Rapid Infusion of Dinutuximab for Neuroblastoma

(RAPID Trial)

Overseen By Araz Marachelian, MD, MS

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: Children's Hospital Los Angeles

Must not be taking: Enzyme-inducing anticonvulsants, CYP3A4 inducers

Disqualifiers: Pregnancy, Uncontrolled hypertension, Myelodysplastic syndrome, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 24

11 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a quicker method to administer dinutuximab, a treatment for high-risk neuroblastoma in children. Typically, the treatment requires 10-20 hours over four days in a hospital setting. The trial aims to determine if it can be safely administered in four hours or less, potentially allowing patients to receive it outside the hospital, thus saving time and resources. Children previously treated for high-risk neuroblastoma and who have received dinutuximab may be suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding the treatment's effects in people, providing an opportunity to contribute to early-stage research.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop all current medications, but some medications may need to be paused during the study. Anti-cancer agents not known to lower blood counts can be used with approval but must be held during the study treatment. Also, certain steroids and anticonvulsants must be stopped before enrollment.

Is there any evidence suggesting that rapid infusion of dinutuximab is likely to be safe for children with high-risk neuroblastoma?

Research shows that dinutuximab, a treatment for high-risk neuroblastoma, is generally well-tolerated when administered quickly to adults. It has been effective with infusion times of less than two hours. This suggests that shorter infusion times might also be safe for children. However, dinutuximab can cause serious side effects, including strong reactions during infusion and nerve pain. In earlier studies with children, some experienced low potassium or sodium levels during treatment. Despite these concerns, dinutuximab remains an important option for treating neuroblastoma. Its approval for use in other situations provides a better understanding of its safety. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the rapid infusion of dinutuximab for neuroblastoma because it offers a potentially quicker and more efficient way to deliver treatment. Unlike standard therapies that often involve prolonged infusions, this method significantly cuts down the time needed for administration, which can improve patient comfort and convenience. Additionally, dinutuximab is an antibody that specifically targets GD2, a molecule found on neuroblastoma cells, offering a focused attack on the cancer while sparing healthy cells. This targeted approach could lead to fewer side effects and improved outcomes compared to conventional chemotherapy regimens.

What evidence suggests that rapid infusion of dinutuximab is effective for high-risk neuroblastoma?

Research has shown that dinutuximab, a type of medicine, greatly improves treatment for children with high-risk neuroblastoma, a kind of cancer. Studies indicate it works well both as an initial treatment and when the cancer returns or doesn't fully respond to other treatments. Dinutuximab has been safely used in children and is now an important part of therapy for this condition. In this trial, participants will receive a rapid infusion of dinutuximab alongside chemotherapy. Evidence from similar treatments, such as naxitamab, suggests that dinutuximab might also be administered more quickly in the future. Overall, dinutuximab is a proven and effective treatment for high-risk neuroblastoma, with the potential for quicker administration. ^{3 6 7 8 9}

Who Is on the Research Team?

Sara-Jane Onyeama, MD

Principal Investigator

Children's Hospital Los Angeles

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

My heart is functioning well.

My brain functions are normal.

My bone marrow is functioning well.

Exclusion Criteria

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

My partner and I agree to use birth control.

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Cycle 1

Participants receive rapid infusion of dinutuximab with chemotherapy

3 weeks

4 visits (in-person)

Treatment Cycles 2-6

Participants continue to receive rapid infusion of dinutuximab with chemotherapy

15 weeks

20 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Dinutuximab

Trial Overview 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.

How Is the Trial Designed?

Group I: Rapid infusion of dinutuximab with chemotherapy Experimental Treatment 1 Intervention

Patients will receive chemotherapy and dinutuximab via rapid infusion

Dinutuximab is already approved in United States, European Union for the following indications:

Approved in United States as Unituxin for:

• Neuroblastoma

Approved in European Union as Dinutuximab for:

• High-risk neuroblastoma

Who Is Running the Clinical Trial?

Children's Hospital Los Angeles

Lead Sponsor

Trials

257

Recruited

5,075,000+

United Therapeutics

Industry Sponsor

Trials

112

Recruited

14,500+

Dr. Martine Rothblatt

United Therapeutics

Chief Executive Officer since 1996

PhD in Medical Ethics from the Royal London College of Medicine and Dentistry, JD and MBA from UCLA

Dr. Michael Benkowitz

United Therapeutics

Chief Medical Officer since 2023

MD from Harvard Medical School

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05421897

Rapid Administration Pilot for Infusing Dinutuximab Studies have shown that the anti-GD2 human-mouse chimeric monoclonal antibody dinutuximab has contributed significantly to the improvement of treatment for ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12471212/

Dinutuximab Beta for the Treatment of High-Risk Neuroblastoma Conclusions: Dinutuximab beta was an effective immunotherapy for patients with HR-NB in routine clinical practice, with a generally manageable ...

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.e22000

Efficacy and safety of dinutuximab in the management ... High-risk neuroblastoma (HR-NB) has a poor prognosis despite a number of treatment strategies. Dinutuximab, an anti-GD2 monoclonal antibody ...

4. nature.com nature.com/articles/s41467-025-56619-x

The anti-GD2 monoclonal antibody naxitamab plus GM ... Naxitamab demonstrated clinically meaningful efficacy with manageable safety in patients with residual neuroblastoma in bone/BM.

5. journals.lww.com journals.lww.com/md-journal/fulltext/2022/01280/dinutuximab_beta_in_the_treatment_of_high_risk.54.aspx

Dinutuximab beta in the treatment of high-risk neuroblastoma Dinutuximab beta is an effective immunotherapy for patients with high-risk neuroblastoma in routine clinical practice when coupled with optimal supportive ...

6. aetna.com aetna.com/cpb/medical/data/800_899/0895.html

Dinutuximab (Unituxin) - Medical Clinical Policy Bulletins Dinutuximab (Unituxin) is administered as 17.5 mg/m 2 /day as an intravenous infusion over 10 to 20 hours for 4 consecutive days for a maximum of 5 cycles.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6306059/

Spotlight on dinutuximab in the treatment of high-risk ... A major toxicity of anti-GD2 antibody infusions is neuropathic pain, which is thought to be a result of complement activation at GD2-expressing nerve fibers.

8. unituxin.com unituxin.com/US-UTX-0111-Unituxin-Parent-Book-1-24-20.pdf

Understanding Antibody Therapy Boxed WARNING. • Serious Infusion Reactions. — Unituxin can cause serious and potentially life-threatening infusion reactions.

9. oncologynewscentral.com oncologynewscentral.com/drugs/monograph/168737-316005/dinutuximab-intravenous

Dinutuximab: uses, dosing, warnings, adverse events, ... In the principal efficacy study in pediatric patients with high-risk neuroblastoma, grade 3 or 4 hypokalemia or hyponatremia occurred in 37 or 23%, respectively ...