What side effects are associated with this type of intervention?

The most common treatments for neuroblastoma using anti-GD2 monoclonal antibodies, such as Dinutuximab, are associated with several side effects. These include severe pain, which often requires opioid management, infusion reactions like fever, chills, and hypotension, and capillary leak syndrome. Other notable side effects are peripheral neuropathy, which can cause long-term nerve damage, and hematologic toxicities such as neutropenia. Additionally, patients may experience gastrointestinal symptoms, including nausea and vomiting, and increased risk of infections due to immunosuppression.

What prior FDA approvals exist?

Dinutuximab, an anti-GD2 monoclonal antibody, has significantly improved the treatment of high-risk neuroblastoma in children and is a mainstay in both upfront and relapsed/refractory therapy. Another similar drug, naxitamab, also an anti-GD2 monoclonal antibody, has been FDA approved for administration over shorter infusion times and is used in outpatient settings. These treatments target the GD2 antigen on neuroblastoma cells, enhancing the immune system's ability to attack the cancer.

What other types of research exist?

Promising alternatives to Dinutuximab for neuroblastoma patients include Naxitamab, another anti-GD2 monoclonal antibody, which has a similar toxicity profile and is FDA approved for administration over shorter infusion times. Additionally, novel immunotherapies and targeted therapies such as CAR-T cell therapy and ALK inhibitors (e.g., Lorlatinib) are being explored in clinical trials and have shown potential in treating high-risk neuroblastoma.

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Monoclonal Antibodies

Rapid Infusion of Dinutuximab for Neuroblastoma (RAPID Trial)

Phase 4

Waitlist Available

Led By Sara-Jane Onyeama, MD

Research Sponsored by Children's Hospital Los Angeles

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must have adequate bone marrow function

Patients must have a diagnosis of relapsed, refractory, or persistent high-risk neuroblastoma or ganglioneuroblastoma

Must not have

Patients with a significant intercurrent illness or disease of any major organ system that would impair their ability to withstand protocol therapy are not eligible

Patients who could not tolerate standard dose of dinutuximab infusion in 20 hours or less are not eligible

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 22 of study therapy until day 126

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Approved for 20 Other Conditions

Pivotal Trial

Drug Has Already Been Approved

Summary

This trial aims to test if a cancer-fighting antibody can be given more quickly to children with high-risk neuroblastoma. The goal is to see if faster treatment is safe and effective, which could make it easier and less costly by allowing it to be done outside the hospital.

Who is the study for?

This trial is for children with high-risk neuroblastoma who have previously been treated with dinutuximab, either alone or with chemotherapy. They must not be breastfeeding or pregnant and agree to use contraception if of childbearing potential. Participants need normal organ function, no severe allergies to anti-GD2 antibodies, and cannot be on certain medications that affect the immune system.

What is being tested?

The study tests a rapid infusion method of dinutuximab over four hours or less in children with high-risk neuroblastoma. The goal is to see if this faster delivery can reduce hospital stays and costs while maintaining safety and effectiveness compared to the standard longer infusion times.

What are the potential side effects?

Possible side effects include allergic reactions to the antibody treatment, which could range from mild skin reactions to more serious issues affecting breathing or blood pressure. Other common side effects might involve fever, pain at the injection site, vomiting, diarrhea, low blood pressure, rash, and difficulty breathing.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My bone marrow is functioning well.

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My diagnosis is high-risk neuroblastoma or ganglioneuroblastoma that has come back or not responded to treatment.

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I am at least 1 year old.

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I completed at least 4 rounds of strong chemotherapy for my cancer.

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I have been treated with dinutuximab before.

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My liver is working well.

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My kidneys are working well.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any major illness that would make it hard for me to handle the treatment.

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I cannot tolerate a full dose of dinutuximab in 20 hours or less.

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I am of childbearing age and my pregnancy test is negative.

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I have not been diagnosed with myelodysplastic syndrome or any cancer other than neuroblastoma.

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I have never had a severe allergic reaction to anti-GD2 antibodies.

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I do not have any infections that aren't responding to treatment.

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I am willing to stop breastfeeding if I join the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 22 of study therapy until day 126

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 22 of study therapy until day 126

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 22 of study therapy until day 126 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Determine average dinutuximab infusion time in Cycle 1

Determine feasibility of administering dinutuximab in Cycle 1

Secondary study objectives

Determine average dinutuximab infusion time in Cycles 2-6

Determine feasibility of administering Dinutuximab in Cycle 2-6 in 4 hrs or less

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Approved for 20 Other Conditions

This treatment demonstrated efficacy for 20 other conditions.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Rapid infusion of dinutuximab with chemotherapyExperimental Treatment1 Intervention

Patients will receive chemotherapy and dinutuximab via rapid infusion

0 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Neuroblastoma treatments often include immunotherapy and targeted therapy, which are crucial for improving patient outcomes. Dinutuximab, an anti-GD2 monoclonal antibody, targets the GD2 antigen on neuroblastoma cells, marking them for destruction by the immune system. This mechanism is significant because it enhances the body's natural ability to fight cancer cells, leading to better survival rates. Other treatments, such as chemotherapy, work by killing rapidly dividing cells, but they can also harm healthy cells. Immunotherapy like Dinutuximab offers a more targeted approach, reducing collateral damage and improving the quality of life for patients.

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