Malignant NeoplasmPhase 1/2NCT07630974
A Study of Ifinatamab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors (MK-9999-01D/LIGHTBEAM-U01)
A Phase 1/2 study testing Ifinatamab Deruxtecan in people with Malignant Neoplasm, run by Merck Sharp & Dohme LLC at 34 sites in 13 countries.
Trial summary
Researchers are looking for new ways to treat children with relapsed or refractory solid tumors: Relapsed means the cancer came back after treatment Refractory means the cancer did not respond (get smaller or go away) to treatment Solid tumors are cancers mostly in body organs and tissues, not in the blood or other… Read more about this study
- Who is it for?
- Ages 1 month to 17 years
- Conditions the study is for
- Malignant Neoplasm
- What is being tested?
- Ifinatamab Deruxtecan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 134 participants
- Where
- 34 sitesin 13 countries
- Study dates
- 6 Jul 2026to 17 Aug 2031
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 17 years
- All sexes
- No healthy volunteers
- Child
The main inclusion criteria include but are not limited to the following:
- In Part 1, participant has recurrent or relapsed, refractory solid tumors (excluding primary central nervous system (CNS)); and in Part 2, participant has recurrent or relapsed, refractory and histologically confirmed diagnosis of osteosarcoma (OST), neuroblastoma (NBL), rhabdomyosarcoma (RMS), or Wilms tumor (WT). All participants must meet the following criteria: Has documented radiological disease progression after at least 1 line of prior therapy in the locally advanced/metastatic setting and who has no satisfactory alternative treatment option (ie, is ineligible for other standard treatment regimens).
- Is an individual of any sex/gender, ≥1 month to <12 years of age for Part 1 and ≥1 month to <18 years for Part 2 at the time of providing the informed consent or assent, as applicable
- Participants who have 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.
The main exclusion criteria include but are not limited to the following:
- Has clinically significant corneal disease
- Has a history of cerebrovascular accident, transient ischemic attack, or another arterial thromboembolic event within 6 months before screening
- Has uncontrolled or significant cardiovascular disease, including conduction abnormalities, hypertension, ischemic heart disease, heart failure, and peripheral vascular disease
- Has any history of interstitial lung disease (ILD)/pneumonitis, irrespective of steroid use, except for a history of radiation pneumonitis that did not require steroids, current ILD, or Clinical or radiographic suspicion of ILD for which the diagnosis of ILD cannot be ruled out
- Has clinically severe respiratory compromise resulting from intercurrent pulmonary illnesses
- Has an active, known or suspected autoimmune disease.
- Has history of solid organ transplant.
- Has history of allogeneic stem cell transplant (SCT).
- Has known active CNS metastases and/or carcinomatous meningitis/leptomeningeal disease/spinal cord compression. Participants with untreated and asymptomatic brain metastases or previously treated brain metastases may participate provided they are radiologically stable, (i.e, without evidence of progression) for at least 4 weeks
- Has history of human immunodeficiency virus (HIV) infection.
- Has known additional malignancy that is progressing or has required active treatment within the past 1 year.
- Has active infection requiring systemic therapy
- Has known hypersensitivity or contraindication to either the study intervention substance or inactive ingredients in the study intervention product
- Participants who have not adequately recovered from major surgery or have ongoing surgical complications
Trial locations and contacts
34 sites in 13 countries. Contact the study team or a site near you to ask about taking part.
