NCT05843253 · Nationwide Children's Hospital
Study of Ribociclib and Everolimus in HGG and DIPG
What this study is about
The goal of this study is to determine the effectiveness of the study drugs ribociclib and everolimus to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K/mTOR) that these drugs target.
View original scientific description
The goal of this study is to determine the efficacy of the study drugs ribociclib and everolimus to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K/mTOR) that these drugs target. The main question the study aims to answer is whether the combination of ribociclib and everolimus can prolong the life of patients diagnosed with HGG, including DIPG.
Interventions
DRUG
Ribociclib
Ribociclib PO qd on days 1-21
DRUG
Everolimus
Everolimus PO qd on days 1-28
DRUG
Temozolomide (TMZ)
Temozolomide PO qd on days 1-5 for the first 13 cycles
Primary outcome measures
Progression-Free Survival (PFS) in HGG (Part 2, Stratum A)
Time frame: From date on treatment until date of Progressive Disease or death due to any cause or date of last follow-up, assessed up to 60 months
To assess the efficacy of ribociclib and everolimus in pediatric and young adult patients newly diagnosed with HGG by estimating the distribution of PFS compared to molecularly-stratified and matched historical controls.
Overall Survival (OS) in DIPG (Part 2, Stratum B)
Time frame: From date on treatment until date of death due to any cause or date of last follow-up, assessed up to 60 months
To assess the efficacy of ribociclib and everolimus in pediatric and young adult patients newly diagnosed with DIPG by estimating the distribution of OS compared to molecularly-stratified and matched historical controls.
Establish MTD and RP2D of ribociclib and everolimus (Part 2, Stratum D)
Time frame: Completion of Cycle 1 (28 days)
To identify the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of the combination of ribociclib and everolimus given to patients with metastatic HGG who have received craniospinal irradiation CSI.
Number of participants with ribociclib and everolimus-related adverse events as assessed by CTCAE v5.0 (Part 1- initial feasibility study)
Time frame: Completion of Cycle 1 (28 days)
Identify the safe dose of ribociclib powder for oral solution (PfOS) formulation in combination with everolimus that is feasible in pediatric patients with newly-diagnosed HGG, including DIPG, with cell cycle and/or PI3K/mTOR pathway alterations. This will be achieved by calculating the number of participants with, as well as frequency and severity of, ribociclib and everolimus-related Adverse Events as assessed by CTCAE v5.0 in the first 6-12 patients enrolled
Establish RP2D of ribociclib and temozolomide (Phase 1 Run-In Stratum E)
Time frame: Completion of Cycle 1 (28 days)
Establish RP2D of ribociclib and temozolomide (Phase 1 Run-In Stratum E) Description: To identify the Recommended Phase 2 Dose (RP2D) of the combination of ribociclib and temozolomide given to patients with newly diagnosed localized DHG, H3G34-mutant who have received RT.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
TarGeT-A study strata definitions Part1: Initial Feasibility Study for the combination of ribociclib PfOS formulation with everolimus: Enrollment on this cohort will be limited to patients aged \<21 years with primary intracranial localized HGG and DIPG Part 2 * Stratum A: Patients with localized, intracranial, non-pontine, and non-thalamic HGG (who do not meet criteria for strata C-D) * Stratum B: Patients with DIPG * Stratum C: Patients with primary thalamic, spinal cord, and/or secondary/radiation-related HGG. * Stratum D: Patients with metastatic/disseminated HGG, multifocal HGG, and/or gliomatosis cerebri who received CSI. Inclusion Criteria: 1. Inclusion criteria already met to enroll on TarGeT-SCR (central molecular and histopathologic screening) based on: 1.1) Age: patients must be ≥12 months and ≤39 years of age at the time of enrollment on TarGeT-SCR. For the Part 1 Initial Feasibility Cohort only: patients must be \<21 years of age at the time of enrollment on this p
Where
- Aurora, Colorado
- Washington D.C., District of Columbia
- Chicago, Illinois
- Boston, Massachusetts
- Ann Arbor, Michigan
- Durham, North Carolina
- Cincinnati, Ohio
- Columbus, Ohio
- Philadelphia, Pennsylvania
- Houston, Texas
- Seattle, Washington
Collaborators
Novartis
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 29, 2026 · Source of record for eligibility and locations