Patritumab Deruxtecan for Cancer

Recruiting at 30 trial locations

Overseen By Toll Free Number

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Merck Sharp & Dohme LLC

Disqualifiers: Interstitial lung disease, Organ transplant, CNS metastases, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 2

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called HER3-DXd (also known as Patritumab Deruxtecan) for children whose liver cancer (hepatoblastoma) or muscle cancer (RMS) has returned after treatment or did not respond initially. The main goal is to determine if this treatment is safe and effective in shrinking or eliminating the cancer. HER3-DXd is a specialized medicine that targets cancer cells directly. Children eligible for this trial have already tried other treatments without success and lack other available options. As a Phase 1, Phase 2 trial, this study aims to understand how HER3-DXd works in children and measure its effectiveness in an initial, smaller group, offering hope for those without other treatment options.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Previous studies have identified some safety concerns with patritumab deruxtecan. The most common serious side effects reported were low white blood cell count in 26% of patients, tiredness in 17%, and low platelet count in 15%. While important to consider, these side effects are not unusual for cancer treatments.

In another study, patients received the treatment for an average of about 251 days, suggesting that many could continue with it for some time. This indicates that the side effects might have been manageable for those patients.

Researchers continue to study this treatment, as it is not yet fully proven to be safe or effective for everyone. They are carefully monitoring its effects to ensure the benefits outweigh the risks. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Patritumab Deruxtecan is unique because it combines an antibody with a cancer-killing agent, acting like a guided missile to target and destroy cancer cells specifically. Unlike traditional chemotherapy, which affects both healthy and cancerous cells, this treatment is designed to minimize damage to normal cells, potentially leading to fewer side effects. Researchers are excited about its targeted approach and the potential for more effective treatment outcomes, especially for patients who have not responded well to existing therapies.

What evidence suggests that patritumab deruxtecan might be an effective treatment for hepatoblastoma or rhabdomyosarcoma?

Research has shown that patritumab deruxtecan, also known as HER3-DXd, effectively treats other types of cancer. In patients with non-small cell lung cancer, the cancer remained stable for about 5.5 months on average, and patients lived for about 15.2 months after starting treatment. This treatment targets a specific protein on cancer cells and delivers a drug to help destroy them. Although information is limited for hepatoblastoma or rhabdomyosarcoma, the treatment's mechanism suggests potential effectiveness for these cancers. Participants in this trial will receive patritumab deruxtecan via IV infusion on Day 1 of each 3-week cycle until discontinuation or progression. ^{3 4 6 7 8}

Who Is on the Research Team?

Medical Director

Principal Investigator

Merck Sharp & Dohme LLC

Are You a Good Fit for This Trial?

This trial is for children with specific types of cancer: hepatoblastoma (a liver cancer) or rhabdomyosarcoma (RMS, a muscle cancer), which have either returned after treatment or didn't respond to previous treatments. Participants must have tried at least one systemic treatment and not be eligible for standard options. They should also be recovered from any serious effects of past cancer therapies.

Inclusion Criteria

The main

I had hepatitis C but my viral load is now undetectable.

My cancer is either advanced Rhabdomyosarcoma or Hepatoblastoma.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Safety Lead-in

Participants receive patritumab deruxtecan to demonstrate a tolerable safety profile and confirm a preliminary recommended phase 2 dose

3 weeks per cycle

1 visit per cycle (in-person)

Efficacy Evaluation

Participants continue to receive patritumab deruxtecan to evaluate the efficacy of the treatment

3 weeks per cycle, up to approximately 5 years

1 visit per cycle (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Patritumab Deruxtecan

Trial Overview The study tests HER3-DXd, also known as MK-1022 or patritumab deruxtecan, an antibody-drug conjugate designed to target and destroy cancer cells in children. The aim is to assess its safety, how it's processed by the body over time, and its effectiveness in reducing or eliminating the targeted cancers.

How Is the Trial Designed?

Group I: Patritumab Deruxtecan Experimental Treatment 1 Intervention

Participants receive patritumab deruxtecan via IV infusion on Day 1 of each 3-week cycle until discontinuation or progression.

Who Is Running the Clinical Trial?

Merck Sharp & Dohme LLC

Lead Sponsor

Trials

4,096

Recruited

5,232,000+

Chirfi Guindo

Merck Sharp & Dohme LLC

Chief Marketing Officer since 2022

Degree in Engineering from Ecole Centrale de Paris, MBA from New York University Stern School of Business

Robert M. Davis

Merck Sharp & Dohme LLC

Chief Executive Officer since 2021

JD from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University

Daiichi Sankyo

Industry Sponsor

Trials

443

Recruited

493,000+

Hiroyuki Okuzawa

Daiichi Sankyo

Chief Executive Officer

Degree in Social Sciences from Hitotsubashi University

Yuki Abe

Daiichi Sankyo

Chief Medical Officer since 2023

MD

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40554742/

MK-1022) in Non-Small Cell Lung Cancer After Platinum- ... The median progression-free survival was 5.5 (95% CI, 4.0 to 11.2) months, and the median overall survival was 15.2 (95% CI, 10.8 to 17.7) ...

2. ascopubs.org ascopubs.org/doi/10.1200/JCO-24-02744

Patritumab Deruxtecan (HER3-DXd; MK-1022) in Non– ... The median progression-free survival was 5.5 (95% CI, 4.0 to 11.2) months, and the median overall survival was 15.2 (95% CI, 10.8 to 17.7) ...

3. nature.com nature.com/articles/s41591-025-03885-3

Patritumab deruxtecan in HR+HER2− advanced breast ... Among the six patients with available data on ILD duration, two cases resolved within approximately 3 weeks, two cases within 1 month and two ...

4. merck.com merck.com/news/patritumab-deruxtecan-demonstrated-statistically-significant-improvement-in-progression-free-survival-versus-doublet-chemotherapy-in-patients-with-locally-advanced-or-metastatic-egfr-mutated-non-small/

Patritumab Deruxtecan Demonstrated Statistically ... Daiichi Sankyo and Merck's patritumab deruxtecan demonstrates a statistically significant progression-free survival improvement in this EGFR-mutated non-small ...

5. targetedonc.com targetedonc.com/view/patritumab-deruxtecan-falls-short-in-egfrm-nsclc

Patritumab Deruxtecan Falls Short in EGFRm NSCLC Additional Efficacy Data​​ The median progression-free survival (PFS) was 5.8 months with patritumab deruxtecan vs 5.4 months with chemotherapy ( ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9401524/

Efficacy and Safety of Patritumab Deruxtecan (HER3-DXd) in ... 1) was 39% [95% confidence interval (CI), 26.0–52.4], and median progression-free survival was 8.2 (95% CI, 4.4–8.3) months. Responses were observed in patients ...

7. ascopubs.org ascopubs.org/doi/10.1200/JCO.23.01476

HERTHENA-Lung01, a Phase II Trial of Patritumab ... Confirmed ORR by BICR was 29.8% (95% CI, 23.9 to 36.2); median duration of response, 6.4 months; median progression-free survival, 5.5 months; ...

8. jto.org jto.org/article/S1556-0864(22)01660-4/fulltext

PP01.48 Efficacy and Safety of Patritumab Deruxtecan ... The most common grade ≥3 treatment-emergent adverse events (TEAEs) were neutropenia (26%), fatigue (17%), and thrombocytopenia (15%). Drug-related interstitial ...