34 sites in 13 countries
United States15 sites, 15 recruiting
- Recruiting
Children's Hospital Colorado-Center for Cancer and Blood Disorders ( Site 4016)
Aurora, Colorado, 80045
Study Coordinator, 720-777-6740
- Recruiting
Dana Farber Cancer Center ( Site 4013)
Boston, Massachusetts, 02215
Study Coordinator, 617-632-4580
- Recruiting
Sanford Fargo Medical Center-Roger Maris Cancer Center ( Site 4003)
Fargo, North Dakota, 58102
Study Coordinator, 701-234-2000
- Recruiting
Corewell Health ( Site 4001)
Grand Rapids, Michigan, 49503
Study Coordinator, 616-486-0746
- Recruiting
University of Texas M.D. Anderson Cancer Center ( Site 4007)
Houston, Texas, 77030
Study Coordinator, 713-792-5410
- Recruiting
University of Iowa Hospitals ( Site 4017)
Iowa City, Iowa, 52242
Study Coordinator, 319-356-2296
- Recruiting
The Children's Mercy Hospitals & Clinics ( Site 4024)
Kansas City, Missouri, 64108
Study Coordinator, 816-302-6808
- Recruiting
Children's Hospital Los Angeles ( Site 4006)
Los Angeles, California, 90027
Study Coordinator, 323-361-2121
- Recruiting
Rutgers Cancer Institute of New Jersey ( Site 4008)
New Brunswick, New Jersey, 08901
Study Coordinator, 732-235-2465
- Recruiting
Memorial Sloan Kettering Cancer Center ( Site 4010)
New York, New York, 10065
Study Coordinator, 888-492-8401
- Recruiting
Children's Hospital of Philadelphia (CHOP) ( Site 4021)
Philadelphia, Pennsylvania, 19104
Study Coordinator, 267-425-5544
- Recruiting
Intermountain - Primary Children's Hospital ( Site 4014)
Salt Lake City, Utah, 84113
Study Coordinator, 801-662-4700
- Recruiting
Sanford Children's Hospital ( Site 4015)
Sioux Falls, South Dakota, 57105
Study Coordinator, 605-312-1000
- Recruiting
Johns Hopkins All Children's Hospital ( Site 4025)
St. Petersburg, Florida, 33701
Study Coordinator, 727-767-4176
- Recruiting
New York Medical College ( Site 4023)
Valhalla, New York, 10595
Study Coordinator, 914-614-4270
France3 sites, 3 recruiting
- Recruiting
Bordeaux University Hospital - Pellegrin ( Site 4105)
Bordeaux, Aquitaine, 33076
Study Coordinator, +33 5 56 79 56 79
- Recruiting
CENTRE LEON BERARD ( Site 4100)
Lyon, Rhone, 69373
Study Coordinator, +33469166572
- Recruiting
Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes ( Site 4104)
Nantes, Loire-Atlantique, 44093
Study Coordinator, +33240087805
Israel3 sites, 3 recruiting
- Recruiting
Rambam Health Care Campus ( Site 4674)
Haifa, 3109601
Study Coordinator, +97247776755
- Recruiting
Schneider Children's Medical Center ( Site 4676)
Petah Tikva, 4920235
Study Coordinator, +97239253507
- Recruiting
Sheba Medical Center ( Site 4675)
Ramat Gan, 5265601
Study Coordinator, +97235302996
Spain3 sites, 3 recruiting
- Recruiting
Hospital Universitari Vall d Hebron ( Site 4716)
Barcelona, 08035
Study Coordinator, +34934893093
- Recruiting
Hospital Sant Joan de Déu ( Site 4717)
Esplugues de Llobregat, Barcelona, 8950
Study Coordinator, +34671600093
- Recruiting
Hospital Niño Jesús ( Site 4715)
Madrid, Madrid, Comunidad de, 28009
Study Coordinator, +34915035900
South Korea2 sites, 2 recruiting
- Recruiting
Seoul National University Hospital-Pediatrics ( Site 4972)
Seoul, 03080
Study Coordinator, 82220723304
- Recruiting
Asan Medical Center-Pediatrics - Pedicatric Oncology ( Site 4973)
Seoul, 05505
Study Coordinator, 82230105994
Australia1 site, 1 recruiting
- Recruiting
The Royal Children's Hospital ( Site 4994)
Parkville, Victoria, 3052
Study Coordinator, +6139345 4107
Belgium1 site, 1 recruiting
- Recruiting
UZ Gent ( Site 4428)
Ghent, Oost-Vlaanderen, 9000
Study Coordinator, +32 9 332 16 53
Czechia1 site, 1 recruiting
- Recruiting
Fakultní nemocnice Motol a Homolka ( Site 4387)
Prague, 150 06
Study Coordinator, +420224436483
Denmark1 site, 1 recruiting
- Recruiting
Rigshospitalet ( Site 4467)
Copenhagen, Capital Region, DK-2100
Study Coordinator, +4535455002
Hungary1 site, 1 recruiting
- Recruiting
Semmelweis Egyetem ( Site 4838)
Budapest, Pest County, 1094
Study Coordinator, +36 1 215 1380
Netherlands1 site, 1 recruiting
- Recruiting
Prinses Maxima Centrum voor Kinderoncologie ( Site 4510)
Utrecht, 3584 CS
Study Coordinator, +31889727272
Slovakia1 site, 1 recruiting
- Recruiting
Narodny ustav detskych chorob ( Site 4592)
Bratislava, Bratislava Region, 833 40
Study Coordinator, +421259371205
Sweden1 site, 1 recruiting
- Recruiting
Sahlgrenska Universitetssjukhuset ( Site 4634)
Gothenburg, Västra Götaland County, 416 85
Study Coordinator, +46313435865
About this study
Official title: LIGHTBEAM-U01 Substudy 01D: A Phase 1b/2 Substudy to Evaluate the Safety and Efficacy of Ifinatamab Deruxtecan in Pediatric Participants With Relapsed or Refractory Solid Tumors
