","definition_or_measurement_approach":"PK measure: Cmax of DXd in plasma."}
  • {"endpoint_text":"- Part 1: Concentration Immediately Before the Next Dose is Administered (Ctrough) of anti-HER3 antibody LC-MS in plasma","definition_or_measurement_approach":"PK trough concentration (Ctrough) of anti-HER3 antibody in plasma by LC-MS measured pre-dose."}
  • {"endpoint_text":"- Part 1: Ctrough of anti-HER3-ac-DXd","definition_or_measurement_approach":"PK trough concentration of anti-HER3-ac-DXd in plasma measured pre-dose."}
  • {"endpoint_text":"- Part 1: Ctrough of DXd in plasma","definition_or_measurement_approach":"PK trough concentration of DXd in plasma measured pre-dose."}
  • {"endpoint_text":"- Part 1 and Part 2: Objective Response Rate (ORR)","definition_or_measurement_approach":"Efficacy measure: ORR assessed by RECIST 1.1 per investigator assessment by tumor type."}

Secondary endpoints

  • {"endpoint_text":"- Part 2: Percentage of Participants Who Experience an AE","definition_or_measurement_approach":"Percentage/incidence of participants experiencing AEs during Part 2."}
  • {"endpoint_text":"- Part 2: Percentage of Participants Who Discontinue Study Treatment Due to an AE","definition_or_measurement_approach":"Percentage of participants discontinuing treatment due to AEs during Part 2."}
  • {"endpoint_text":"- Part 1 and Part 2: Disease Control Rate (DCR)","definition_or_measurement_approach":"Disease control rate assessed by RECIST 1.1 per investigator assessment."}
  • {"endpoint_text":"- Part 1 and Part 2: Time to Response (TTR)","definition_or_measurement_approach":"Time from treatment start to first documented response."}
  • {"endpoint_text":"- Part 1 and Part 2: Duration of Response (DOR)","definition_or_measurement_approach":"Duration from initial documented response to progression or death."}
  • {"endpoint_text":"- Part 1 and Part 2: Progressive-free Survival (PFS)","definition_or_measurement_approach":"Time from treatment start to disease progression or death by RECIST 1.1."}
  • {"endpoint_text":"- Part 1 and Part 2: Overall Survival (OS)","definition_or_measurement_approach":"Time from treatment start to death from any cause."}
  • {"endpoint_text":"- Part 2: AUC of total anti-HER3 antibody LC-MS in plasma","definition_or_measurement_approach":"PK measure: AUC of total anti-HER3 antibody in plasma by LC-MS during Part 2."}
  • {"endpoint_text":"- Part 2: AUC of anti-HER3-ac-DXd in plasma","definition_or_measurement_approach":"PK measure: AUC of anti-HER3-ac-DXd in plasma during Part 2."}
  • {"endpoint_text":"- Part 2: AUC of DXd in plasma","definition_or_measurement_approach":"PK measure: AUC of DXd in plasma during Part 2."}
  • {"endpoint_text":"- Part 2: Cmax of anti-HER3 antibody LC-MS in plasma","definition_or_measurement_approach":"PK measure: Cmax of anti-HER3 antibody by LC-MS in plasma during Part 2."}
  • {"endpoint_text":"- Part 2: Cmax of anti-HER3-ac-DXd in plasma","definition_or_measurement_approach":"PK measure: Cmax of anti-HER3-ac-DXd in plasma during Part 2."}
  • {"endpoint_text":"- Part 2: Cmax of DXd in plasma","definition_or_measurement_approach":"PK measure: Cmax of DXd in plasma during Part 2."}
  • {"endpoint_text":"- Part 2: Ctrough of anti-HER3 antibody LC-MS in plasma","definition_or_measurement_approach":"PK trough concentration (Ctrough) of anti-HER3 antibody in plasma during Part 2."}
  • {"endpoint_text":"- Part 2: Ctrough of anti-HER3-ac-DXd in plasma","definition_or_measurement_approach":"PK trough concentration of anti-HER3-ac-DXd in plasma during Part 2."}
  • {"endpoint_text":"- Part 2: Ctrough of DXd in plasma","definition_or_measurement_approach":"PK trough concentration of DXd in plasma during Part 2."}

Recruitment

Planned Sample Size
45
Recruitment Window Months
67
Consent Approach
Informed consent and assent processes are age-stratified. Assent documents are provided for young age groups (examples of available assent documents: 02-05 yr, 06-09 yr, 10-14 yr, 15-17 yr). Parent/guardian consent forms are provided for minors; 'child become adult' consent materials are included for participants transitioning to adulthood. There are separate FBR (full burden reduction) and optional consent forms (e.g., genetic consent, pregnancy follow-up, optional Greenphire payment consent) and emergency/patient ID cards. Consent/assent materials are country- and language-specific (documents available for Sweden [SWE/SV], Hungary [HUN/HU], Netherlands [NLD/NL], Greece [GRC/EL], France [FRA/FR], Spain [ESP/ES], Germany [DEU/DE], Czechia [CZE/CS], Slovakia [SVK/SK], Italy [ITA/IT], Denmark [DNK/DA], Belgium [BEL/EN, BEL/NL, BEL/FR]).

