High Risk NeuroblastomaPhase 1NCT07688252
Dinutuximab-beta and Chemotherapy in Newly Diagnosed High-Risk Neuroblastoma
A Phase 1 study testing Dinutuximab Beta and Induction chemotherapy in people with High Risk Neuroblastoma, run by Chang Gung Memorial Hospital at 2 sites in Taiwan.
Trial summary
This clinical trial investigates the safety, side effects, and effectiveness of combining an immunotherapy drug called dinutuximab-beta with standard induction chemotherapy for patients newly diagnosed with high-risk neuroblastoma. Read more about this study
- Who is it for?
- Ages 1 year to 30 years
- Conditions the study is for
- High Risk Neuroblastoma
- What is being tested?
- Dinutuximab Beta and Induction chemotherapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesTaiwan
- Study dates
- 1 Jul 2026to 31 Dec 2032
- 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 1 year to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Must be 1 to 30 years of age at the time of initial diagnosis.
- Must have histological verification of neuroblastoma or ganglioneuroblastoma, OR demonstration of neuroblastoma cells in the bone marrow with elevated urinary VMA at the time of initial diagnosis.
- Must have newly diagnosed high-risk neuroblastoma according to the revised COG NBL risk classifier (version 2), defined as meeting at least ONE of the following:
- INRG Stage M: MYCN amplification (> 4-fold increase), OR age 12 to < 18 months without MYCN amplification but with unfavorable histology/DI = 1/SCA, OR age > 18 months regardless of biologic features.
- INRG Stage MS: MYCN amplification, OR age 12 to < 18 months without MYCN amplification but with unfavorable histology/DI = 1/SCA.
- INRG Stage L2: MYCN amplification, OR age 18 months to < 5 years without MYCN amplification but with unfavorable histology, OR age ≥ 5 years without MYCN amplification but with unfavorable histology (undifferentiated or poorly differentiated tumor).
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- INRG Stage L1: Tumor with incomplete resection and MYCN amplification.
- Patients > 18 months of age initially diagnosed with INRG Stage L1, L2, or MS who are subsequently upgraded to high-risk disease.
- Must have had no prior systemic therapy, or have only completed the first cycle of induction chemotherapy under the TPOG N2020-HR protocol.
- Must have measurable disease, defined as at least ONE of the following (Note: Patients with elevated VMA only are NOT eligible):
- Measurable tumor on MRI or CT scan within 3 weeks prior to study entry (≥ 10 mm in at least one dimension) that is MIBG avid or demonstrates increased FDG/FDOPA uptake on PET scan.
- MIBG or FDG/FDOPA PET scan within 2 weeks prior to study entry with positive uptake at a minimum of one site.
- ECOG Performance Status of 0, 1, or 2.
- Adequate organ function, including:
- Renal: Creatinine clearance or estimated GFR ≥ 60 mL/min/1.73 m², or serum creatinine ≤ upper limit of normal (ULN) based on age/gender.
- Liver: Total bilirubin ≤ 1.5 x ULN for age AND SGPT (ALT) ≤ 5.0 x ULN (if liver tumor is present) or ≤ 3.0 x ULN (if no liver tumor).
- CNS: No clinical or radiological evidence of active CNS disease (well-controlled seizures allowed), and CNS toxicity ≤ Grade 2.
- Cardiac: Shortening fraction ≥ 27% or Ejection fraction ≥ 50% by ECHO or gated radionuclide study.
- Pulmonary: No dyspnea at rest, no exercise intolerance, no chronic oxygen requirement, and room air pulse oximetry > 94%.
- Must be enrolled on the TPOG N2020-GD2 protocol with completed informed consent forms, and be willing to proceed to standard therapy of high-risk neuroblastoma with a 10-year follow-up.
Who cannot join
- Participation in other interventional clinical trials within 1 month.
- Inability to obey the trial instructions.
- Patients with bone marrow failure syndromes.
- Current requirement for immunosuppressive medications (e.g., tacrolimus, cyclosporine, systemic corticosteroids for reasons other than prevention/treatment of acute allergic reactions or adrenal replacement therapy).
- Females who are pregnant or breastfeeding (A pregnancy test is required for female patients of childbearing potential).
- Female patients who are pregnant are ineligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential.