Researchers are looking for new ways to treat children with relapsed or refractory solid tumors:
- Relapsed means the cancer came back after treatment
- Refractory means the cancer did not respond (get smaller or go away) to treatment
- Solid tumors are cancers mostly in body organs and tissues, not in the blood or other body liquids
Read more
The study treatment I-DXd (also known as MK-2400 or ifinatamab 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 I-DXd and if children younger than 12 years old tolerate it
- How many children who receive I-DXd have the cancer get smaller or go away
Read the detailed description
This study will have 2 parts: Part 1 will evaluate the safety and tolerability and determine the recommended dose for expansion (RDE) of I-DXd, followed by Part 2 an efficacy expansion.
- Conditions
- Malignant Neoplasm
- Being tested
- Ifinatamab Deruxtecan
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ifinatamab DeruxtecanBiological
Also called MK-2400, I-DXd, DS-7300a
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Ifinatamab Deruxtecan | Experimental | Participants receive ifinatamab deruxtecan via intravenous (IV) infusion on day 1 of each 3-week cycle until discontinuation or progression Receives: Biological: Ifinatamab Deruxtecan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience a Dose-limiting Toxicity (DLT)
A DLT is any of a prespecified list of adverse events (AEs) that occur during Cycle 1 (up to 21 days) if attributed to the study treatment and not attributed to any other clearly identifiable cause. The percentage of participants who experience DLTs will be reported. Each cycle is 21 days.
Measured: Cycle 1 (up to approximately 21 days); each cycle is 21 days
Part 1: Number of Participants From ≥1 Month to <12 Years Who Experience One or More Adverse Events (AEs)
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who experience AEs will be reported.
Measured: Up to approximately 5 years
Part 1: Number of Participants From ≥1 Month to <12 Years Who Discontinue Study Intervention Due to an AE
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who discontinue study treatment due to an AE will be reported.
Measured: Up to approximately 5 years
Part 1: Number of Participants From ≥1 Month to <12 Years Who Receive Dose Modifications Due to AEs
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who receive dose modification due to an AE will be reported.
Measured: Up to approximately 5 years
Part 1 and Part 2: Objective Response Rate (ORR) for Participants with neuroblastoma (NBL), rhabdomyosarcoma (RMS), and Wilms tumor (WT)
ORR is defined as the percentage of participants with Complete Response (CR: disappearance of all target lesions) or Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions). The percentage of participants who experience CR or PR as assessed by the investigator will be presented.
Measured: Up to approximately 5 years
Part 1 and Part 2: Disease Control Success at 4 Months (DCS-4) for Participants with osteosarcoma (OST)
DCS-4 is defined as no occurrence of disease progression per disease specific criteria as assessed by investigator or death due to any cause by Month 4 following the first administration of study intervention for participants with OST. Participants who discontinue from study for any reason prior to completing the third post baseline (or at least 16 weeks) response assessments will be considered disease control failures.
Measured: Up to 4 Months
Secondary outcomes (16)
Part 1 and Part 2: Duration of Response (DOR) For Participants With NBL, RMS, WT, or OST
For participants who demonstrate a confirmed CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1, DOR is defined as the time from first documented evidence of CR or PR until PD or death. PD is defined as at least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5 mm. Note: The appearance of one or more new lesions is also considered PD. DOR as assessed by the investigator will be presented.
Measured: Up to approximately 5 years
Part 1 and Part 2: Disease Control Rate (DCR) For Participants With NBL, RMS, WT, or OST
DCR is defined, per RECIST 1.1, as the percentage of participants who have a CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions) or Stable Disease (SD). SD is defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD: at least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5 mm). Note: The appearance of one or more new lesions is also considered PD. The time from the first dose until the date of SD must be greater than or equal to 6 weeks. The DCR as assessed by the investigator will be presented.
