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PBTC-050: Phase I Ribociclib and Everolimus in Treating Children With Recurrent or Refractory Malignant Brain Tumors

Summary

Please note, this study is complete and is no longer accepting patients.


The main purpose of this study is to determine the best dose and side effects of a combination of drugs and to see how well the work in treating children and young adults with brain cancer that has come back or did not respond to treatment. These drugs may stop the growth of tumor cells by inhibiting the enzymes needed for the cells to grow. Giving ribociclib and everolimus together may work better in treating these aggressive types of brain tumors.

Trial Information

There are three parts to this study:


  • Screening – Doctors will review the patient’s medical history to make sure that this study treatment is appropriate.

  • Treatment – Treatment can take up to 24 months if the cancer does not continue to grow. Patients will take two pills at certain times, as directed by the treating doctor.  Patients must be able to swallow pills (medication may be given through a g-tube, but a g-tube cannot be placed only for the administration of study medication)

  • Follow-up – After treatment ends, patients will be followed up at 30 days after the last treatment was completed.

Approach

Treatment:


  1. Ribociclib: Patients receive ribociclib once a day on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2.

  2. Everolimus: Patients will receive everolimus once a day on days 3-28 of course 1 and days 1-28 of subsequent courses.

  3. Patients who are undergoing surgery also receive ribociclib PO QD on days 7-10 before surgery.

  4. Treatment will be repeated every 28 days for up to 13 courses if the disease does not progress and there are no unacceptable side effects. Patients with stable disease after 13 courses may continue receiving ribociclib and everolimus every 28 days for up to 13 additional courses if the disease does not progress and there are no unacceptable side effects

Additional Information

NCT03387020

Full Details on ClinicalTrials.Gov

This study is only available at 9 at hospitals throughout the United States.

Eligibility

Inclusion Criteria


  • Patient’s must be 1-21 years of age at the time of determining study eligibility

  • Recurrent, progressive or refractory ATRT (measurable disease, disease in the cerebral spinal fluid, or disease presence on imaging)

  • Tumor tissue available to assess Rb1 protein status

  • Able to swallow pills

  • Patient must have received prior therapy other than surgery and fully recovered from all side effects of all prior therapies.

  • Patients with neurological deficits should be stable at least 1 week prior to study enrollment

  • Patients on corticosteroids must be on a stable or decreasing dose at least 1 week prior to study enrollment

  • Labs confirming adequate organ and bone marrow function

  • Females of childbearing age will need to have a negative pregnancy test

  • Patients of childbearing or child fathering age must agree to use appropriate contraception


Exclusion Criteria


  • Use of other anticancer or investigational medications

  • Previous exposure to palbociclib, abernaciclib, or ribociclib (CDK4/6 inhibitor)

  • Abnormal heart function (LVEF < 50%)

  • Significant medical disorders (ex. Serious infections, significant cardiac, pulmonary hepatic, psychiatric, GI, or other organ issues)

  • Females that are pregnant or breastfeeding

  • Currently receiving warfarin for treatment or otherwise

  • Known hypersensitivity to ribociclib

Contact Information

Principal Investigator: Mariko DeWire-Schottmiller

Phone: (513) 803-1126

Email: mariko.dewire@cchmc.org


Secondary Contact: Stacye Richardson, MSHS

Phone: (901) 595-378

Email: stacye.richardson@st.jude.org

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