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- Lactating females who plan to breastfeed their infants.
- The subject or their partner is planning to conceive
- Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method during study therapy and for two months after the last dose of (ch14.18/CHO) (dinutuximab-β) are not eligible.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Meng-Yao Lu, M.D. contact
Taiwan2 sites, 2 recruiting
- Recruiting
National Taiwan University Hospital
Taipei, Taiwan, 110
Meng Yao Lu, MD, 886-2-2312-3456, lmy1079@gmail.com
Meng-Yao Lu, MD
- Recruiting
Chang Gung Memorial Hospital Linkou Branch
Taoyuan, Taiwan, 333
Shih-Hsiang Chen, MD, 886-3-328-1200, samechen@cgmh.org.tw
Shih-Hsiang Chen, MD
About this study
Official title: Pilot Study of Combining of Dinutuximab-β With Induction Chemotherapy in Newly Diagnosed High Risk Neuroblastoma
This clinical trial investigates the safety, side effects, and effectiveness of combining an immunotherapy drug called dinutuximab-beta with standard induction chemotherapy for patients newly diagnosed with high-risk neuroblastoma. Dinutuximab-beta is an antibody designed to target specific molecules on the surface of neuroblastoma cells.
In this pilot study, enrolled patients will receive dinutuximab-beta as a continuous intravenous infusion alongside a standard 6-cycle induction chemotherapy regimen. The primary goals of this study are to monitor how well patients tolerate the new combination treatment closely and to determine how effectively tumors shrink before patients proceed to the next phase of their standard consolidation therapy.
Read the detailed description
This is a prospective, single-arm, pilot study aiming to evaluate the tolerability, safety, and clinical efficacy of incorporating GD2-directed immunotherapy (Dinutuximab-beta) into the frontline induction chemotherapy for high-risk neuroblastoma.
Patients with newly diagnosed high-risk neuroblastoma will receive Dinutuximab-beta administered as a 10-day continuous intravenous infusion (10 mg/m²/day) during Cycles 2 to 6 of the standard 6-cycle induction chemotherapy. The chemotherapy backbone includes cyclophosphamide, vincristine, doxorubicin/epirubicin, etoposide, and cisplatin.
The study is designed with a safety monitoring phase for the initial cohort to evaluate unacceptable toxicities closely. Following the completion of the 6-cycle induction therapy, patients will be assessed for clinical response based on the revised International Neuroblastoma Response Criteria (INRC) before proceeding to standard consolidation therapy, which includes high-dose chemotherapy with stem cell rescue, radiotherapy, and maintenance therapy. Exploratory evaluations will also be conducted to monitor minimal residual disease (MRD), assess event-free and overall survival, and explore the correlation between immune biomarkers and clinical outcomes.
- Conditions
- High Risk Neuroblastoma
- Being tested
- Dinutuximab Beta, Induction chemotherapy
- Keywords
- Neuroblastoma, High-risk, chemoimmumotherapy, newly-diagnosed, dinutuximab-beta
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dinutuximab BetaBiological
Also called Qarziba
Dinutuximab-beta 10 mg/m²/day administered as a continuous intravenous infusion on Days 0-9 of Cycles 2-6.
Induction chemotherapyDrug
Six cycles of standard induction chemotherapy consisting of cyclophosphamide, vincristine, doxorubicin (or epirubicin), etoposide, and cisplatin administered according to the study treatment schedule.
| Study group | Role | What they receive |
|---|---|---|
| Dinutuximab-Beta with Induction Chemotherapy | Experimental | Participants will receive dinutuximab-beta administered as a continuous intravenous infusion in combination with an induction chemotherapy regimen consisting of cyclophosphamide, vincristine, doxorubicin (or epirubicin), etoposide, and cisplatin. Receives: Biological: Dinutuximab Beta; Drug: Induction chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Unacceptable Toxicities or Toxic Death
Tolerability is assessed by the number of toxic deaths and the number of patients experiencing unacceptable toxicities.
Unacceptable toxicities are assessed according to CTCAE criteria and include:
- toxicity requiring pressors for ≥ 24 hours (e.g., Grade 4 capillary leak syndrome, Grade 4 anaphylaxis/allergic reaction, or Grade 3-4 hypotension),
- toxicity requiring ventilatory support for > 24 hours,
- Grade 4 or unresolved Grade 3 peripheral motor/sensory neuropathy,
- Grade 4 acute infusion reaction.
