Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M)Phase 1MRG003NCT07584499
Phase I Study of Becotatug Vedotin for Safety and Efficacy in EGFR-Positive Pediatric Relapsed/Refractory or Metastatic Solid Tumors
A Phase 1 study testing Becotatug Vedotin in people with Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M) and Nasopharyngeal Carcinoma, run by Sun Yat-sen University at 1 site in China.
Trial summary
There is a significant unmet medical need for effective therapies for pediatric relapsed/refractory solid tumors. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes, and adult Phase I data of Becotatug Vedotin demonstrated a manageable safety profile and promising antitumor activity in… Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M), Nasopharyngeal Carcinoma, Lymphoepithelial Carcinoma
- What is being tested?
- Becotatug Vedotin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 51 participants
- Where
- 1 siteChina
- Study dates
- 30 May 2026to 30 May 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- All participants must meet all of the following criteria to be eligible for enrollment:
- Informed Consent: The patient (and/or legal guardian, as age-appropriate) fully understands the study, voluntarily agrees to participate, and signs a written informed consent form (ICF). A separate biomarker consent form is required for EGFR testing prior to screening.
- Age: 2 to 18 years old at the time of consent. Life Expectancy: Estimated overall survival of at least 3 months.
- Histologically Confirmed Disease: Pathologically confirmed relapsed/refractory or metastatic EGFR-positive solid tumor, belonging to one of the following subtypes:
- Head and neck squamous cell carcinoma, nasopharyngeal carcinoma, or lymphoepithelial carcinoma that progressed during or after at least one line of platinum-based chemotherapy and PD-1/PD-L1 inhibitor therapy Rhabdomyosarcoma Neuroblastoma Medulloblastoma Wilms tumor Atypical teratoid/rhabdoid tumors (AT/RTs) Diffuse intrinsic pontine gliomas (DIPGs) Other EGFR-positive solid tumor subtypes deemed eligible by the investigator Measurable Disease: At least one measurable tumor lesion by computed tomography (CT) or magnetic resonance imaging (MRI) per RECIST v1.1 criteria (longest diameter ≥10 mm; pathological lymph node short axis ≥15 mm).
- Performance Status: Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
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- Adequate Bone Marrow Function:
- Absolute neutrophil count (ANC) ≥1.5 × 10⁹/L Platelet count ≥75 × 10⁹/L Hemoglobin ≥80 g/L Exception for patients with bone marrow involvement: ANC ≥1.0 × 10⁹/L, platelets ≥50 × 10⁹/L, hemoglobin ≥75 g/L
- Adequate Hepatic and Renal Function:
- Serum creatinine ≤1.5 × upper limit of normal (ULN) Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 × ULN Total bilirubin ≤1.5 × ULN Exception for patients with liver involvement: AST/ALT ≤5 × ULN, total bilirubin ≤3 × ULN
Who cannot join
- Participants will be excluded from the study if they meet any of the following criteria:
- Hypersensitivity: Known hypersensitivity to any component of Becotatug Vedotin (MRG003) or its excipients.
- Symptomatic CNS Metastases: Presence of symptomatic central nervous system (CNS) metastases.
- Prior Malignancies: History of other primary malignant tumors, except for:
- Locally excised basal cell or squamous cell carcinoma of the skin Cervical carcinoma in situ Any prior malignancy that has been in complete remission for ≥3 years without treatment Note: Melanoma (any stage) is explicitly excluded
- Significant Liver Disease: Clinically significant liver disease, including:
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- Positive hepatitis C virus (HCV) antibody Chronic active hepatitis B (HBV DNA >20,000 IU/mL) HIV Infection: Known human immunodeficiency virus (HIV) infection. Severe Ocular Abnormalities: History of severe ophthalmologic conditions, such as severe dry eye syndrome or exposure keratitis.
- Uncontrolled Systemic Diseases: Severe or uncontrolled medical conditions, including:
- Interstitial lung disease or pneumonitis Active autoimmune diseases requiring systemic immunosuppressive therapy
- Cardiac Disease: Clinically significant cardiac dysfunction or cardiac disease, including:
- Congestive heart failure (New York Heart Association Class ≥II) Uncontrolled arrhythmias QTc interval prolongation >450 ms (males) or >470 ms (females) Recent Antitumor Therapy: Received any systemic antitumor therapy (chemotherapy, biological therapy, immunotherapy, targeted therapy) within 3 weeks prior to the first dose of study drug, and have not recovered to CTCAE v4.03 Grade ≤1 (except alopecia).
