Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

NCT ID: NCT01802567

Last Updated: 2024-08-06

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 52 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-03-04
• Study Completion Date: 2024-01-17

Brief Summary

The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations. This technology called the "Pediatric Gene Analysis Platform" includes a genomic report (gene expression profile) and a DNA Mutation Panel Report that are being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.

Conditions

Neuroblastoma; Medulloblastoma; Brain Tumors; Rare Tumors

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Guided Therapy- Pediatric Gene Analysis Platform

A total of 48 neuroblastoma, brain tumor, and rare tumor 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).

Group Type: EXPERIMENTAL

Guided Therapy- Pediatric Gene Analysis Platform

Intervention Type: DEVICE

A total of 48 neuroblastoma, brain tumor, and rare tumor 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).

Interventions

Guided Therapy- Pediatric Gene Analysis Platform

A total of 48 neuroblastoma, brain tumor, and rare tumor 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).

Intervention Type: DEVICE

Other Intervention Names

Molecular Guided Therapy

Eligibility Criteria

Inclusion Criteria

• Subjects must have histologically proven neuroblastoma, brain tumor, or rare tumor and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
• Subjects must be age >12 months at enrollment.
• Subjects must be age ≤ 21 years at initial diagnosis.
• Subjects must have measurable disease as 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% tumor are eligible to enroll.
• Current disease state must be one for which there is currently no known curative therapy
• Lansky or Karnofsky Score must be more than 50
• Subjects without bone marrow metastases must have an ANC > 750/μl
• Adequate liver function must be demonstrated, defined as:

1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND

2. SGPT (ALT) < 10 x upper limit of normal (ULN) for age

• 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 subjects 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.

Exclusion Criteria

• Subjects who have received any cytotoxic chemotherapy within the last 7 days prior to enrollment and 14 days prior to study treatment start date.
• Subjects who have received any radiotherapy to the primary sample site within the last 14 days (radiation may be included in treatment decision after biopsy).
• Subjects receiving anti-tumor therapy for their disease or any investigational drug concurrently
• Subjects 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.
• Subjects 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 subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study

• Minimum Eligible Age: 13 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Dell, Inc.

INDUSTRY

Sponsor Role: collaborator

Giselle Sholler

OTHER

Sponsor Role: lead

Responsible Party

Giselle Sholler

Beat Childhood Cancer Chair

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Giselle Sholler, MD

Role: STUDY_CHAIR

Beat Childhood Cancer at Atrium Health

Locations

Phoenix Children's Hospital

Phoenix, Arizona, United States

Rady Children's Hospital

San Diego, California, United States

Connecticut Children's Hospital

Hartford, Connecticut, United States

Arnold Palmer Hospital for Children- MD Anderson

Orlando, Florida, United States

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, United States

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, United States

Cardinal Glennon Children's Medical Center

St Louis, Missouri, United States

Levine Children's Hospital

Charlotte, North Carolina, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, United States

Dell Children's Blood and Cancer Center

Austin, Texas, United States

Primary Children's Hospital

Salt Lake City, Utah, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Related Links

http://www.beatcc.org

Beat Childhood Cancer Consortium website

Other Identifiers

NMTRC008

Identifier Type: -

Identifier Source: org_study_id