Geography

Total Number Of Sites
26
Total Number Of Participants
32

Sweden

Earliest CTIS Part Ii Submission Date
30-04-2025
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
345
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Queen Silvia Childrens Hospital - Sahlgrenska University Hospital - Vaestra Goetalandsregionen
Department Name
Barncancercentrum
Contact Person Name
Karin Mellgren
Contact Person Email
karin.mellgren@vgregion.se

Hungary

Earliest CTIS Part Ii Submission Date
06-08-2025
Latest Decision Or Authorization Date
17-04-2026
Processing Time Days
254
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Semmelweis University
Department Name
Gyermekgyógyászati Klinika, Tűzoltó utcai részleg
Contact Person Name
Andrea Ponyi
Contact Person Email
ponyi.andrea@gyerekklinika.com

Netherlands

Earliest CTIS Part Ii Submission Date
15-08-2025
Latest Decision Or Authorization Date
13-04-2026
Processing Time Days
241
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Prinses Maxima Centrum voor Kinderoncologie B.V.
Department Name
Kinderoncologie
Contact Person Name
Natasha van Eijkelenburg

Slovakia

Earliest CTIS Part Ii Submission Date
22-08-2025
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
231
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Narodny Ustav Detskych Chorob
Department Name
Klinika detskej hematologie a onkologie
Contact Person Name
Alexandra Kolenova
Contact Person Email
alexandra.kolenova@nudch.eu

Greece

Earliest CTIS Part Ii Submission Date
14-07-2025
Latest Decision Or Authorization Date
15-04-2026
Processing Time Days
275
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Nosokomeio Paidon I Agia Sofia
Department Name
Division of Pediatric Hematology-Oncology, 1st Pediatric Clinic
Contact Person Name
Antonios Kattamis
Contact Person Email
ankatt@med.uoa.gr

Italy

Earliest CTIS Part Ii Submission Date
21-08-2025
Latest Decision Or Authorization Date
14-04-2026
Processing Time Days
236
Number Of Sites
3
Number Of Participants
7

Sites

Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
S.C. Oncoematologia Pediatrica
Contact Person Name
Franca Fagioli
Contact Person Email
franca.fagioli@unito.it
Site Name
Fondazione IRCCS Istituto Nazionale Dei Tumori
Department Name
S.C. Pediatria Oncologica
Contact Person Name
Michela Casanova
Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Contact Person Name
Franco Locatelli
Contact Person Email
franco.locatelli@opbg.net

Belgium

Earliest CTIS Part Ii Submission Date
11-08-2025
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
242
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Universitair Ziekenhuis Gent
Department Name
Pediatric Oncology
Contact Person Name
Bram De Wilde
Contact Person Email
bram.dewilde@uzgent.be

Czechia

Earliest CTIS Part Ii Submission Date
18-08-2025
Latest Decision Or Authorization Date
14-04-2026
Processing Time Days
239
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Fakultni Nemocnice Brno
Department Name
Klinika dětské onkologie
Contact Person Name
Peter Múdry
Contact Person Email
mudry.peter@fnbrno.cz
Site Name
Fakultni Nemocnice Motol A Homolka
Department Name
Klinika dětské hematologie a onkologie
Contact Person Name
Michaela Čepelová
Contact Person Email
Michaela.Cepelova@fnmotol.cz

Denmark

Earliest CTIS Part Ii Submission Date
29-08-2025
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
224
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Rigshospitalet
Department Name
Department of paediatrics and adolescent medicine, Section of Paed haem-onc
Contact Person Name
Ruta Tuckuviene
Contact Person Email
ruta.tuckuviene@regionh.dk

