Molecular-Guided Therapy for Childhood Cancer

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Brief Title

Molecular-Guided Therapy for Childhood Cancer

Official Title

Molecular-guided Therapy for the Treatment of Patients With Relapsed and Refractory Childhood Cancers

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. This technology includes a genomic
      report based on DNA exomes and RNA sequencing that will be used to discover new ways to
      understand cancers and potentially predict the best treatments for patients with cancer in
      the future.
    



Study Type

Interventional


Primary Outcome

Days to treatment will be used in order to determine feasibility of using tumor samples to assess genomic sequencing using predictive modeling to make real-time treatment decisions for children with relapsed/refractory cancers.

Secondary Outcome

 Number of Participants with Adverse Events as a Measure of Safety

Condition

Neuroblastoma

Intervention

Guided Therapy

Study Arms / Comparison Groups

 Guided Therapy
Description:  A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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).

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Device

Estimated Enrollment

184

Start Date

June 2014

Completion Date

June 2026

Primary Completion Date

June 2025

Eligibility Criteria

        Inclusion Criteria:

          1. Subjects must have proven pediatric cancer with confirmation at diagnosis or at the
             time of recurrence/progression and clinical determination of disease for which there
             is no known effective curative therapy or disease that is refractory to established
             proven therapies fitting into one of the following categories:

               -  Neuroblastoma- Patients that have relapsed following standard of care therapy
                  (such as high risk patients, patient presenting after age 15 months or MYCN
                  amplified, and only following (for eligible patients) high-dose chemotherapy
                  followed by hematopoietic stem cell transplantation and maintenance therapy with
                  retinoic acid and antibody therapy) or having progressed during standard of care
                  therapy and non-responsive/progressive to accepted curative chemotherapy.

               -  Brain Tumors

               -  Medulloblastomas (At relapse after standard of care therapy [surgery,
                  chemotherapy and/or radiation] and/or non-responsive/progressive on accepted
                  curative therapy)

               -  Gliomas (At relapse after standard of care therapy [surgery and/or radiation
                  and/or chemotherapy] and/or non-responsive/progressive on accepted curative
                  therapy)

               -  Ependymomas (At relapse after standard of care therapy [surgery with or without
                  radiation] and/or non-responsive/progressive on accepted curative therapy)

               -  Choroid plexus tumors (At relapse after standard of care therapy [surgery] and/or
                  non-responsive/progressive on accepted curative therapy)

               -  Craniopharyngiomas (At relapse after standard of care therapy [surgery or
                  suppressive therapy] and/or non-responsive/progressive on accepted curative
                  therapy)

               -  Dysembryoplastic neuroepithelial tumors (DNETs) (At relapse after standard of
                  care therapy [surgery] and/or non-responsive/progressive on accepted curative
                  therapy)

               -  Meningiomas (At relapse after standard of care therapy [surgery] and/or
                  non-responsive/progressive on accepted curative therapy)

               -  Primitive Neuroectodermal Tumors (PNETs) (At relapse after standard of care
                  therapy [surgery, chemotherapy, and/or radiation] and/or
                  non-responsive/progressive on accepted curative therapy)

               -  Germ cell tumors (At relapse after standard of care therapy [surgery, and/or
                  radiation and/or chemotherapy] and/or non-responsive/progressive on accepted
                  curative therapy)

               -  Rare Tumors:

               -  Soft tissue sarcoma Rhabdomyosarcoma (At relapse after standard of care therapy
                  [surgery, and/or radiation, chemotherapy] and/or non-responsive/progressive to
                  accepted curative chemotherapy) Non-rhabdomyosarcoma (At relapse after standard
                  of care therapy [surgery, and/or radiation, chemotherapy] and/or
                  non-responsive/progressive to accepted curative chemotherapy)

               -  Bone Ewings sarcoma (At relapse after standard of care therapy [surgery, and/or
                  radiation, chemotherapy] and/or non- responsive/progressive to accepted curative
                  chemotherapy) Osteosarcoma (At relapse after standard of care therapy [surgery,
                  chemotherapy] and/or non- responsive/progressive to accepted curative
                  chemotherapy)

               -  Renal Wilms tumor (At relapse after standard of care therapy [surgery, and/or
                  radiation, chemotherapy] and/or non- responsive/progressive to accepted
                  chemotherapy) Renal cell carcinoma (At relapse after standard of care therapy
                  [surgery, chemotherapy] and/or non- responsive/progressive to accepted curative
                  chemotherapy) Malignant rhabdoid tumor (At diagnosis, as there is no known
                  curative therapy) Clear Cell Sarcoma- (At relapse after standard of care therapy
                  [radiation, chemotherapy] and/or non- responsive/progressive to accepted curative
                  chemotherapy) Germ Cell tumors (At relapse after standard of care therapy
                  [surgery, chemotherapy] and/or non-responsive/progressive to accepted curative
                  chemotherapy)

               -  Liver Tumors (At relapse after standard of care therapy [surgery, chemotherapy]
                  and/or non- responsive/progressive to accepted curative chemotherapy)

          2. Subjects must be age >12 months at enrollment

          3. Subjects must be age ≤ 21 years at initial diagnosis

          4. Subjects must have measurable disease as demonstrated by residual abnormal tissue at a
             primary or metastatic site (measurable on CT or MRI) at the time of biopsy; tumor must
             be accessible for biopsy. In addition, subjects with bone or bone marrow only disease
             expected to be >75% tumor are eligible to enroll.

          5. Current disease state must be one for which there is currently no known effective
             therapy

          6. Specimens will be obtained only in a non-significant risk manner and not solely for
             the purpose of investigational testing.

          7. Lansky or Karnofsky Score must be ≥ 50

          8. Subjects without bone marrow metastases must have an ANC > 750/μl to begin treatment.

          9. Subjects with CNS disease must have been on a stable dose of steroids for 2 weeks
             prior to their biopsy and must not have progressive hydrocephalus at enrollment.

         10. Adequate liver function must be demonstrated, defined as:

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

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

         11. A negative serum pregnancy test is required for female participants of child bearing
             potential (≥13 years of age or after onset of menses)

         12. 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.

         13. Informed Consent: All subjects and/or legal guardians must sign informed written
             consent. Assent, when appropriate, will be obtained according to institutional
             guidelines

        Exclusion Criteria:

          1. Subjects who have received any cytotoxic chemotherapy within the last 7 days prior to
             biopsy

          2. 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).

          3. Subjects receiving any investigational drug concurrently.

          4. Subjects with uncontrolled serious infections or a life-threatening illness (unrelated
             to tumor)

          5. 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
      

Gender

All

Ages

13 Months - 21 Years

Accepts Healthy Volunteers

No

Contacts

Giselle Sholler, MD, , 

Location Countries

Lebanon

Location Countries

Lebanon

Administrative Informations


NCT ID

NCT02162732

Organization ID

NMTRC009


Responsible Party

Sponsor-Investigator

Study Sponsor

Giselle SaulnierSholler

Collaborators

 Translational Genomics Research Institute

Study Sponsor

Giselle Sholler, MD, Study Chair, Beat Childhood Cancer at Atrium Health


Verification Date

November 2020