Brain Tumor Clinical Trials

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:

• Eugene Hwang, MD 

*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:

• Eugene Hwang, MD

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:

• Eugene Hwang, MD

ClinicalTrials.gov Identifier: NCT03033992

Description

• The Optune Device produce intermediate frequency electric fields impair the growth of tumor cells through the arrest of cell division and inducing apoptosis.

Key Eligibility Criteria

• Stratum 1 for patients with supratentorial high grade glioma or ependymoma
• Stratum 2 for patients with newly diagnosed DIPG both for ages 5-21
• For stratum 2 patients must not have had any prior antitumor therapy or radiation 

Sponsor

• Pediatric Brain Tumor Consortium

Investigator

• Eugene Hwang, MD

Disease Type

• Recurrent or progressive supratentorial high grade glioma or ependymoma, and or patients with newly diagnosed DIPG

ClinicalTrials.gov Identifier: NCT04978727

Description

• The primary objective is to assess the toxicity profile of SurVaxM Vaccines in emulsion with Montanide plus sargramostim in children with relapsed or progressive medulloblastoma and high grade glioma (HGG), ependymoma and non-recurrent diffuse intrinsic pontine glioma (DIPG) post-radiation therapy (RT)

Key Eligibility Criteria

• Demonstration of survivin expression as assessed after screening consent/assent of at least 1% on tumor tissue by immunohistochemistry
• Patients must be progressive or relapsed
• Patients must have received their last dose of known myelosuppressive anticancer therapy at least 21 days prior to enrollment

Sponsor

• Pediatric Brain Tumor Consortium

Investigator

• Eugene Hwang, MD

Disease Type

• Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma

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

• Lindsay Kilburn, M.D.

Disease Type

• Diffuse midline glioma (DMG)

*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:

• Lindsay Kilburn, MD

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 therapy
• Cohort B: Patients > 12 months and < 39 years of age with progressive / refractory DIPG or progressive / recurrent / refractory H3K27-altered HGG

Sponsor:

• Nationwide Children's Hospital

Investigator:

• Eugene Hwang, MD

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

• AeRang Kim, MD, PhD

Disease Type

• Solid tumors, lymphoma

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

• Lindsay Kilburn, M.D.

Disease Type

• Diffuse midline glioma (DMG)

*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:

• Lindsay Kilburn, MD

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 therapy
• Cohort B: Patients > 12 months and < 39 years of age with progressive / refractory DIPG or progressive / recurrent / refractory H3K27-altered HGG

Sponsor:

• Nationwide Children's Hospital

Investigator:

• Eugene Hwang, MD

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

• Lindsay Kilburn, M.D.

Disease Type

• Diffuse midline glioma (DMG)

ClinicalTrials.gov Identifier: NCT06413706

Description:

• The purpose of this study is to measure the benefit of adding abemaciclib to the chemotherapy, temozolomide, for newly diagnosed high-grade glioma following radiotherapy. 

Key Eligibility Criteria:

• Patients less than 21 years of age with biopsy proven high grade glioma

Sponsor:

• Eli Lilly and Company

Investigator:

• Lindsay Kilburn, MD

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:

• Eugene Hwang, MD 

*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:

• Eugene Hwang, MD

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:

• Eugene Hwang, MD

*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:

• Lindsay Kilburn, MD

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

• AeRang Kim, MD, PhD

Disease Type

• Solid tumors, lymphoma

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

• Lindsay Kilburn, MD

Disease Type

• Low grade glioma
• High grade glioma

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

• Lindsay Kilburn, M.D.

Disease Type

• Diffuse midline glioma (DMG)

*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:

• Lindsay Kilburn, MD

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 therapy
• Cohort B: Patients > 12 months and < 39 years of age with progressive / refractory DIPG or progressive / recurrent / refractory H3K27-altered HGG

Sponsor:

• Nationwide Children's Hospital

Investigator:

• Eugene Hwang, MD

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:

• Eugene Hwang, MD

ClinicalTrials.gov Identifier: NCT03033992

Description

• The Optune Device produce intermediate frequency electric fields impair the growth of tumor cells through the arrest of cell division and inducing apoptosis.

Key Eligibility Criteria

• Stratum 1 for patients with supratentorial high grade glioma or ependymoma
• Stratum 2 for patients with newly diagnosed DIPG both for ages 5-21
• For stratum 2 patients must not have had any prior antitumor therapy or radiation 

Sponsor

• Pediatric Brain Tumor Consortium

Investigator

• Eugene Hwang, MD

Disease Type

• Recurrent or progressive supratentorial high grade glioma or ependymoma, and or patients with newly diagnosed DIPG

ClinicalTrials.gov Identifier: NCT04903080

Description

• Ependymoma is the third most common central nervous system (CNS) tumor in children and is associated with poor long-term survival. This study evaluates the safety profile of a type of immune therapy called HER2 CAR T cells (short for HER2 chimeric antigen receptor T cells). In addition to looking for side effects, we will study how well this treatment works against ependymoma that has come back after treatment (recurrent) or has not responded well to treatment (progressive) in children.

