The Brain Tumor Institute at Children’s National is leading the way to find new and better treatments for children. Our clinical trials give families the chance to try different options that may help their child and others. We understand this is a big decision and our team is here to support you. For more information about the trials listed below, contact research nurse coordinators Julia Mascia and Julia Batarseh, or call 202-476-2907.
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Explore Clinical Trials by Disease Type (A-G)
Disease Status: Newly Diagnosed
CONNECT1905: Phase 2 Study of Systemic IL-6 Receptor Antagonist ACTEMRA® (tocilizumab) for the treatment of Pediatric Adamantinomatous Craniopharyngioma
ClinicalTrials.gov Identifier: NCT05233397
Description:
- In this Phase II, tociluzumab will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and/or radiation therapy.
Key Eligibility Criteria:
- Stratum 1: Patients ≥ 12 months and ≤ 39 years of age with recurrent or progressive adamantinomatous craniopharyngioma at least 6 months post completion of radiation therapy
- Stratum 2: Patients ≥ 12 months and ≤ 39 years of age with recurrent or progressive adamantinomatous craniopharyngioma who have undergone surgery but have NOT previously undergone irradiation
- Progressive disease is allowed but not required
- Nationwide Children's Hospital
Investigator:
CONNECT2108: Phase 2 Study of the MEK inhibitor MEKTOVI® (binimetinib) for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
ClinicalTrials.gov Identifier: NCT05286788
Description
- Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating pediatric brain tumor that lacks medical anti-tumor therapies. Current therapy, which depends largely on surgery and radiation, is associated with poor quality of life and becomes more challenging and risky in the setting of recurrent disease. MEKTOVI (binimetinib) is an oral, highly selective reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2 that is being evaluated in pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma (ACP), including patients who have undergone surgery and/or radiation therapy.
Key Eligibility Criteria
- Patients aged >1 year and <25 years and must have progressive or recurrent ACP and have recovered or stabilized from the acute toxic effects of prior treatments
- Progressive disease is allowed but not required
Sponsor
- Nationwide Children’s Hospital
Investigator
Disease Type
- Recurrent Adamantinomatous Craniopharyngioma (ACP)
Disease Status: Recurrent
CONNECT1905: Phase 2 Study of Systemic IL-6 Receptor Antagonist ACTEMRA® (tocilizumab) for the treatment of Pediatric Adamantinomatous Craniopharyngioma
ClinicalTrials.gov Identifier: NCT05233397
Description:
- In this Phase II, tociluzumab will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and/or radiation therapy.
Key Eligibility Criteria:
- Stratum 1: Patients ≥ 12 months and ≤ 39 years of age with recurrent or progressive adamantinomatous craniopharyngioma at least 6 months post completion of radiation therapy
- Stratum 2: Patients ≥ 12 months and ≤ 39 years of age with recurrent or progressive adamantinomatous craniopharyngioma who have undergone surgery but have NOT previously undergone irradiation
- Progressive disease is allowed but not required
- Nationwide Children's Hospital
Investigator:
CONNECT2108: Phase 2 Study of the MEK inhibitor MEKTOVI® (binimetinib) for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
ClinicalTrials.gov Identifier: NCT05286788
Description
- Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating pediatric brain tumor that lacks medical anti-tumor therapies. Current therapy, which depends largely on surgery and radiation, is associated with poor quality of life and becomes more challenging and risky in the setting of recurrent disease. MEKTOVI (binimetinib) is an oral, highly selective reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2 that is being evaluated in pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma (ACP), including patients who have undergone surgery and/or radiation therapy.
Key Eligibility Criteria
- Patients aged >1 year and <25 years and must have progressive or recurrent ACP and have recovered or stabilized from the acute toxic effects of prior treatments
- Progressive disease is allowed but not required
Sponsor
- Nationwide Children’s Hospital
Investigator
Disease Type
- Recurrent Adamantinomatous Craniopharyngioma (ACP)
Disease Status: Newly Diagnosed
BT-016: A Safety and Feasibility Study to Evaluate Blood Brain Barrier Disruption Using Exablate
ClinicalTrials.gov Identifier: NCT05630209
Description
- A feasibility of Blood Brain Barrier disruption using the Exablate 4000 Type 2 System in patients with DIPG undergoing Doxorubicin chemotherapy
Key Eligibility Criteria
- Newly diagnosed DIPG with a Tumor size volume approximately between 10 cc and 30 cc
- At least 4-week and not greater than 12 weeks from completion of radiation therapy
Sponsor
- Insightec
Investigator
Disease Type
- Newly diagnosed diffuse intrinsic pontine glioma
CONNECT: TarGeT Screening: Targeted Pediatric High-Grade Glioma Therapy
ClinicalTrials.gov Identifier: NCT05839379
Official title:
- Targeted Pediatric High-Grade Glioma Therapy
Description:
- The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with a high-grade glioma. This molecular profiling will decide if patients are eligible to participate in a subsequent treatment-based clinical trial based on the genetic alterations identified in their tumor.
