The goal of this study is to determine the efficacy of the 1) 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 or 2) ribociclib and temozolomide to treat pediatric and young adult patients newly diagnosed with diffuse hemispheric glioma (DHG), H3G34-mutant. The main question the study aims to answer is whether the combinations of ribociclib and everolimus or ribociclib and temozolomide can prolong the life of patients diagnosed with HGG/DIPG or DHG H3G34-mutant.
This is a multicenter, international, phase 2 study of post-radiotherapy (RT) combination of ribociclib and everolimus to treat pediatric, adolescent, and young adult patients newly diagnosed with HGG and DIPG that harbor alterations of the cell cycle and/or PI3K/mTOR pathways to assess treatment efficacy (Stratum A-D Part 2). The study will include a feasibility cohort (Strata A-D, Part 1) to identify the dose of ribociclib PfOS (Powder for Oral Suspension) that is safe and tolerable in combination with everolimus. Efficacy for Strata A-D Part 2 study will be defined by progression-free survival (PFS; HGG \[stratum A\]) and Overall Survival (OS; DIPG \[stratum B\]), with key longitudinal biomarker correlatives. Outcomes among patients with primary thalamic, spinal cord, and/or secondary (radiation related) HGG (strata C) will be descriptively analyzed. Objective radiographic response rates and agent-specific toxicities will also be assessed, with a feasibility cohort to determine the recommended phase 2 dose (RP2D) of the combination of ribociclib and everolimus in patients with metastatic disease who received upfront craniospinal irradiation (stratum D). The study was amended in October 2025 to also include an additional stratum, Stratum E, which will serve patients with localized DHG, H3G34-mutant with the combination of ribociclib and temozolomide. As most DHG, H3G34-mutant tumors are MGMT promoter methylated and receive temozolomide as part of standard of care, we are studying the adjuvant combination of ribociclib and temozolomide following upfront RT. Stratum E will open with a Phase 1 Run-In to determine the RP2D of ribociclib PfOS formulation in combination with temozolomide in patients newly-diagnosed with DHG, H3G34-mutant post-RT, with PK testing. Once the RP2D is determined, patients with localized DHG, H3G34-mutant will be enrolled on the Stratum E efficacy expansion cohort to descriptively assess survival outcomes, with similar longitudinal biomarker correlatives to the other strata. Protocol therapy with the maintenance combination of either 1) ribociclib and everolimus or 2) ribociclib and temozolomide must begin no later than 35 calendar days post-completion of RT. The earliest patients can begin protocol treatment is 28 calendar days post-completion of RT. Each cycle will be 28 days in duration and treatment can continue up to a total of 26 cycles. For Strata A-D, Ribociclib will be given orally once daily for 3 weeks (days 1-21), with one week off. Everolimus will be given orally daily continuously (days 1-28). For Stratum E, Ribociclib will be given orally once daily for 3 weeks (days 1-21), and temozolomide will be given days 1-5 for the first 13 cycles (Year 1), and ribociclib alone will be given orally once daily for 3 weeks (days 1-21) for the subsequent 13 cycles (Year 2).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
120
Ribociclib PO qd on days 1-21
Everolimus PO qd on days 1-28
Temozolomide PO qd on days 1-5 for the first 13 cycles
Children's Hospital Colorado
Aurora, Colorado, United States
NOT_YET_RECRUITINGChildren's National Medical Center
Washington D.C., District of Columbia, United States
RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
NOT_YET_RECRUITINGDana-Farber Cancer Institute
Boston, Massachusetts, United States
NOT_YET_RECRUITINGC.S. Mott Children's Hospital
Ann Arbor, Michigan, United States
RECRUITINGDuke University Health System
Durham, North Carolina, United States
NOT_YET_RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGNationwide Children's Hospital
Columbus, Ohio, United States
RECRUITINGChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGTexas Children's Hospital
Houston, Texas, United States
RECRUITING...and 11 more locations
Progression-Free Survival (PFS) in HGG (Part 2, Stratum A)
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.
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
Overall Survival (OS) in DIPG (Part 2, Stratum B)
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.
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
Establish MTD and RP2D of ribociclib and everolimus (Part 2, Stratum D)
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.
Time frame: Completion of Cycle 1 (28 days)
Number of participants with ribociclib and everolimus-related adverse events as assessed by CTCAE v5.0 (Part 1- initial feasibility study)
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
Time frame: Completion of Cycle 1 (28 days)
Establish RP2D of ribociclib and temozolomide (Phase 1 Run-In Stratum E)
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.
Time frame: Completion of Cycle 1 (28 days)
Overall Survival in HGG
Determine distribution of OS for pediatric and young adult patients newly-diagnosed with HGG which harbor cell cycle and/or PI3K/mTOR pathway alterations treated with post-RT ribociclib and everolimus.
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
Number of participants with ribociclib and everolimus-related adverse events as assessed by CTCAE v5.0
Assess and further characterize the safety and toxicity of post-RT combination of ribociclib and everolimus in pediatric and young adult patients newly diagnosed with HGG, including DIPG. 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.
Time frame: From Day 1 of protocol treatment through 30 days following end of protocol treatment
Evaluate Health-Related Quality of Life Outcomes
Evaluate health-related quality of life outcomes of pediatric and young adult patients newly-diagnosed with either 1) HGG, including DIPG, or 2) DHG, H3G34-mutant treated with combination of ribociclib and everolimus or ribociclib and temozolomide, by patient and/or parent reporting at key timepoints in therapy using the patient reported outcomes measurement information system (PROMIS) survey.
Time frame: At the end of every other Cycle (each cycle is 28 days)
Overall Survival in DHG, H3G34-mutant
Determine distribution of OS for pediatric and young adult patients newly-diagnosed with DHG, H3G34-mutant treated with post-RT ribociclib and temozolomide.
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
Number of participants with ribociclib and temozolomide-related adverse events as assessed by CTCAE v5.0
Assess and further characterize the safety and toxicity of post-RT combination of ribociclib and temozolomide in pediatric and young adult patients newly diagnosed with DHG, H3G34-mutant. This will be achieved by calculating the number of participants with, as well as frequency and severity of, ribociclib and temozolomide-related Adverse Events as assessed by CTCAE v5.0.
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Time frame: From Day 1 of protocol treatment through 30 days following end of protocol treatment