Measured: Up to approximately 5 years
Part 1 and Part 2: Time to Response (TTR) For Participants With NBL, RMS, WT, or OST
For participants who demonstrate a confirmed CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1, TTR is defined as the time from the first dose to the first documented evidence of a CR or PR. The TTR as assessed by the investigator will be presented.
Measured: Up to approximately 5 years
Part 1 and Part 2: Progression-free Survival (PFS) For Participants With NBL, RMS, WT, or OST
PFS is defined as the time from randomization to the first documented PD or death due to any cause, whichever occurs first as assessed by RECIST 1.1. PD is defined as at least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5 mm. Note: The appearance of one or more new lesions is also considered PD. PFS as assessed by the investigator will be presented.
Measured: Up to approximately 5 years
Part 1 and Part 2: ORR For participants with OST
ORR is defined as the percentage of participants with Complete Response (CR: disappearance of all target lesions) or Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions). The percentage of participants with OST who experience CR or PR as assessed by the investigator will be presented.
Measured: Up to approximately 5 years
Part 1 and Part 2: Overall Survival (OS)
OS is defined as time from first dose of study treatment to death due to any cause.
Measured: Up to approximately 5 years
Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Experience an AE
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who experience AEs will be reported.
Measured: Up to approximately 5 years
Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Discontinue Study Treatment Due to an AE
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who discontinue study treatment due to an AE will be reported.
Measured: Up to approximately 5 years
Part 1 and Part 2: Number of Participants With NBL, RMS, WT, or OST Who Receive Dose Modification Due to an AE
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The percentage of participants who receive dose modification due to an AE will be reported.
Measured: Up to approximately 5 years
Part 1 and Part 2: Maximum Plasma Concentration (Cmax) of I-Dxd
Blood samples will be collected at specified intervals to determine the Cmax of I-Dxd.
Measured: At designated timepoints (up to approximately 5 years)
Part 1 and Part 2: Area under the concentration time curve from Time 0 to the End of the Dosing Period (AUCtau) of I-Dxd
Blood samples will be collected at specified intervals to determine the AUCtau of I-Dxd.
Measured: At designated timepoints (up to approximately 5 years)
Part 1 and Part 2: Plasma Trough Concentration (Ctrough) of I-Dxd
Blood samples will be collected at specified intervals to determine the Ctrough of I-Dxd.
Measured: At designated timepoints (up to approximately 5 years)
Part 1 and Part 2: Maximum Plasma Concentration (Cmax) of released drug payload (Dxd)
Blood samples will be collected at specified intervals to determine the Cmax of Dxd.
Measured: At designated timepoints (up to approximately 5 years)
Part 1 and Part 2: Area under the concentration time curve from Time 0 to the End of the Dosing Period (AUCtau) of Dxd
Blood samples will be collected at specified intervals to determine the AUCtau of Dxd.
Measured: At designated timepoints (up to approximately 5 years)
Part 1 and Part 2: Plasma Trough Concentration (Ctrough) of Dxd
Blood samples will be collected at specified intervals to determine the Ctrough of Dxd.
Measured: At designated timepoints (up to approximately 5 years)
Part 1 and Part 2: Number of Participants with antidrug antibodies (ADA) against I-Dxd
Blood samples will be collected at specified intervals to assess I-Dxd immunogenicity by determining the incidence of ADA of I-Dxd.
Measured: At designated timepoints (up to approximately 5 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2026
- Study start6 Jul 2026
- Primary completion17 Aug 2031 (estimated)
- Study completion17 Aug 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Malignant NeoplasmStudies of the same condition, open and under way.
- More trials run by Merck Sharp & Dohme LLCThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 134 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Merck Sharp & Dohme LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Daiichi Sankyo
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Merck Sharp & Dohme LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07630974
- Sponsor's protocol ID
- 9999-01D
- Other IDs
- U1111-1322-6561, 2025-522339-32-00, LIGHTBEAM-U01, MK-9999-01D, 2025
- Status last verified
- Oct 2026
- First submitted
- 1 Jun 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: https://trialstransparency.msdclinicaltrials.com/pdf/ProcedureAccessClinicalTrialData.pdf