Key Eligibility Criteria:
- Patients ≥12 months and ≤39 years with newly diagnosed high-grade glioma (HGG), including diffuse intrinsic pontine glioma (DIPG) who have had a biopsy
Sponsor:
- Nationwide Children's Hospital
Investigator:
CONNECT: TarGeT-A: Phase 2 Study of Ribociclib and Everolimus Following Radiotherapy in Pediatric and Young Adult Patients Newly Diagnosed with HighGrade Glioma (HGG), including Diffuse Intrinsic Pontine Glioma (DIPG), which Harbor Alterations of the Cell Cycle and/or PI3K/mTOR Pathway
*For patients with PI3K/mTOR alterations
ClinicalTrials.gov ID: NCT05843253
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.
Key Eligibility Criteria:
- For patients with PI3K/mTOR alterations
Sponsor:
- Nationwide Children's Hospital
Investigator:
CONNECT 1906: Phase 2 trial of a novel peptide vaccine (PEP-CMV) targeting CMV antigen for newly diagnosed pediatric high-grade glioma and diffuse intrinsic pontine glioma and recurrent medulloblastoma
ClinicalTrials.gov Identifier: NCT05096481
Description:
- This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB).
Key Eligibility Criteria:
- Patients aged ≥3 and ≤39 years with recurrent/progressive medulloblastoma, newly diagnosed high grade glioma, or newly diagnosed diffuse intrinsic pontine glioma (biopsy not required)
Sponsor:
- Nationwide Children's Hospital
Investigator:
PNOC-022: A Trial Using an Adaptive Platform Design for Children and Young Adults Therapy with Diffuse Midline Gliomas, and DIPG and at Time of Progression Using ONC201
ClinicalTrials.gov Identifier: NCT05009992
Description
- This study evaluates the efficacy of the combination of ONC201 and panobinostat or paxalisib for treating patients with diffuse midline gliomas (DMGs). ONC201, panobinostat, and paxalisib are all enzyme inhibitors that may stop the growth of tumor cells by clocking some of the enzymes needed for cell growth. Given the limited treatment options for patients with DMGs, we also aim to offer trial enrollment at different disease stages – newly-diagnosed, after completion of radiation therapy, and at time of progression.
Key Eligibility Criteria
-
Diagnosis of DMG, including spinal cord tumors
-
This study will enroll children and young adults (2-39 years of age) with diffuse midline gliomas (DMG) at different stages of their disease.
Sponsor
- University of California, San Francisco
Investigator
Disease Type
- Diffuse midline glioma (DMG)
CONNECT 2110: A Phase II Trial of ACT001 in Children and Adolescents with Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
ClinicalTrials.gov Identifier: NCT06838676
Description:
This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.
Key Eligibility Criteria:
- Cohort A: Patients > 12 months and < 39="" years="" of="" age="" with="" newly="" diagnosed="" dipg="" with="" or="" without="" biopsy="" and="" have="" completed="" radiation="" therapy="" within="" 28="" to="" 35="" calendar="" day="" prior="" to="" start="" of="" />
- Cohort B: Patients > 12 months and < 39="" years="" of="" age="" with="" progressive="" refractory="" dipg="" or="" progressive="" recurrent="" refractory="" h3k27-altered="" />
Sponsor:
- Nationwide Children's Hospital
Investigator:
UPCOMING: PNOC-023: Open label Phase 1 and Target Validation study of ONC206 in Children and Young Adults with Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Central Nervous System (CNS) Tumors o Description: This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors
*Biopsy required
ClinicalTrials.gov Identifier: NCT04732065
Description:
- This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors.
Key Eligibility Criteria:
- Arm A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy
- Arm B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) with a diagnosis of DMG, H3K27 altered are eligible, including spinal cord DMGs
- Arm C: Children and young adults with DMGs (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression and are recommended to get re-irradiation
- Arm D: Children and young adults with recurrent primary malignant CNS tumors (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression
Sponsor:
- Sabine Mueller, MD, PhD
Investigator:
Disease Status: Recurrent
PEPN2111: A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
ClinicalTrials.gov Identifier: NCT04870944
Description
- This phase I/II trial evaluates the best dose, side effects, and possible benefit of CBL0137 in treating patients with relapsed, or refractory solid tumors, including central nervous system (CNS) tumors or lymphoma.
Key Eligibility Criteria
- Parts A and B1: ≥ 12 months and ≤ 21 years old
- Part B2 (relapsed/refractory osteosarcoma): ≥ 12 months and ≤ 30 years old
Sponsor
- Children's Oncology Group
Investigator
Disease Type
- Solid tumors, lymphoma
PNOC-022: A Trial Using an Adaptive Platform Design for Children and Young Adults Therapy with Diffuse Midline Gliomas, and DIPG and at Time of Progression Using ONC201
ClinicalTrials.gov Identifier: NCT05009992
Description
- This study evaluates the efficacy of the combination of ONC201 and panobinostat or paxalisib for treating patients with diffuse midline gliomas (DMGs). ONC201, panobinostat, and paxalisib are all enzyme inhibitors that may stop the growth of tumor cells by clocking some of the enzymes needed for cell growth. Given the limited treatment options for patients with DMGs, we also aim to offer trial enrollment at different disease stages – newly-diagnosed, after completion of radiation therapy, and at time of progression.