Measured: During Cycles 2-6 of induction therapy (up to approximately 6 months).
Clinical Response Rate (RR)
Clinical response rate is defined as the percentage of eligible participants who achieve a Partial Response (PR) or better (e.g., Complete Response or Very Good Partial Response). The tumor response will be evaluated using the revised International Neuroblastoma Response Criteria (INRC).
Measured: At the end of induction therapy (up to approximately 6 months).
Secondary outcomes (2)
Event-Free Survival (EFS)
Event-free survival (EFS) is defined as the time from the date of study enrollment to the occurrence of disease relapse or progression, secondary malignancy, or death.
Measured: Up to 10 years from the date of study enrollment.
Overall Survival (OS)
Overall survival (OS) is defined as the time from the date of study enrollment to death from any cause. Patients without death will be censored at the time of last follow-up.
Measured: Up to 10 years from the date of study enrollment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2026
- Study start1 Jul 2026
- Primary completion31 Dec 2032 (estimated)
- Study completion31 Dec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for High Risk NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Chang Gung Memorial HospitalThe 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
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chang Gung Memorial Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Taiwan University Hospital
- Responsible party
- Principal investigator, CHEN, SHIH-HSIANG, Chang Gung Memorial Hospital
- Investigators
- Shih-Hsiang Chen, M.D., Study chair, Chang Gung Memorial HospitalMeng-Yao Lu, M.D., Study chair, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07688252
- Sponsor's protocol ID
- TPOG N2020-GD2
- Status last verified
- Oct 2026
- First submitted
- 30 Jun 2026
- Last updated
- 6 Oct 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 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Combining of Dinutuximab-β With Induction Chemotherapy in Newly Diagnosed High Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT07688252
- Acronym / sponsor ID
- TPOG N2020-GD2
- Conditions
- High Risk Neuroblastoma
- Keywords
- Neuroblastoma, High-risk, chemoimmumotherapy, newly-diagnosed, dinutuximab-beta
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Chang Gung Memorial HospitalUniversity, hospital or other
- Collaborators
- National Taiwan University Hospital
- Study start
- 1 Jul 2026 (actual)
- Primary completion
- 31 Dec 2032 (estimated)
- Study completion
- 31 Dec 2032 (estimated)
- First posted
- 7 Jul 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.
This clinical trial investigates the safety, side effects, and effectiveness of combining an immunotherapy drug called dinutuximab-beta with standard induction chemotherapy for patients newly diagnosed with high-risk neuroblastoma. Dinutuximab-beta is an antibody designed to target specific molecules on the surface of neuroblastoma cells.
In this pilot study, enrolled patients will receive dinutuximab-beta as a continuous intravenous infusion alongside a standard 6-cycle induction chemotherapy regimen. The primary goals of this study are to monitor how well patients tolerate the new combination treatment closely and to determine how effectively tumors shrink before patients proceed to the next phase of their standard consolidation therapy.
Detailed description
This is a prospective, single-arm, pilot study aiming to evaluate the tolerability, safety, and clinical efficacy of incorporating GD2-directed immunotherapy (Dinutuximab-beta) into the frontline induction chemotherapy for high-risk neuroblastoma.
Patients with newly diagnosed high-risk neuroblastoma will receive Dinutuximab-beta administered as a 10-day continuous intravenous infusion (10 mg/m²/day) during Cycles 2 to 6 of the standard 6-cycle induction chemotherapy. The chemotherapy backbone includes cyclophosphamide, vincristine, doxorubicin/epirubicin, etoposide, and cisplatin.
