Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

NCT01802567 | Completed Results DMC

• 1 other identifier: NMTRC008
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 13

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

Outcome Measures

Primary Outcomes (1)

• Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment

  Days to treatment is one data point that will be used in order to determine feasibility. The definition of feasibility for this study will include: "Enrollment onto study, RNA expression profile completed, DNA Mutation Panel completed, genomic analysis and report generation, tumor board held with treatment decision, treatment review completed and start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."

  Date from biopsy to completion of 1 cycle of therapy, generally about 30 days

Secondary Outcomes (2)

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

  Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 3 years.

• Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans.

  Followed until off therapy, generally 3 years

Study Arms (1)

Guided Therapy- Pediatric Gene Analysis Platform

EXPERIMENTAL

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).

Device: Guided Therapy- Pediatric Gene Analysis Platform

Interventions

Guided Therapy- Pediatric Gene Analysis Platform 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).

Also known as: Molecular Guided Therapy

Guided Therapy- Pediatric Gene Analysis Platform

Eligibility Criteria

• Age: 13 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Giselle Sholler: lead
• Dell, Inc.: collaborator

Study Sites (13)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Rady Children's Hospital

San Diego, California, 92123, United States

Location

Connecticut Children's Hospital

Hartford, Connecticut, 06106, United States

Location

Arnold Palmer Hospital for Children- MD Anderson

Orlando, Florida, 32806, United States

Location

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96813, 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

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, 37232, United States

Location

Dell Children's Blood and Cancer Center

Austin, Texas, 78723, United States

Location

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

Related Links

• Beat Childhood Cancer Consortium website

MeSH Terms

Conditions

Neuroblastoma; Medulloblastoma; Brain Neoplasms

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; Glioma; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Results Point of Contact

• Title: Giselle Sholler, MD
• Organization: Penn State Health Children's Hospital

gsaulniersholler@pennstatehealth.psu.edu

7175310003

Study Officials

• Giselle Sholler, MD

  Beat Childhood Cancer at Atrium Health

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

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

Study Record Dates

• First Submitted: February 26, 2013
• First Posted: March 1, 2013
• Study Start: March 4, 2013
• Primary Completion: January 17, 2024
• Study Completion: January 17, 2024
• Last Updated: August 6, 2024
• Results First Posted: April 26, 2024

Record last verified: 2024-08

Locations

US (13)