Key Eligibility Criteria
-
Diagnosis of DMG, including spinal cord tumors
-
This study will enroll children and young adults (2-39 years of age) with diffuse midline gliomas (DMG) at different stages of their disease.
Sponsor
- University of California, San Francisco
Investigator
Disease Type
- Diffuse midline glioma (DMG)
CONNECT 2110: A Phase II Trial of ACT001 in Children and Adolescents with Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
ClinicalTrials.gov Identifier: NCT06838676
Description:
This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.
Key Eligibility Criteria:
- Cohort A: Patients > 12 months and < 39="" years="" of="" age="" with="" newly="" diagnosed="" dipg="" with="" or="" without="" biopsy="" and="" have="" completed="" radiation="" therapy="" within="" 28="" to="" 35="" calendar="" day="" prior="" to="" start="" of="" />
- Cohort B: Patients > 12 months and < 39="" years="" of="" age="" with="" progressive="" refractory="" dipg="" or="" progressive="" recurrent="" refractory="" h3k27-altered="" />
Sponsor:
- Nationwide Children's Hospital
Investigator:
UPCOMING: PNOC-023: Open label Phase 1 and Target Validation study of ONC206 in Children and Young Adults with Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Central Nervous System (CNS) Tumors o Description: This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors
*Biopsy required
ClinicalTrials.gov Identifier: NCT04732065
Description:
- This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors.
Key Eligibility Criteria:
- Arm A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy
- Arm B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) with a diagnosis of DMG, H3K27 altered are eligible, including spinal cord DMGs
- Arm C: Children and young adults with DMGs (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression and are recommended to get re-irradiation
- Arm D: Children and young adults with recurrent primary malignant CNS tumors (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression
Sponsor:
- Sabine Mueller, MD, PhD
Investigator:
Disease Status: Newly Diagnosed
PNOC-022: A Trial Using an Adaptive Platform Design for Children and Young Adults Therapy with Diffuse Midline Gliomas, and DIPG and at Time of Progression Using ONC201
ClinicalTrials.gov Identifier: NCT05009992
Description
- This study evaluates the efficacy of the combination of ONC201 and panobinostat or paxalisib for treating patients with diffuse midline gliomas (DMGs). ONC201, panobinostat, and paxalisib are all enzyme inhibitors that may stop the growth of tumor cells by clocking some of the enzymes needed for cell growth. Given the limited treatment options for patients with DMGs, we also aim to offer trial enrollment at different disease stages – newly-diagnosed, after completion of radiation therapy, and at time of progression.
Key Eligibility Criteria
-
Diagnosis of DMG, including spinal cord tumors
-
This study will enroll children and young adults (2-39 years of age) with diffuse midline gliomas (DMG) at different stages of their disease.
Sponsor
- University of California, San Francisco
Investigator
Disease Type
- Diffuse midline glioma (DMG)
CONNECT: TarGeT Screening: Targeted Pediatric High-Grade Glioma Therapy
ClinicalTrials.gov Identifier: NCT05839379
Official title:
- Targeted Pediatric High-Grade Glioma Therapy
Description:
- The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with a high-grade glioma. This molecular profiling will decide if patients are eligible to participate in a subsequent treatment-based clinical trial based on the genetic alterations identified in their tumor.
Key Eligibility Criteria:
- Patients ≥12 months and ≤39 years with newly diagnosed high-grade glioma (HGG), including diffuse intrinsic pontine glioma (DIPG) who have had a biopsy
Sponsor:
- Nationwide Children's Hospital
Investigator:
CONNECT: TarGeT-A: Phase 2 Study of Ribociclib and Everolimus Following Radiotherapy in Pediatric and Young Adult Patients Newly Diagnosed with HighGrade Glioma (HGG), including Diffuse Intrinsic Pontine Glioma (DIPG), which Harbor Alterations of the Cell Cycle and/or PI3K/mTOR Pathway
*For patients with PI3K/mTOR alterations
ClinicalTrials.gov ID: NCT05843253
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.
Key Eligibility Criteria:
- For patients with PI3K/mTOR alterations
Sponsor:
- Nationwide Children's Hospital
Investigator:
CONNECT 1906: Phase 2 trial of a novel peptide vaccine (PEP-CMV) targeting CMV antigen for newly diagnosed pediatric high-grade glioma and diffuse intrinsic pontine glioma and recurrent medulloblastoma
ClinicalTrials.gov Identifier: NCT05096481
Description:
- This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB).