Spain

Earliest CTIS Part Ii Submission Date
03-03-2025
Latest Decision Or Authorization Date
15-04-2026
Processing Time Days
408
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Hospital Sant Joan De Deu Barcelona
Department Name
Oncological Pediatric Unit
Contact Person Name
Alicia Castañeda Heredia
Contact Person Email
alicia.castanedah@sjd.es
Site Name
Hospital Infantil Universitario Nino Jesus
Department Name
Pediatric
Contact Person Name
Alba Rubio San Simón
Contact Person Email
alba.rubio@salud.madrid.org
Site Name
Hospital Universitari Vall D Hebron
Department Name
Pediatric Oncology and Hematology Department
Contact Person Name
Raquel Hladun Alvaro
Contact Person Email
raquel.hladun@vallhebron.cat

Germany

Earliest CTIS Part Ii Submission Date
30-04-2025
Latest Decision Or Authorization Date
13-04-2026
Processing Time Days
348
Number Of Sites
5
Number Of Participants
5

Sites

Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
Abteilung für päd. Hämatologie und Onkologie
Contact Person Name
Martin Ebinger
Site Name
Universitaet Muenster
Department Name
Pädiatrische Hämatologie und Onkologie
Contact Person Name
Claudia Rössig
Contact Person Email
paedonc@ukmuenster.de
Site Name
University Hospital Cologne AöR
Department Name
Pädiatrische Onkologie und Hämatologie
Contact Person Name
Matthias Fischer
Contact Person Email
matthias.fischer@uk-koeln.de
Site Name
Justus-Liebig-Universitaet Giessen
Department Name
Zentrum für Kinderheilkunde
Contact Person Name
Christine Mauz-Körholz
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Klinik für Pädiatrie m. S. Onkologie und Hämatologie
Contact Person Name
Anne Thorwarth
Contact Person Email
cathrin.schmeller@charite.de

France

Earliest CTIS Part Ii Submission Date
24-04-2025
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
351
Number Of Sites
6
Number Of Participants
6

Sites

Site Name
Institut Curie
Department Name
SIREDO (Soins, innovation, Recherche, en oncologie de l'Enfant, de l'adolescent et de l'adulte)
Contact Person Name
Amaury Leruste
Contact Person Email
amaury.leruste@curie.fr
Site Name
Centre Leon Berard
Department Name
Institut d'Hématologie et d'Oncologie Pédiatrique (IHOPe)
Contact Person Name
Nadège Corradini
Contact Person Email
nadege.corradini@ihope.fr
Site Name
Centre Hospitalier Universitaire De Nantes
Department Name
Oncologie - Hématologie Pédiatrique
Contact Person Name
Morgane Cleirec
Contact Person Email
morgane.cleirec@chu-nantes.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Service de Pédiatrie
Contact Person Name
Stéphane Ducassou
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Service d'immunoogie hématologie et Oncologie Pédiatrique
Contact Person Name
Nicolas André
Contact Person Email
nicolas.andre@ap-hm.fr
Site Name
Institut Gustave Roussy
Department Name
Cancérologie Enfant et Adolescent
Contact Person Name
Pablo Berlanga

Sponsor

Primary sponsor

Full Name
Merck Sharp & Dohme LLC
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
PPD Development LP
Responsibilities
sponsorDuties codes: 4
Name
Parexel International Corp.
Responsibilities
EUB services (call center and medical services)
Name
Almac Clinical Services LLC
Responsibilities
sponsorDuties codes: 3
Name
Bioclinica Inc.
Responsibilities
Site imaging acquisition training and the collection of trial images.
Name
Fortrea Inc.
Responsibilities
sponsorDuties codes: 1
Name
Labcorp Central Laboratory Services SARL
Responsibilities
sponsorDuties codes: 4
Name
Infinity Biologix LLC
Responsibilities
sponsorDuties codes: 4

Third parties

  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Parexel International Corp.","duties_or_roles":"EUB services (call center and medical services) (sponsorDuties code 15)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Ventana Medical Systems Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"sponsorDuties codes: 1","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Infinity Biologix LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Almac Clinical Services LLC","duties_or_roles":"sponsorDuties codes: 3","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Site imaging acquisition training and the collection of trial images. (sponsorDuties code 15)","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
MK-1022
Active Substance
PATRITUMAB DERUXTECAN
Modality
ADC
Routes Of Administration
INTRAVENOUS
Route
INTRAVENOUS
Investigational Product Name
DEXAMETHASONE
Active Substance
CINCHOCAINE HYDROCHLORIDE, DEXAMETHASONE, 1,3-BUTYLENE GLYCOL
Modality
Small molecule
Routes Of Administration
OTHER USE
Investigational Product Name
Serotonin (5HT3) antagonists
Modality
Small molecule
Routes Of Administration
OTHER USE
Investigational Product Name
Other antiemetics
Modality
Small molecule
Routes Of Administration
OTHER USE

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