The study is designed with a safety monitoring phase for the initial cohort to evaluate unacceptable toxicities closely. Following the completion of the 6-cycle induction therapy, patients will be assessed for clinical response based on the revised International Neuroblastoma Response Criteria (INRC) before proceeding to standard consolidation therapy, which includes high-dose chemotherapy with stem cell rescue, radiotherapy, and maintenance therapy. Exploratory evaluations will also be conducted to monitor minimal residual disease (MRD), assess event-free and overall survival, and explore the correlation between immune biomarkers and clinical outcomes.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dinutuximab-Beta with Induction Chemotherapy | Experimental | Biological: Dinutuximab Beta, Drug: Induction chemotherapyParticipants will receive dinutuximab-beta administered as a continuous intravenous infusion in combination with an induction chemotherapy regimen consisting of cyclophosphamide, vincristine, doxorubicin (or epirubicin), etoposide, and cisplatin. |
Who may qualify
- Ages
- 1 year to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (20 listed)
- Must be 1 to 30 years of age at the time of initial diagnosis.
- Must have histological verification of neuroblastoma or ganglioneuroblastoma, OR demonstration of neuroblastoma cells in the bone marrow with elevated urinary VMA at the time of initial diagnosis.
- Must have newly diagnosed high-risk neuroblastoma according to the revised COG NBL risk classifier (version 2), defined as meeting at least ONE of the following:
- Must have had no prior systemic therapy, or have only completed the first cycle of induction chemotherapy under the TPOG N2020-HR protocol.
- Must have measurable disease, defined as at least ONE of the following (Note: Patients with elevated VMA only are NOT eligible):
Key exclusion criteria (9 listed)
- Participation in other interventional clinical trials within 1 month.
- Inability to obey the trial instructions.
- Patients with bone marrow failure syndromes.
- Current requirement for immunosuppressive medications (e.g., tacrolimus, cyclosporine, systemic corticosteroids for reasons other than prevention/treatment of acute allergic reactions or adrenal replacement therapy).
- Females who are pregnant or breastfeeding (A pregnancy test is required for female patients of childbearing potential).
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 |
|---|---|---|
| Dinutuximab BetaAlso: QarzibaDinutuximab-beta 10 mg/m²/day administered as a continuous intravenous infusion on Days 0-9 of Cycles 2-6. | Biological | Dinutuximab-Beta with Induction Chemotherapy |
| Induction chemotherapySix cycles of standard induction chemotherapy consisting of cyclophosphamide, vincristine, doxorubicin (or epirubicin), etoposide, and cisplatin administered according to the study treatment schedule. | Drug | Dinutuximab-Beta with Induction Chemotherapy |
Primary outcome
Number of Participants with Unacceptable Toxicities or Toxic Death
Time frame: During Cycles 2-6 of induction therapy (up to approximately 6 months).
Tolerability is assessed by the number of toxic deaths and the number of patients experiencing unacceptable toxicities. Unacceptable toxicities are assessed according to CTCAE criteria and include: 1. toxicity requiring pressors for ≥ 24 hours (e.g., Grade 4 capillary leak syndrome, Grade 4 anaphylaxis/allergic reaction, or Grade 3-4 hypotension), 2. toxicity requiring ventilatory support for > 24 hours, 3. Grade 4 or unresolved Grade 3 peripheral motor/sensory neuropathy, 4. Grade 4 acute infusion reaction.
Clinical Response Rate (RR)
Time frame: At the end of induction therapy (up to approximately 6 months).
Clinical response rate is defined as the percentage of eligible participants who achieve a Partial Response (PR) or better (e.g., Complete Response or Very Good Partial Response). The tumor response will be evaluated using the revised International Neuroblastoma Response Criteria (INRC).
2 secondary outcomes
Event-Free Survival (EFS)
Time frame: Up to 10 years from the date of study enrollment.
Overall Survival (OS)
Time frame: Up to 10 years from the date of study enrollment.
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 6 Oct 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.
Meng-Yao Lu, M.D. contact
Shih-Hsiang Chen, M.D. study chair
Chang Gung Memorial Hospital
Meng-Yao Lu, M.D. study chair
National Taiwan University Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Dinutuximab Beta and Induction chemotherapy in people with High Risk Neuroblastoma, run by Chang Gung Memorial Hospital at 2 sites in Taiwan.
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 20 inclusion and 9 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 6 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Taiwan. 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 Shih-Hsiang Chen, M.D. (+886-3-328-1200, samechen@cgmh.org.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Unacceptable Toxicities or Toxic Death (time frame: During Cycles 2-6 of induction therapy (up to approximately 6 months).). The study lists 2 primary outcomes in all. 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 6 Oct 2026.