Key Eligibility Criteria:
- Patients aged ≥3 and ≤39 years with recurrent/progressive medulloblastoma, newly diagnosed high grade glioma, or newly diagnosed diffuse intrinsic pontine glioma (biopsy not required)
Sponsor:
- Nationwide Children's Hospital
Investigator:
UPCOMING: PNOC-023: Open label Phase 1 and Target Validation study of ONC206 in Children and Young Adults with Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Central Nervous System (CNS) Tumors o Description: This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors
*Biopsy required
ClinicalTrials.gov Identifier: NCT04732065
Description:
- This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors.
Key Eligibility Criteria:
- Arm A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy
- Arm B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) with a diagnosis of DMG, H3K27 altered are eligible, including spinal cord DMGs
- Arm C: Children and young adults with DMGs (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression and are recommended to get re-irradiation
- Arm D: Children and young adults with recurrent primary malignant CNS tumors (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression
Sponsor:
- Sabine Mueller, MD, PhD
Investigator:
Disease Status: Recurrent
PEPN2111: A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
ClinicalTrials.gov Identifier: NCT04870944
Description
- This phase I/II trial evaluates the best dose, side effects, and possible benefit of CBL0137 in treating patients with relapsed, or refractory solid tumors, including central nervous system (CNS) tumors or lymphoma.
Key Eligibility Criteria
- Parts A and B1: ≥ 12 months and ≤ 21 years old
- Part B2 (relapsed/refractory osteosarcoma): ≥ 12 months and ≤ 30 years old
Sponsor
- Children's Oncology Group
Investigator
Disease Type
- Solid tumors, lymphoma
PNOC-021: A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas and High Grade Gliomas
ClinicalTrials.gov Identifier: NCT04485559
Description
- This will be the first study to determine the optimal dosing schedule and probable side effects for a combination therapy of trametinib and everolimus in pediatric LGG.
Key Eligibility Criteria
- Participants must have histologically confirmed diagnosis of a LGG (WHO grade I-II) that is recurrent or progressive after prior treatment
- Participants who have had surgery alone are not eligible
Sponsor
- Pacific Neuro-Oncology Consortium
Investigator
Disease Type
- Low grade glioma
- High grade glioma
PNOC-022: A Trial Using an Adaptive Platform Design for Children and Young Adults Therapy with Diffuse Midline Gliomas, and DIPG and at Time of Progression Using ONC201
ClinicalTrials.gov Identifier: NCT05009992
Description
- This study evaluates the efficacy of the combination of ONC201 and panobinostat or paxalisib for treating patients with diffuse midline gliomas (DMGs). ONC201, panobinostat, and paxalisib are all enzyme inhibitors that may stop the growth of tumor cells by clocking some of the enzymes needed for cell growth. Given the limited treatment options for patients with DMGs, we also aim to offer trial enrollment at different disease stages – newly-diagnosed, after completion of radiation therapy, and at time of progression.
Key Eligibility Criteria
-
Diagnosis of DMG, including spinal cord tumors
-
This study will enroll children and young adults (2-39 years of age) with diffuse midline gliomas (DMG) at different stages of their disease.
Sponsor
- University of California, San Francisco
Investigator
Disease Type
- Diffuse midline glioma (DMG)
CONNECT 2110: A Phase II Trial of ACT001 in Children and Adolescents with Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas
ClinicalTrials.gov Identifier: NCT06838676
Description:
This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.
Key Eligibility Criteria:
- Cohort A: Patients > 12 months and < 39="" years="" of="" age="" with="" newly="" diagnosed="" dipg="" with="" or="" without="" biopsy="" and="" have="" completed="" radiation="" therapy="" within="" 28="" to="" 35="" calendar="" day="" prior="" to="" start="" of="" />
- Cohort B: Patients > 12 months and < 39="" years="" of="" age="" with="" progressive="" refractory="" dipg="" or="" progressive="" recurrent="" refractory="" h3k27-altered="" />
Sponsor:
- Nationwide Children's Hospital
Investigator:
UPCOMING: PNOC-023: Open label Phase 1 and Target Validation study of ONC206 in Children and Young Adults with Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Central Nervous System (CNS) Tumors o Description: This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors
*Biopsy required
ClinicalTrials.gov Identifier: NCT04732065
Description:
- This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors.
Key Eligibility Criteria:
- Arm A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy
- Arm B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) with a diagnosis of DMG, H3K27 altered are eligible, including spinal cord DMGs
- Arm C: Children and young adults with DMGs (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression and are recommended to get re-irradiation
- Arm D: Children and young adults with recurrent primary malignant CNS tumors (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression
Sponsor:
- Sabine Mueller, MD, PhD
Investigator:
Disease Status: Newly Diagnosed
CONNECT 1906: Phase 2 trial of a novel peptide vaccine (PEP-CMV) targeting CMV antigen for newly diagnosed pediatric high-grade glioma and diffuse intrinsic pontine glioma and recurrent medulloblastoma
ClinicalTrials.gov Identifier: NCT05096481
Description:
- This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB).
