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ALK Inhibitor

Advanced Therapy for Neuroblastoma (PEDS-PLAN Trial)

Phase 2

Recruiting

Research Sponsored by Giselle Sholler

Eligibility Criteria Checklist

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

Must have

Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites

Subjects must be age ≤ 21 years at initial diagnosis

Must not have

Subjects who are 12-18 months of age with INSS Stage 4 and all stage 3 subjects with favorable biologic features are not eligible

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 years plus 5 years follow up

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether adding a new drug to standard treatment for neuroblastoma is safe and effective.

Who is the study for?

This trial is for children and young adults up to 21 years old with high-risk neuroblastoma. Participants must have proper heart, liver, and kidney function, not have had more than one cycle of certain chemotherapies, and be able to undergo stem cell collection. Pregnant or breastfeeding individuals can't join; neither can those with conditions that might affect the study's results.

What is being tested?

The study tests molecularly guided therapy combined with standard treatments for neuroblastoma. It includes a randomized trial comparing standard immunotherapy alone versus with DFMO (an investigational drug), followed by maintenance therapy using DFMO.

What are the potential side effects?

Possible side effects may include digestive issues, blood disorders, fatigue, allergic reactions to drugs like DFMO or vorinostat, and potential complications from procedures like stem cell collection.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with neuroblastoma or ganglioneuroblastoma.

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I was 21 years old or younger when first diagnosed.

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My liver tests are within the required range.

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I can safely undergo a stem cell collection procedure.

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My kidney function is good based on my age and gender.

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My heart is strong, with a good pumping efficiency.

Exclusion Criteria

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

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I am not between 12-18 months old with specific advanced neuroblastoma stages.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 years plus 5 years follow up

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 years plus 5 years follow up

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 years plus 5 years follow up for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of days from start of therapy to date of first relapse

Therapeutic procedure

Number of subjects that receive 75% of dosing of medications while on study protocol during cycles 3-6.

Secondary study objectives

Analgesics

Number of days that subjects remain alive

Therapeutic procedure

+2 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Active Control

Group I: Standard Immunotherapy without DFMOActive Control4 Interventions

One of the following drugs will be chosen for each subject based on molecular guided results: ceritinib, dasatinib, sorafenib or vorinostat. This will be followed standard immunotherapy with Dinutuximab/GM-CSF/IL-2 and isotretinoin. At the end of immunotherapy, DFMO will be given to all subjects BID for 730 days.

Group II: Standard Immunotherapy with DFMOActive Control5 Interventions

One of the following drugs will be chosen for each subject based on molecular guided results: ceritinib, dasatinib, sorafenib or vorinostat. This will be followed standard immunotherapy with Dinutuximab/GM-CSF/IL-2 and isotretinoin PLUS 1000mg/m2 BID of DFMO. At the end of immunotherapy, all subjects will go on to receive DFMO BID for 730 days.

0 / 7Eligibility criteria met