- Recent Major Surgery: Underwent major surgical procedure within 3 weeks prior to the first dose of study drug.
- Planned Surgery: Planned surgical procedure during the study period, or any surgery deemed necessary by the investigator.
- Prior EGFR Therapy Toxicity: History of severe skin toxicity caused by prior EGFR-targeted therapy, or chronic skin disease requiring ongoing oral or intravenous treatment.
- Other Significant Risks: Any other concurrent medical condition that, in the investigator's judgment, would increase the risk of toxicity or compromise the patient's ability to complete the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong, 510060
Yi Que, 020-87342460, queyi@sysucc.org.cn
Yizhuo Zhang
About this study
Official title: A Phase I Clinical Study to Explore the Safety and Efficacy of Becotatug Vedotin in Pediatric Patients With EGFR-Positive Relapsed/Refractory or Metastatic Solid Tumors
There is a significant unmet medical need for effective therapies for pediatric relapsed/refractory solid tumors. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes, and adult Phase I data of Becotatug Vedotin demonstrated a manageable safety profile and promising antitumor activity in EGFR-positive advanced solid tumors.This is a multicenter, non-randomized, single-arm, open-label Phase I clinical trial sponsored by Sun Yat-sen University Cancer Center (SYSUCC). The trial evaluates the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary efficacy of Becotatug Vedotin-an EGFR-targeted antibody-drug conjugate (ADC)-in pediatric patients with EGFR-positive relapsed/refractory or metastatic solid tumors.
Read the detailed description
Pediatric relapsed/refractory solid tumors represent a major unmet medical need, with conventional chemotherapy and immunotherapy showing limited efficacy and significant toxicities in this population. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes. Becotatug Vedotin consists of a humanized anti-EGFR monoclonal antibody conjugated to monomethyl auristatin E (MMAE), a potent microtubule inhibitor, via a cleavable valine-citrulline linker. Adult Phase I data demonstrated a manageable safety profile and promising antitumor activity in EGFR-positive advanced solid tumors, with an objective response rate (ORR) of 29% in nasopharyngeal carcinoma and 31% in head and neck squamous cell carcinoma. This trial extends these findings to the pediatric population.The trial evaluates the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary efficacy of Becotatug Vedotin-an EGFR-targeted antibody-drug conjugate (ADC)-in pediatric patients with EGFR-positive relapsed/refractory or metastatic solid tumors.
- Conditions
- Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M), Nasopharyngeal Carcinoma, Lymphoepithelial Carcinoma, Rhabdomyosarcoma, Neuroblastoma, Medulloblastoma
- Being tested
- Becotatug Vedotin
- Medical subject headings
- Squamous Cell Carcinoma of Head and Neck, Recurrence, Neoplasm Metastasis, Nasopharyngeal Carcinoma, Rhabdomyosarcoma, Neuroblastoma, Medulloblastoma, Wilms Tumor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Becotatug VedotinDrug
Study Drug: Becotatug Vedotin (MRG003) for Injection (lyophilized powder, 20 mg/vial) Route: Intravenous (IV) infusion over 30 minutes to 3 hours Schedule: Every 3 weeks (Q3W) on Day 1 of each 21-day cycle Maximum Treatment Duration: Up to 8 cycles (24 weeks); patients with confirmed clinical benefit (objective response or stable disease) may continue treatment beyond 8 cycles until disease progression, unacceptable toxicity, withdrawal of consent, or study termination
| Study group | Role | What they receive |
|---|---|---|
| Becotatug Vedotin (MRG003) Phase I Dose-Escalation & Expansion in EGFR-Positive Pediatric R/M Solid | Experimental | This is a single-arm, open-label study where all enrolled participants receive Becotatug Vedotin (MRG003), a novel EGFR-targeted antibody-drug conjugate (ADC), as monotherapy.