Key Eligibility Criteria:
- Patients aged ≥3 and ≤39 years with recurrent/progressive medulloblastoma, newly diagnosed high grade glioma, or newly diagnosed diffuse intrinsic pontine glioma (biopsy not required)
Sponsor:
- Nationwide Children's Hospital
Investigator:
Disease Status: Recurrent
PNOC-027 : A pilot trial of real time drug screening and genomic testing to determine an individualized treatment plan in children and young adults with relapsed medulloblastoma
ClinicalTrials.gov Identifier: NCT05057702
Description
- The current study will use a new treatment approach based on the molecular characteristics of each participant’s tumor.
Key Eligibility Criteria
- Participants must undergo surgery and have surgically accessible disease
- Participants must have recurrent medulloblastoma
- Participants must have had at leapt one prior therapy
Sponsor
- Pacific Neuro-Oncology Consortium
Investigator
JACOB: Joint Administration of cDNA for TP53, checkpOint inhibition and Boost/Hypofractionated radiation. A phase 0/1 study in children with recurrent, progressive or refractory CNS malignancies
Description:
- This study will assess the maximum tolerated dose and/or the recommended phase II dose (RP2D) of SGT-53 in conjunction with hypofractionated radiotherapy and immune checkpoint blockade in children with recurrent, progressive, or refractory CNS malignancies
Key Eligibility Criteria:
- Children aged ≥3yr to ≤21 years old with recurrent, progressive or refractory CNS malignancy
Sponsor:
Investigator:
- Eugene Hwang, MD
Explore Clinical Trials by Disease Type (H-Z)
Disease Status: Newly Diagnosed
CONNECT1903: A Pilot and Surgical Study of Larotrectinib for Treatment of Children with Newly Diagnosed High-Grade Glioma with NTRK Fusion
ClinicalTrials.gov Identifier: NCT04655404
Description:
- This study will evaluate disease status in children that have been newly diagnosed with high-grade glioma with NTRK fusion after 2 cycles of the medication (Larotrectinib) have been given. The study will also evaluate the safety of larotrectinib when given with chemotherapy or post-focal radiation therapy.
Key Eligibility Criteria:
- Patients ≤ 21 years of age with newly diagnosed high grade glioma with NTRK fusion
Sponsor:
- Nationwide Children's Hospital
Investigator:
CONNECT: TarGeT Screening: Targeted Pediatric High-Grade Glioma Therapy
ClinicalTrials.gov Identifier: NCT05839379
Official title:
- Targeted Pediatric High-Grade Glioma Therapy
Description:
- The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with a high-grade glioma. This molecular profiling will decide if patients are eligible to participate in a subsequent treatment-based clinical trial based on the genetic alterations identified in their tumor.
Key Eligibility Criteria:
- Patients ≥12 months and ≤39 years with newly diagnosed high-grade glioma (HGG), including diffuse intrinsic pontine glioma (DIPG) who have had a biopsy
Sponsor:
- Nationwide Children's Hospital
Investigator:
CONNECT: TarGeT-A: Phase 2 Study of Ribociclib and Everolimus Following Radiotherapy in Pediatric and Young Adult Patients Newly Diagnosed with HighGrade Glioma (HGG), including Diffuse Intrinsic Pontine Glioma (DIPG), which Harbor Alterations of the Cell Cycle and/or PI3K/mTOR Pathway
*For patients with PI3K/mTOR alterations
ClinicalTrials.gov ID: NCT05843253
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.
Key Eligibility Criteria:
- For patients with PI3K/mTOR alterations
Sponsor:
- Nationwide Children's Hospital
Investigator:
CONNECT 1906: Phase 2 trial of a novel peptide vaccine (PEP-CMV) targeting CMV antigen for newly diagnosed pediatric high-grade glioma and diffuse intrinsic pontine glioma and recurrent medulloblastoma
ClinicalTrials.gov Identifier: NCT05096481
Description:
- This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB).
Key Eligibility Criteria:
- Patients aged ≥3 and ≤39 years with recurrent/progressive medulloblastoma, newly diagnosed high grade glioma, or newly diagnosed diffuse intrinsic pontine glioma (biopsy not required)
Sponsor:
- Nationwide Children's Hospital
Investigator:
LCH24C02: A Pilot Study Evaluating the Toxicity and Clinical Benefit of Mitogen-activated Protein Kinase (MAPK) Pathway Inhibition Combined with Programmed Cell Death-1 Checkpoint Blockade (anti-PD1) for the Treatment of BRAF-altered Pediatric Gliomas
*For patients with BRAF or NF-1 alterations
ClinicalTrials.gov Identifier: NCT06712875
Description:
- This is a pilot study evaluating the toxicity and early efficacy of dabrafenib and/or trametinib combined with nivolumab for the treatment of BRAF-altered or NF altered gliomas.
