Actively Recruiting

Phase 2
Age: 0 - 22Years
All Genders
NCT02559778

Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

Led by Giselle Sholler · Updated on 2026-04-28

500

Participants Needed

28

Research Sites

1043 weeks

Total Duration

On this page

Sponsors

G

Giselle Sholler

Lead Sponsor

D

Dell, Inc.

Collaborating Sponsor

AI-Summary

What this Trial Is About

A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma.

CONDITIONS

Official Title

Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

Who Can Participate

Age: 0 - 22Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of neuroblastoma or ganglioneuroblastoma confirmed by histology or bone marrow tests
  • Newly diagnosed neuroblastoma with INSS Stage 4, age > 18 months regardless of biology, or age 12-18 months with unfavorable features, or MYCN amplification
  • Newly diagnosed neuroblastoma with INSS Stage 3 with MYCN amplification or age > 18 months with unfavorable pathology
  • Newly diagnosed neuroblastoma with INSS Stage 2A/2B with MYCN amplification
  • Age 21 years or younger at initial diagnosis
  • No prior systemic therapy except limited emergency radiation or one cycle of low/intermediate risk chemotherapy
  • Ability to tolerate peripheral blood stem cell collection
  • Adequate cardiac function (shortening fraction ≥ 27% or ejection fraction ≥ 50%)
  • Adequate liver function (total bilirubin ≤ 1.5 x ULN and ALT < 10 x ULN)
  • Adequate renal function based on age and gender serum creatinine limits
  • Negative pregnancy test for females of childbearing potential
  • Agreement to use effective birth control methods during treatment and six months after
  • Signed informed consent and assent as appropriate
  • For Part B, pathological diagnosis of neuroblastoma, age ≤ 21, classified as high risk, and meeting staging and timing requirements
Not Eligible

You will not qualify if you...

  • Subjects 12-18 months old with INSS Stage 4 and all Stage 3 subjects with favorable biology are not eligible
  • Lactating females unless agreeing not to breastfeed
  • Receiving any other investigational drug concurrently
  • Presence of other medical conditions like malabsorption syndromes, mental illness, or substance abuse that interfere with consent or study participation

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 28 locations

1

University of Alabama/Children's of Alabama

Birmingham, Alabama, United States, 35233

Actively Recruiting

2

Arkansas Children's Hospital

Little Rock, Arkansas, United States, 72202

Actively Recruiting

3

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States, 94609

Actively Recruiting

4

Rady Children's Hospital

San Diego, California, United States, 92123

Actively Recruiting

5

Connecticut Children's Hospital

Hartford, Connecticut, United States, 06106

Actively Recruiting

6

Nicklaus Children's Miami

Miami, Florida, United States, 33155

Actively Recruiting

7

Arnold Palmer Hospital for Children

Orlando, Florida, United States, 32806

Actively Recruiting

8

St. Joseph's Children's Hospital

Tampa, Florida, United States, 33614

Actively Recruiting

9

Augusta University Health

Augusta, Georgia, United States, 30912

Actively Recruiting

10

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, United States, 96813

Actively Recruiting

11

St. Lukes

Boise, Idaho, United States, 83712

Completed

12

Advocate Aurora Research Institute

Chicago, Illinois, United States, 60453

Completed

13

Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine

Louisville, Kentucky, United States, 40202

Actively Recruiting

14

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States, 49503

Actively Recruiting

15

Children's Hospital and Clinics of Minnesota

Minneapolis, Minnesota, United States, 55404

Actively Recruiting

16

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, United States, 64108

Completed

17

Cardinal Glennon Children's Medical Center

St Louis, Missouri, United States, 63104

Actively Recruiting

18

Hackensack University Medical Center

Hackensack, New Jersey, United States, 07601

Completed

19

Levine Children's Hospital

Charlotte, North Carolina, United States, 28204

Actively Recruiting

20

Randall Children's Hospital

Portland, Oregon, United States, 97227

Actively Recruiting

21

Penn State Milton S. Hershey Medical Center and Children's Hospital

Hershey, Pennsylvania, United States, 17033

Actively Recruiting

22

Medical University of South Carolina

Charleston, South Carolina, United States, 29425

Actively Recruiting

23

Dell Children's Blood and Cancer Center

Austin, Texas, United States, 78723

Actively Recruiting

24

Children's Medical Center

Dallas, Texas, United States, 75235

Actively Recruiting

25

Alberta Children's Hospital

Calgary, Alberta, Canada, AB T3B 6A8

Actively Recruiting

26

CHU Sainte-Justine

Montreal, Quebec, Canada, QC H3S 2G4

Actively Recruiting

27

CHUQ

Québec, Quebec, Canada, QC G1V 4W6

Actively Recruiting

28

CIUSSS de l'Estrie-CHUS

Sherbrooke, Quebec, Canada, QC J1H 5H3

Actively Recruiting

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Research Team

B

BCC Enroll

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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Pediatric Precision Laboratory Advanced Neuroblastoma Therapy | DecenTrialz