Intervention Details Study Drug: Becotatug Vedotin (MRG003) for Injection Route of Administration: Intravenous (IV) infusion over 30 minutes to 3 hours Dosing Schedule: Every 3 weeks (Q3W) on Day 1 of each 21-day cycle Maximum Treatment Duration: Up to 8 cycles (24 weeks)
Dose Levels:
Ia Dose-Escalation Phase: 4 planned dose levels (1.0, 1.5, 2.0, 2.3 mg/kg) Starting dose: 1.0 mg/kg (modified accelerated titration design, 1 patient per cohort) Subsequent doses: Standard 3+3 design Ib Dose-Expansion Phase: All patients receive the determined Recommended Phase II Dose (RP2D) Receives: Drug: Becotatug Vedotin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose-limiting toxicities
DLTs are defined as treatment-related adverse events or laboratory abnormalities (excluding hypersensitivity reactions) that meet CTCAE v4.03 grade 3-4 criteria and occur during the first 21 days of treatment, including:
- Grade 4 neutropenia lasting >7 days
- Grade 3 thrombocytopenia lasting >7 days or any grade 4 thrombocytopenia
- Grade 3 febrile neutropenia requiring antibiotic therapy
- Grade 3-4 hepatic, renal, cardiovascular, ocular, or neurological toxicities
- Uncontrolled grade 3-4 skin, gastrointestinal, or other systemic toxicities
Measured: First 21-day treatment cycle (Cycle 1)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2026
- Study start30 May 2026 (estimated)
- Primary completion30 May 2027 (estimated)
- Study completion30 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M)Studies of the same condition, open and under way.
- More trials run by Sun Yat-sen UniversityThe 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 51 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sun Yat-sen University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yizhuo Zhang, Sun Yat-sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07584499
- Sponsor's protocol ID
- MRG003-pediatirc-001
- Other IDs
- Sun Yat-sen University Cancer
- Acronym
- MRG003
- Status last verified
- May 2026
- First submitted
- 7 May 2026
- Last updated
- 13 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Clinical Study to Explore the Safety and Efficacy of Becotatug Vedotin in Pediatric Patients With EGFR-Positive Relapsed/Refractory or Metastatic Solid Tumors
- ClinicalTrials.gov ID
- NCT07584499
- Acronym / sponsor ID
- MRG003 / MRG003-pediatirc-001
- Conditions
- Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M), Nasopharyngeal Carcinoma, Lymphoepithelial Carcinoma, Rhabdomyosarcoma, Neuroblastoma, Medulloblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 51 participants (estimated)
- Lead sponsor
- Sun Yat-sen UniversityUniversity, hospital or other
- Study start
- 30 May 2026 (estimated)
- Primary completion
- 30 May 2027 (estimated)
- Study completion
- 30 May 2028 (estimated)
- First posted
- 13 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
There is a significant unmet medical need for effective therapies for pediatric relapsed/refractory solid tumors. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes, and adult Phase I data of Becotatug Vedotin demonstrated a manageable safety profile and promising antitumor activity in EGFR-positive advanced solid tumors.This is a multicenter, non-randomized, single-arm, open-label Phase I clinical trial sponsored by Sun Yat-sen University Cancer Center (SYSUCC). The trial evaluates the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary efficacy of Becotatug Vedotin-an EGFR-targeted antibody-drug conjugate (ADC)-in pediatric patients with EGFR-positive relapsed/refractory or metastatic solid tumors.
Detailed description
Pediatric relapsed/refractory solid tumors represent a major unmet medical need, with conventional chemotherapy and immunotherapy showing limited efficacy and significant toxicities in this population. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes. Becotatug Vedotin consists of a humanized anti-EGFR monoclonal antibody conjugated to monomethyl auristatin E (MMAE), a potent microtubule inhibitor, via a cleavable valine-citrulline linker. Adult Phase I data demonstrated a manageable safety profile and promising antitumor activity in EGFR-positive advanced solid tumors, with an objective response rate (ORR) of 29% in nasopharyngeal carcinoma and 31% in head and neck squamous cell carcinoma. This trial extends these findings to the pediatric population.The trial evaluates the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary efficacy of Becotatug Vedotin-an EGFR-targeted antibody-drug conjugate (ADC)-in pediatric patients with EGFR-positive relapsed/refractory or metastatic solid tumors.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Becotatug Vedotin (MRG003) Phase I Dose-Escalation & Expansion in EGFR-Positive Pediatric R/M Solid | Experimental | Drug: Becotatug VedotinThis is a single-arm, open-label study where all enrolled participants receive Becotatug Vedotin (MRG003), a novel EGFR-targeted antibody-drug conjugate (ADC), as monotherapy. Intervention Details Study Drug: Becotatug Vedotin (MRG003) for Injection Route of Administration: Intravenous (IV) infusion over 30 minutes to 3 hours Dosing Schedule: Every 3 weeks (Q3W) on Day 1 of each 21-day cycle Maximum Treatment Duration: Up to 8 cycles (24 weeks) Dose Levels: Ia Dose-Escalation Phase: 4 planned dose levels (1.0, 1.5, 2.0, 2.3 mg/kg) Starting dose: 1.0 mg/kg (modified accelerated titration design, 1 patient per cohort) Subsequent doses: Standard 3+3 design Ib Dose-Expansion Phase: All patients receive the determined Recommended Phase II Dose (RP2D) |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- All participants must meet all of the following criteria to be eligible for enrollment:
- Informed Consent: The patient (and/or legal guardian, as age-appropriate) fully understands the study, voluntarily agrees to participate, and signs a written informed consent form (ICF). A separate biomarker consent form is required for EGFR testing prior to screening.