Key Eligibility Criteria:
- Cohort A: Patients ≥1 and ≤26 years of age with a recurrent or progressive low grade glioma harboring a KIAA1549-BRAF fusion, or a newly diagnosed high grade glioma harboring a KIAA1549-BRAF fusion
- Cohort B: Patients ≥1 and ≤26 years of age with NF-1 associated or altered low-grade glioma that is recurrent or progressive, high-grade glioma that is newly diagnosed or recurrent, or transforming glioma that is newly diagnosed or recurrent
Sponsor:
- Ann & Robert H Lurie Children's Hospital of Chicago
Investigator:
UPCOMING: PNOC-020: A Phase I/II Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM)
ClinicalTrials.gov Identifier: NCT04573140
Description:
- The primary objective will be to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in (Stratum 1) adult patients with newly diagnosed GBM (MGMT low level or unmethylated in adults only) and (Stratum 2) in pediatric patients with newly diagnosed HGG (pHGG).
Key Eligibility Criteria:
- Patients age > 3 and < 25 years with newly diagnosed high grade glioma, prior to surgical resection and radiation
Sponsor:
- University of Florida
Investigator:
Disease Status: Recurrent
PEPN2111: A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
ClinicalTrials.gov Identifier: NCT04870944
Description
- This phase I/II trial evaluates the best dose, side effects, and possible benefit of CBL0137 in treating patients with relapsed, or refractory solid tumors, including central nervous system (CNS) tumors or lymphoma.
Key Eligibility Criteria
- Parts A and B1: ≥ 12 months and ≤ 21 years old
- Part B2 (relapsed/refractory osteosarcoma): ≥ 12 months and ≤ 30 years old
Sponsor
- Children's Oncology Group
Investigator
Disease Type
- Solid tumors, lymphoma
PNOC-021: A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas and High Grade Gliomas
ClinicalTrials.gov Identifier: NCT04485559
Description
- This will be the first study to determine the optimal dosing schedule and probable side effects for a combination therapy of trametinib and everolimus in pediatric LGG.
Key Eligibility Criteria
- Participants must have histologically confirmed diagnosis of a LGG (WHO grade I-II) that is recurrent or progressive after prior treatment
- Participants who have had surgery alone are not eligible
Sponsor
- Pacific Neuro-Oncology Consortium
Investigator
Disease Type
- Low grade glioma
- High grade glioma
LCH24C02: A Pilot Study Evaluating the Toxicity and Clinical Benefit of Mitogen-activated Protein Kinase (MAPK) Pathway Inhibition Combined with Programmed Cell Death-1 Checkpoint Blockade (anti-PD1) for the Treatment of BRAF-altered Pediatric Gliomas
*For patients with BRAF or NF-1 alterations
ClinicalTrials.gov Identifier: NCT06712875
Description:
- This is a pilot study evaluating the toxicity and early efficacy of dabrafenib and/or trametinib combined with nivolumab for the treatment of BRAF-altered or NF altered gliomas.
Key Eligibility Criteria:
- Cohort A: Patients ≥1 and ≤26 years of age with a recurrent or progressive low grade glioma harboring a KIAA1549-BRAF fusion, or a newly diagnosed high grade glioma harboring a KIAA1549-BRAF fusion
- Cohort B: Patients ≥1 and ≤26 years of age with NF-1 associated or altered low-grade glioma that is recurrent or progressive, high-grade glioma that is newly diagnosed or recurrent, or transforming glioma that is newly diagnosed or recurrent
Sponsor:
- Ann & Robert H Lurie Children's Hospital of Chicago
Investigator:
JACOB: Joint Administration of cDNA for TP53, checkpOint inhibition and Boost/Hypofractionated radiation. A phase 0/1 study in children with recurrent, progressive or refractory CNS malignancies
Description:
- This study will assess the maximum tolerated dose and/or the recommended phase II dose (RP2D) of SGT-53 in conjunction with hypofractionated radiotherapy and immune checkpoint blockade in children with recurrent, progressive, or refractory CNS malignancies
Key Eligibility Criteria:
- Children aged ≥3yr to ≤21 years old with recurrent, progressive or refractory CNS malignancy
Sponsor:
Investigator:
- Eugene Hwang, MD
UPCOMING: PNOC-023: Open label Phase 1 and Target Validation study of ONC206 in Children and Young Adults with Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Central Nervous System (CNS) Tumors o Description: This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors
*Biopsy required
ClinicalTrials.gov Identifier: NCT04732065
Description:
- This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors.