Key Eligibility Criteria

• Diagnosis of ependymoma that is recurrent or progressive

• Patient must have received standard of care therapy including maximal safe surgical resection followed by local adjuvant radiation therapy prior to enrollment

• Patient must be ≥ 1 but ≤ 21 years of age at the time of screening consent.

Sponsor

• Pediatric Brain Tumor Consortium (PBTC)

Investigator

• Eugene I. Hwang, MD

Disease Type

• Refractory or Recurrent Ependymoma

ClinicalTrials.gov Identifier: NCT04978727

Description

• The primary objective is to assess the toxicity profile of SurVaxM Vaccines in emulsion with Montanide plus sargramostim in children with relapsed or progressive medulloblastoma and high grade glioma (HGG), ependymoma and non-recurrent diffuse intrinsic pontine glioma (DIPG) post-radiation therapy (RT)

Key Eligibility Criteria

• Demonstration of survivin expression as assessed after screening consent/assent of at least 1% on tumor tissue by immunohistochemistry
• Patients must be progressive or relapsed
• Patients must have received their last dose of known myelosuppressive anticancer therapy at least 21 days prior to enrollment

Sponsor

• Pediatric Brain Tumor Consortium

Investigator

• Eugene Hwang, MD

Disease Type

• Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma

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

• Lindsay Kilburn, MD

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:

• Eugene Hwang, MD 

Investigator:

• Eugene Hwang, MD

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

• Lindsay Kilburn, MD

Disease Type

• Low grade glioma

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

• Lindsay Kilburn, MD

Disease Type

• Low grade glioma
• High grade glioma

*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:

• Lindsay Kilburn, MD

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:

• Eugene Hwang, MD

ClinicalTrials.gov Identifier: NCT03904862

Description

• Use of oral CX-4945 which is a tetracyclic synthetically derived small molecule carboxylate acid salt that exhibits. potent and highly selective inhibition of Casein Kinase 2 (CK2).

Key Eligibility Criteria

• Recurrent or progressive medulloblastoma with the SHH subtype only
• Tissue is required to send to sponsor for confirmatory results of subgroup type
• Ages ≥ 3 and ≤ 18 years

Sponsor

• Pediatric Brain Tumor Consortium

Investigator

• Eugene Hwang, MD

Disease Type

• Sonic HedgeHog Medulloblastoma

ClinicalTrials.gov Identifier: NCT04978727

Description

• The primary objective is to assess the toxicity profile of SurVaxM Vaccines in emulsion with Montanide plus sargramostim in children with relapsed or progressive medulloblastoma and high grade glioma (HGG), ependymoma and non-recurrent diffuse intrinsic pontine glioma (DIPG) post-radiation therapy (RT)

Key Eligibility Criteria

• Demonstration of survivin expression as assessed after screening consent/assent of at least 1% on tumor tissue by immunohistochemistry
• Patients must be progressive or relapsed
• Patients must have received their last dose of known myelosuppressive anticancer therapy at least 21 days prior to enrollment

Sponsor

• Pediatric Brain Tumor Consortium

Investigator

• Eugene Hwang, MD

Disease Type

• Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma

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

• Lindsay Kilburn, MD

*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:

• Lindsay Kilburn, MD

*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:

• H. Lee Moffitt Cancer Center and Research Institute

Investigator:

• Lindsay Kilburn, MD
  
   

*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:

• Eugene Hwang, MD

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:

• Eugene Hwang, MD 

Investigator:

• Eugene Hwang, MD

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: 

• Brian Rood, MD

ClinicalTrials.gov Identifier: NCT05934630

Description:

• There is no treatment provided on this study. Patients who have CSF samples taken as part of regular care will be asked to provide extra samples for this study as a "liquid biopsy” for the detection of cell-free DNA (cfDNA).

Key Eligibility Criteria:

Patients ≤ 21 years of age, or ˂ 40 with a primary brain tumor more common in children than adults

• Stratum 1: Medulloblastoma 
• Stratum 2: High-grade glioma (IDH-wildtype) and DIPG 
• Stratum 3: Low-grade glioma (IDH-wildtype) with multifocal/disseminated and/or leptomeningeal disease 
• Stratum 4: Diffuse Leptomeningeal Glioneuronal Tumor Stratum 
• Stratum 5: Pineoblastoma 
• Stratum 6: All other eligible tumor types

Sponsor: 

• Pediatric Brain Tumor Consortium

Investigator:

• Eugene Hwang, MD

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

• AeRang Kim, MD

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

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

• AeRang Kim, MD, PhD

Disease Type

• CNS tumors