- Age: 2 to 18 years old at the time of consent. Life Expectancy: Estimated overall survival of at least 3 months.
- Histologically Confirmed Disease: Pathologically confirmed relapsed/refractory or metastatic EGFR-positive solid tumor, belonging to one of the following subtypes:
- Head and neck squamous cell carcinoma, nasopharyngeal carcinoma, or lymphoepithelial carcinoma that progressed during or after at least one line of platinum-based chemotherapy and PD-1/PD-L1 inhibitor therapy Rhabdomyosarcoma Neuroblastoma Medulloblastoma Wilms tumor Atypical teratoid/rhabdoid tumors (AT/RTs) Diffuse intrinsic pontine gliomas (DIPGs) Other EGFR-positive solid tumor subtypes deemed eligible by the investigator Measurable Disease: At least one measurable tumor lesion by computed tomography (CT) or magnetic resonance imaging (MRI) per RECIST v1.1 criteria (longest diameter ≥10 mm; pathological lymph node short axis ≥15 mm).
Key exclusion criteria (15 listed)
- Participants will be excluded from the study if they meet any of the following criteria:
- Hypersensitivity: Known hypersensitivity to any component of Becotatug Vedotin (MRG003) or its excipients.
- Symptomatic CNS Metastases: Presence of symptomatic central nervous system (CNS) metastases.
- Locally excised basal cell or squamous cell carcinoma of the skin Cervical carcinoma in situ Any prior malignancy that has been in complete remission for ≥3 years without treatment Note: Melanoma (any stage) is explicitly excluded
- Positive hepatitis C virus (HCV) antibody Chronic active hepatitis B (HBV DNA >20,000 IU/mL) HIV Infection: Known human immunodeficiency virus (HIV) infection. Severe Ocular Abnormalities: History of severe ophthalmologic conditions, such as severe dry eye syndrome or exposure keratitis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Becotatug VedotinStudy Drug: Becotatug Vedotin (MRG003) for Injection (lyophilized powder, 20 mg/vial) Route: Intravenous (IV) infusion over 30 minutes to 3 hours Schedule: Every 3 weeks (Q3W) on Day 1 of each 21-day cycle Maximum Treatment Duration: Up to 8 cycles (24 weeks); patients with confirmed clinical benefit (objective response or stable disease) may continue treatment beyond 8 cycles until disease progression, unacceptable toxicity, withdrawal of consent, or study termination | Drug | Becotatug Vedotin (MRG003) Phase I Dose-Escalation & Expansion in EGFR-Positive Pediatric R/M Solid |
Primary outcome
Incidence of dose-limiting toxicities
Time frame: First 21-day treatment cycle (Cycle 1)
DLTs are defined as treatment-related adverse events or laboratory abnormalities (excluding hypersensitivity reactions) that meet CTCAE v4.03 grade 3-4 criteria and occur during the first 21 days of treatment, including: * Grade 4 neutropenia lasting >7 days * Grade 3 thrombocytopenia lasting >7 days or any grade 4 thrombocytopenia * Grade 3 febrile neutropenia requiring antibiotic therapy * Grade 3-4 hepatic, renal, cardiovascular, ocular, or neurological toxicities * Uncontrolled grade 3-4 skin, gastrointestinal, or other systemic toxicities
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Becotatug Vedotin in people with Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M) and Nasopharyngeal Carcinoma, run by Sun Yat-sen University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yizhuo Zhang (020-87342460, zhangyzh@sysucc.org.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of dose-limiting toxicities (time frame: First 21-day treatment cycle (Cycle 1)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2026.