Key Eligibility Criteria:
- Arm A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy
- Arm B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) with a diagnosis of DMG, H3K27 altered are eligible, including spinal cord DMGs
- Arm C: Children and young adults with DMGs (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression and are recommended to get re-irradiation
- Arm D: Children and young adults with recurrent primary malignant CNS tumors (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression
Sponsor:
- Sabine Mueller, MD, PhD
Investigator:
Disease Status: Newly Diagnosed
Firefly-2: A Phase 3, Randomized, International Multicenter Trial Of DAY101 Monotherapy Versus Standard Of Care Chemotherapy In Patients With Pediatric Low-Grade Glioma Harboring An Activating RAF Alteration Requiring First-Line Systemic Therapy
ClinicalTrials.gov Identifier: NCT05566795
Description
- Comparing the objective response rates of DAY101 monotherapy versus standard of care (SoC) chemotherapy in patients with pediatric low-grade glioma harboring an activating RAF alteration
Key Eligibility Criteria
- Low grade gliomas who has not been treated prior
- Tumor must harbor a RAF alteration
Sponsor
- Day101 Pharmaceuticals
Investigator
Disease Type
- Low grade glioma
Disease Status: Recurrent
PNOC-021: A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas and High Grade Gliomas
ClinicalTrials.gov Identifier: NCT04485559
Description
- This will be the first study to determine the optimal dosing schedule and probable side effects for a combination therapy of trametinib and everolimus in pediatric LGG.
Key Eligibility Criteria
- Participants must have histologically confirmed diagnosis of a LGG (WHO grade I-II) that is recurrent or progressive after prior treatment
- Participants who have had surgery alone are not eligible
Sponsor
- Pacific Neuro-Oncology Consortium
Investigator
Disease Type
- Low grade glioma
- High grade glioma
LCH24C02: A Pilot Study Evaluating the Toxicity and Clinical Benefit of Mitogen-activated Protein Kinase (MAPK) Pathway Inhibition Combined with Programmed Cell Death-1 Checkpoint Blockade (anti-PD1) for the Treatment of BRAF-altered Pediatric Gliomas
*For patients with BRAF or NF-1 alterations
ClinicalTrials.gov Identifier: NCT06712875
Description:
- This is a pilot study evaluating the toxicity and early efficacy of dabrafenib and/or trametinib combined with nivolumab for the treatment of BRAF-altered or NF altered gliomas.
Key Eligibility Criteria:
- Cohort A: Patients ≥1 and ≤26 years of age with a recurrent or progressive low grade glioma harboring a KIAA1549-BRAF fusion, or a newly diagnosed high grade glioma harboring a KIAA1549-BRAF fusion
- Cohort B: Patients ≥1 and ≤26 years of age with NF-1 associated or altered low-grade glioma that is recurrent or progressive, high-grade glioma that is newly diagnosed or recurrent, or transforming glioma that is newly diagnosed or recurrent
Sponsor:
- Ann & Robert H Lurie Children's Hospital of Chicago
Investigator:
Disease Status: Recurrent
CONNECT 1906: Phase 2 trial of a novel peptide vaccine (PEP-CMV) targeting CMV antigen for newly diagnosed pediatric high-grade glioma and diffuse intrinsic pontine glioma and recurrent medulloblastoma
ClinicalTrials.gov Identifier: NCT05096481
Description:
- This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB).
Key Eligibility Criteria:
- Patients aged ≥3 and ≤39 years with recurrent/progressive medulloblastoma, newly diagnosed high grade glioma, or newly diagnosed diffuse intrinsic pontine glioma (biopsy not required)
Sponsor:
- Nationwide Children's Hospital
Investigator:
PNOC-027 : A pilot trial of real time drug screening and genomic testing to determine an individualized treatment plan in children and young adults with relapsed medulloblastoma
ClinicalTrials.gov Identifier: NCT05057702
Description
- The current study will use a new treatment approach based on the molecular characteristics of each participant’s tumor.
Key Eligibility Criteria
- Participants must undergo surgery and have surgically accessible disease
- Participants must have recurrent medulloblastoma
- Participants must have had at leapt one prior therapy
Sponsor
- Pacific Neuro-Oncology Consortium
Investigator
MCC23221: Evaluation of Digoxin for Relapsed Non-WNT, Non-SHH Medulloblastoma
*For non-WNT, non-SHH Medulloblastoma
ClinicalTrials.gov Identifier: NCT06701812
Description:
- The purpose of this study is to evaluate the efficacy of digoxin in treating relapsed non-SHH, non-WNT medulloblastoma in pediatric and young adult patients.
Key Eligibility Criteria:
- Patients >12 months and ≤ 30 years with relapsed non-WNT, non-SHH medulloblastoma
Sponsor:
- National Pediatric Cancer Foundation
Investigator:
JACOB: Joint Administration of cDNA for TP53, checkpOint inhibition and Boost/Hypofractionated radiation. A phase 0/1 study in children with recurrent, progressive or refractory CNS malignancies
Description:
- This study will assess the maximum tolerated dose and/or the recommended phase II dose (RP2D) of SGT-53 in conjunction with hypofractionated radiotherapy and immune checkpoint blockade in children with recurrent, progressive, or refractory CNS malignancies
Key Eligibility Criteria:
- Children aged ≥3yr to ≤21 years old with recurrent, progressive or refractory CNS malignancy
Sponsor:
Investigator:
- Eugene Hwang, MD
UPCOMING: PNOC-020: A Phase I/II Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM)
ClinicalTrials.gov Identifier: NCT04573140
Description:
- The primary objective will be to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in (Stratum 1) adult patients with newly diagnosed GBM (MGMT low level or unmethylated in adults only) and (Stratum 2) in pediatric patients with newly diagnosed HGG (pHGG).
