Table 2 Clinical trials involving the use of targeted therapy drugs in DMG and other pediatric gliomas.

Abemaciclib

NCT02644460

Phase I [Recruiting]

Clinical trial evaluating abemaciclib in patients with newly diagnosed or relapsed/refractory DMG aged 2–25-years.

NCT04238819

Phase I [Recruiting]

Study to determine the safety and efficacy of abemaciclib in combination with temozolomide and irinotecan in patients with relapsed/refractory solid tumors aged up to 18-years.

AZD1390

NCT03215381

Phase I [Completed]

Study to analyze the PK of AZD1390 in healthy adult males aged between 20–65-years.

No results posted

NCT03423628

Phase I [Recruiting]

Study to test safety, tolerability, and PK of AZD1390 and radiotherapy for the treatment of glioblastoma in patients aged18–130-years.

AZD2014

NCT02619864

Phase I [Completed]

Clinical trial to determine MTD of AZD2014 in combination with temozolomide in glioblastoma patients 18- years and older.

No results posted

Binimetinib

NCT02285439

Phase I/II [Active, not recruiting]

Clinical trial to determine MTD of binimetinib (MEK162) in patients with low grade glioma aged 1–18-years.

BMS-986158

NCT03936465

Phase I [Recruiting]

Study investigating the bromodomain inhibitor, BMS-986158, for brain tumors in patients aged 1-21-years.

Cabozantinib

NCT02885324

Phase II [Recruiting]

Clinical trial to test cabozantinib for HGG patients aged 2–21-years.

Crenolanib

NCT01393912

Phase I [Completed]

Phase I clinical evaluated crenolanib in patients with newly diagnosed DMG or in recurrent, progressive, or refractory HGG aged 18 months-21-years.

No results posted

Dasatinib

NCT01644773

Phase I [Completed]

Clinical trial to determine MTD of crizotinib and dasatinib for patients with DMG and other HGG aged 2–21-years.

No results posted

NCT00996723

Phase I [Completed]

Clinical trial to evaluate the combination of vandetanib and dasatinib during and after radiotherapy in patients with DMG 18 months–21-years.

No results posted

Erlotinib

NCT01182350

Phase II [Terminated]

Clinical trial tested combinations of FDA-approved agents (including erlotinib) in patients with DMG aged 3–18-years based on specific biologic targets.

64.4% of patients had a 9-month overall survival rate following treatment

Erlotinib

NCT02233049

Phase II [Unknown]

Clinical trial comparing response of DMG patient to erlotinib, everolimus and/or dasatinib depending on biological abnormalities, aged 6 months–25-years.

Everolimus

NCT03387020

Phase I [Completed]

This study examined the side effects and best dose of ribociclib and everolimus as well as how well they work in treating patients, aged 1-21-years, with recurrent/refractory DMG.

MTD for ribociclib and everolimus was determined to be 120 and 1.2 mg/m²/day respectively.

NCT03355794

Phase I [Active, not recruiting]

Clinical trial examining the safety of ribociclib and everolimus, when administered to DMG patients aged 1-30-years following radiation therapy.

NCT03632317

Phase II [Withdrawn]

Clinical trial evaluated the activity of panobinostat in combination with everolimus for patients aged 2 to 30 years with newly diagnosed HGG or DMG after radiotherapy.

Low accrual

Fimepinostat

NCT03893487

Early Phase I [Recruiting]

Clinical trial studying the efficacy of fimepinostat in treating patients aged 3 to 39 years with newly diagnosed DMG

Gefitinib

NCT00042991

Phase I/II [Completed]

Clinical trial studied the efficacy of gefitinib, in combination with radiation therapy, in treating patients aged 3-21 years with brainstem gliomas and glioblastoma.

This trial found median progression -free survival to be 7.43 months on average while overall survival was 12.12 months

NCT00052208

Phase I/II [Completed]

Study investigated side effects and best dose of gefitinib when administered in conjunction with radiotherapy as well as its effectiveness in treating patients of all ages with glioblastoma.

No results posted

Imetelstat

NCT01836549

Phase II [Terminated]

Phase II clinical trial studied the efficacy of imetelstat in treating patients aged 12 months to 21 years with recurrent or refractory brain tumors.

Terminated due to several intracranial hemorrhages and recommendation by the PBTC DSMB.

Larotrectinib

NCT04655404

Early Phase I [Recruiting]

Clinical trial evaluating the disease status in patients aged up to 21 years with HGG with TRK fusion following larotrectinib treatment.

Mebendazole

NCT01837862

Phase I/II [Recruiting]

Study determining the safety and efficacy of mebendazole in combination with chemotherapy drugs for the treatment of DMG in patients between 1 and 21 years of age.

NCT02644291

Phase I [Recruiting]

Clinical trial investigating the safety and side effects of mebendazole in patients aged 1-21 years for recurrent brain cancers that are no longer responsive to standard therapies.

Olaparib

NCT03233204

Phase II [Recruiting]

Study observing the effectiveness of olaparib in treating patients (12 months to 21 years) with relapsed/refractory solid tumors, which possess defects in DNA damage repair genes.

NCT03155620

Phase II [Recruiting]

Phase II trial studying the efficacy of genetic testing-directed treatment (including the drug olaparib) in patients, between aged 12 months to 21 years, with advanced solid tumors.

ONC201

NCT03416530

Phase I [Recruiting]

Multicenter, seven arm, dose escalation, clinical trial studying ONC201 in patients aged 2 to 18 years with DMG and recurrent/refractory H3 K27M gliomas.

Palbociclib

NCT03709680

Phase I [Recruiting]

Study evaluating palbociclib in combination with chemotherapy in patents aged 2-20 years with medulloblastoma or DMG.

Ponatinib

NCT02478164

Phase II [Completed]

Clinical trial studied ponatinib as a potential treatment for recurrent glioblastoma unresponsive to bevacizumab in patients aged 18 years and older.

This clinical trial had no patients with a 3-month progression free survival and an average overall survival of 98 days.

Rapamycin

NCT02420613

Phase I [Active, not recruiting]

Phase I trial studying the side effects and best dose of rapamycin (temsirolimus) when given together with vorinostat and with or without radiation therapy in patients aged 7 months to 21 years with DMG

Ribociclib

NCT03355794

Phase I [Active, not recruiting]

Clinical trial examining the safety of ribociclib and everolimus, when administered to patients aged 12 months to 30 years with DMG following radiation therapy.

NCT02607124

Phase I/II [Terminated]

Study investigated the effects of ribociclib when given after radiation therapy in DMG patients aged 12 months to 30 years.

Terminated due to a competing trial opened for patient population with combination of ribociclib and everolimus.

NCT03387020

Phase I [Completed]

Clinical trial examined the side effects and best dose of ribociclib and everolimus and their efficacy in treating patients aged 1-21 years with treatment resistant or relapsed malignant brain tumors, including DMG.

No results posted

Talazoparib

NCT04740190

Phase II [Recruiting]

Study testing the effectiveness of talazoparib in recurrent glioblastoma in patients aged 18 years and older.

Trametinib

NCT03919071

Phase II [Recruiting]

Clinical trial examining how well dabrafenib and trametinib works after radiation therapy in HGG patients aged 3- 25 years.