N/A
N=52
Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer
Neuroblastoma · Medulloblastoma · Brain Tumors · Rare Tumors
Bottom Line
View on ClinicalTrials.gov: NCT01802567 ↗Enrolled (actual)
52
Serious AEs
46.7%
Results posted
Apr 2024
Primary outcome: Primary: Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment — 12.2; 19.9; 14.9 Days
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Guided Therapy- Pediatric Gene Analysis Platform (Device)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Giselle Sholler
- Primary completion
- Jan 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment |
12.2; 19.9; 14.9 | — |
| SECONDARY Number of Participants With Adverse Events as a Measure of Safety |
43 | — |
| SECONDARY 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. |
1; 2; 2; 0; 0; 1 | — |
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.
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:
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND
- SGPT (ALT) 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
Data sourced from ClinicalTrials.gov (NCT01802567). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.