Key Eligibility Criteria:
- Patients age > 3 and < 25 years with newly diagnosed high grade glioma, prior to surgical resection and radiation
Sponsor:
- University of Florida
Investigator:
UPCOMING: PNOC-023: Open label Phase 1 and Target Validation study of ONC206 in Children and Young Adults with Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Central Nervous System (CNS) Tumors o Description: This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors
*Biopsy required
ClinicalTrials.gov Identifier: NCT04732065
Description:
- This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors.
Key Eligibility Criteria:
- Arm A: Children and young adults with DMG, H3K27 altered (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who completed at least one line of prior therapy
- Arm B: Newly diagnosed children and young adults (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) with a diagnosis of DMG, H3K27 altered are eligible, including spinal cord DMGs
- Arm C: Children and young adults with DMGs (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression and are recommended to get re-irradiation
- Arm D: Children and young adults with recurrent primary malignant CNS tumors (Dose escalation: 2-21 years of age; Dose expansion: 2 years of age and above) who have evidence of progression but have not been treated for this progression
Sponsor:
- Sabine Mueller, MD, PhD
Investigator:
UPCOMING: MATCHPOINT: Medulloblastoma Adoptive T Cell Therapy, DC Vaccines, and Hematopoietic Stem Cells Combined With Immune checkPOINT Blockade
*For non-WNT, non-SHH Medulloblastoma
ClinicalTrials.gov Identifier: NCT06514898
Description:
- This is a pilot study in a small number of children and young adults with relapsed/progressive medulloblastoma (MB) looking at the feasibility and safety of adoptive cell therapy plus PD-1 blockade.
Key Eligibility Criteria:
- Patients age 4-30 years with suspected recurrence/progression of Group 3 or 4 (non-SHH/non-WNT) medulloblastoma since completion of definitive focal +/- craniospinal irradiation who are a candidate for surgical resection or biopsy
Sponsor:
- University of Florida
Investigator:
IMPACT: Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)
ClinicalTrials.gov Identifier: NCT06193759
Description:
- In this study, individualized TSA-T cells will be generated against proteogenomically determined tumor-specific antigens after standard of care treatment in children less than 5 years of age with embryonal brain tumors.
Key Eligibility Criteria:
- Patients ˂ 5 years of age with newly diagnosed embryonal tumors: medulloblastoma, embryonal tumor with multilayered rosettes (ETMR), pineoblastoma, atypical teratoid/rhabdoid tumor, and embryonal tumor, not otherwise specified (NOS)
Sponsor:
- Children's National Research Institute
Investigator:
PEPN2121: A Phase 1/2 Study of Tiragolumab and Atezolizumab in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
ClinicalTrials.gov Identifier: NCT05286801
Description
- A Phase 1/2 study to evaluate the safety of tiragolumab as monotherapy in pediatric patients (<18 years) with SMARCB1 or SMARCA4 deficient tumors (Part A), to evaluate antitumor activity of the combination of tiragolumab and atezolizumab in patients with SMARCB1 or SMARCA4 deficient tumors (Part B), and to evaluate the safety and adverse event profile of this combination therapy in subjects with SMARCB1 or SMARCA4 deficient tumors, with a particular focus in pediatric patients < 12 years of age.
Key Eligibility Criteria
- Patients must have SMARCB1 (INI1) or SMARCA4 deficient tumors
- Part A age: ≥ 12 years and < 18 years
- Part B age: ≥ 18 years
Sponsor
- Children's Oncology Group
Investigator
Disease Type
- Renal medullary carcinoma, malignant rhabdoid tumor (extra-CNS), atypical teratoid rhabdoid tumor (CNS), poorly differentiated chordoma, epithelioid sarcoma, other SMARCB1 or SMARCA4 deficient tumors
PEPN22P1: A pharmacokinetic study of vincristine in infants dosed according to BSA-banded infant dosing tables and older children dosed by traditional BSA methods, patients 0-12mo
ClinicalTrials.gov Identifier: NCT05359237
Description
- A correlative study of vincristine pharmacokinetics to evaluate a new infant dosing method and determine whether it results in uniform drug exposure (AUC) across the age groups.
Key Eligibility Criteria
- Age: ≤ 12 years
- Newly diagnosed and relapsed cancer diagnosis that is being treated with vincristine at the 1.5 mg/m2 dose level
Sponsor
- Children's Oncology Group
Investigator
Disease Type
- CNS tumors