Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma

NCT01355679 | Completed Results DMC

• 1 other identifier: NMTRC 001
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 7

Brief Summary

The purpose of this study is to test the feasibility (ability to be done) of an experimental test to help plan your cancer treatment. This study plan is not studying the effectiveness of the proposed combinations of therapy for your cancer that you may receive after the experimental testing. This study will look at an experimental technology to determine a tumor's molecular makeup (gene expression profile). This technology (called "OncInsights") is being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future. The experimental technology has not been approved by the U.S. Food and Drug Administration.

Conditions

Neuroblastoma

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants That Are Able to Meet Feasibility Parameters.

  Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."

  1 year

Secondary Outcomes (3)

• Number of Participants With Adverse Events as a Measure of Safety

  1 year

• Overall Response Rate (ORR) of Participants Using RECIST Criteria

  1 year

• Activity of Treatments Chosen Based on Progression Free Survival (PFS)

  1 year

Study Arms (1)

Guided therapy

EXPERIMENTAL

A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Device: Guided Therapy

Interventions

Guided Therapy DEVICE

A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Guided therapy

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must have histologically proven neuroblastoma and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
• Patients must be age \> 12 months and ≤ 21 at initial diagnosis.
• Life expectancy must be more than 3 months
• If measurable disease, this must be demonstrated by residual abnormal tissue at a primary or metastatic site measuring more than 1 cm in any dimension by standardized imaging (CT or MRI); tumor must be accessible for biopsy. Patients with bone marrow only disease expected to be \> 75% are eligible to enroll.
• Current disease state must be one for which there is currently no known curative therapy
• Lansky or KarnofskyScore must be more than 50
• Patients without bone marrow metastases must have an ANC \> 750/μl and platelet count \> 50,000/μl
• Adequate liver function must be demonstrated, defined as:
• Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
• SGPT (ALT) \< 10 x upper limit of normal (ULN) for age
• No other significant organ toxicity defined as \> Grade 2 by National Cancer Institute Common Toxicity Criteria for Adverse Events NCI-CTCAE V4.0
• A negative serum pregnancy test is required for female participants of child bearing potential (≥ 13 years of age or after onset of menses)
• Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.
• Informed Consent: All patients and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines. Voluntary consent for optional biology studies will be included.

You may not qualify if:

• Patients who have received any chemotherapy within the last 7 days prior to enrollment and 14 days prior to study treatment start date.
• Patients who have received any radiotherapy within the last 30 days must have another site of disease to follow.
• Patients receiving anti-tumor therapy for their disease or any investigational drug concurrently
• Patients with serious infection or a life-threatening illness (unrelated to tumor) that is \> Grade 2 (NCI CTCAE V4.0), or active, serious infections requiring parenteral antibiotic therapy.
• Patients with any other medical condition, including malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a patient's ability to sign or the legal guardian's ability to sign the informed consent, and patient's ability to cooperate and participate in the study

Sponsors & Collaborators

• Giselle Sholler: lead

Study Sites (7)

Connecticut Children's Hospital

Hartford, Connecticut, 06106, United States

Location

Arnold Palmer Hospital for Children- MD Anderson

Orlando, Florida, 32806, United States

Location

National Cancer Institute

Bethesda, Maryland, 20877, United States

Location

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States

Location

Cardinal Glennon Children's Medical Center

St Louis, Missouri, 63104, United States

Location

Levine Children's Hospital

Charlotte, North Carolina, 28204, United States

Location

Related Links

• Beat Childhood Cancer

MeSH Terms

Conditions

Neuroblastoma

Interventions

Radiotherapy, Image-Guided

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Radiotherapy; Therapeutics

Results Point of Contact

• Title: Giselle Sholler, MD
• Organization: NMTRC at Spectrum Health

giselle.sholler@helendevoschildrens.org

616-267-0334

Study Officials

• Giselle Sholler, MD

  Beat Childhood Cancer at Atrium Health

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Study Chair

Study Record Dates

• First Submitted: May 13, 2011
• First Posted: May 18, 2011
• Study Start: May 1, 2011
• Primary Completion: September 1, 2013
• Study Completion: January 1, 2015
• Last Updated: August 6, 2024
• Results First Posted: July 28, 2016

Record last verified: 2024-08

Locations

US (7)