Neoplasms

Nervous System Diseases

All Conditions

Behaviors and Mental Disorders

Brain Neoplasms

Central Nervous System Neoplasms

Nervous System Neoplasms

Brain Diseases

Central Nervous System Diseases

Quality of Life

PROMIS pediatric domains for emotional distress (anxiety and depression), physical function (fatigue, pain interference, mobility and upper extremity), and peer relations

PROMIS adult domains. To reduce respondent burden, the multi-form design will be used which includes the short form of physical function, anxiety, depression, fatigue, sleep disturbance, ability to participate in social roles and activities, pain interference and pain intensity.

Parent/guardian will complete the parental proxy PROMIS instruments based on corresponding child age (ages 5 to 17 years)

PROMIS questionnaires completed every 3 months for up to 2 years.

PROMIS-QOL

Mariko DeWire, MD

In this research study the investigators want to learn more about the quality of life before, during and after cancer treatment in patients with central nervous system brain tumors. Often CNS tumors and cancer treatment can cause many physical and emotional problems and side effects. Some of these problems and treatment side effects can cause a change in a patient's qualify of life and overall well-being. Quality of life questionnaires are used to measure well-being and ability to carry out daily activities by asking patients to answer several questions about their physical, emotional, and social well-being.

In this research study we want to find out if patient's answers to these questions change over the course of your treatment. We also want to see if doctors and nurses can use these answers to the questions to help patients feel better and increase their activity during cancer treatment.

Pediatric CNS tumors are the second most common form of pediatric cancer and the leading cause of death related to pediatric malignancies. Over decades of work and through the efforts of collaborative groups, cure rates have increased significantly. However, various types of CNS malignancy outcomes have remained stagnant. Moreover, side effects from treatments of even the most curable CNS tumors may have dramatic short and long-term sequela ranging from cognitive, endocrine malfunction, functional mobility, neurological, and ophthalmologic compromises. As science and protocol directed therapies continue to find cures for these patients, work also must continue in efforts to explore patient reported outcomes (PROs) and health related quality of life (HRQOL) throughout the trajectory of a patient's disease process. Increased efforts in patient reported outcomes will lead to improvements in symptom management, functional status, and overall quality of life (QOL).

Health Related Quality of Life in Pediatric Central Nervous System (CNS) Tumors: A Feasibility Study Utilizing PROMIS

Alex's Lemonade Stand Foundation

Children's Hospital Medical Center, Cincinnati

Rare Diseases

Glioblastoma

Astrocytoma

Glioma

Neoplasms, Neuroepithelial

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Brain Tumor, Adult

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Temozolomide

Immunomodulating Agents

The only arm will receive the DRI modified gamma delta T cells following standard therapy with radiation and temozolomide chemotherapy concurrent.

Drug Resistant Immunotherapy with gamma delta modified T cells to be resistance to temozolomide will be infused into the surgical cavity following the completion of standard concurrent radiation and chemotherapy with temozolomide.

DRI cell therapy

Louis B Nabors, MD

This study is being conducted to find out if the safety and tolerability of an experimental cell therapy is safe to administer to patients with a newly diagnosed glioblastoma multiforme (GBM) in combination with temozolomide (TMZ).

The cell therapy that is being used in this study is called gene-modified gamma delta T cells or Drug Resistant Immunotherapy (DRI). Gamma delta T cells are a type of lymphocyte or white blood cell that may help the immune system to recognize and kill cancer cells. Some chemotherapy can kill these gamma delta T cells. For the cell therapy being used in this study, the genes or DNA within these gamma delta T cells are modified so they are resistant to or may not be affected by chemotherapy. The chemotherapy that is being used in this study is called temozolomide (TMZ) and can reduce the number of these gamma delta T cells or lymphocytes. This study will also find out if these modified gamma delta T cells (DRI) are unaffected by TMZ. The DRI is given in combination with a standard dose of TMZ and is administered into the brain where the tumor is located.

After subjects are consented in Parts A and B of the study, approximately about 2 tablespoons of blood will be collected for tests that may provide additional information about subjects T cells and how they may respond to treatment.

Part A Subjects will have a surgical procedure completed that will remove their tumor (called a surgical resection). They will have a Rickham catheter placed which is a device typically used to deliver chemotherapy into the brain. The infusion catheter will be placed first prior to the tumor resection. A sample of tumor tissue will be taken and examined to confirm the diagnosis of GBM. The diagnosis of GBM must be confirmed prior to beginning Part B.

Part B After the surgical resection and on Study Day 1, subjects will return to the study doctor's office/clinic to undergo a procedure called apheresis. This procedure will separate out cells called peripheral blood mononuclear cells (PBMC). Apheresis is the removal of blood plasma from the body by collecting your blood and separating the plasma from the PBMCs. These cells include the gamma delta T cells that will be used to synthesize the DRI γδ T cells. Once the cells for the DRI γδ T cells are synthesized, they are reintroduced into subject's brain through the Rickham catheter.

Following apheresis and confirmation that the required number of gamma delta T cells were collected, subjects will begin the recommended or standard of care treatment for newly diagnosed GBM. This will include 6 weeks of chemotherapy with TMZ and radiation. Subjects will then have about 4 weeks of no treatment prior to beginning the Part B maintenance phase of treatment, which includes 6 cycles of TMZ. Depending on which dose level they receive, they will be administered either 1 injection of the DRI γδ T cells or 3 and these will be injected through the Rickham catheter.

The first 3 subjects will receive a single dose of the DRI γδ T cells at the lowest dose level. Subjects receiving a single dose of the DRI γδ T cells will be observed for a of minimum 30 days from the time the first subject is enrolled, followed by a minimum of 7 days between each additional subject enrolled to allow for evaluation of potential side effects. If the DRI γδ T cell injection does not cause serious side effects, the second set of 3 subjects will be enrolled. The second set of 3 patients will receive 3 doses of the DRI γδ T cells. The approximate duration of the study may be up to 15 years, or until disease progression or subjects withdraw from the study.

Novel Gamma-Delta (γδ)T Cell Therapy for Treatment of Patients With Newly Diagnosed Glioblastoma

A Phase I Study of Drug Resistant Immunotherapy (DRI) With Activated, Gene Modified γδ T Cells in Patients With Newly Diagnosed Glioblastoma Multiforme Receiving Maintenance Temozolomide Chemotherapy

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Health Related Quality of Life in Pediatric Central Nervous System (CNS) Tumors: A Feasibility Study Utilizing PROMIS	NCT02603692	Brain Neoplasms	PROMIS-QOL	5 Years - 35 Years	Children's Hospital Medical Center, Cincinnati
Novel Gamma-Delta (γδ)T Cell Therapy for Treatment of Patients With Newly Diagnosed Glioblastoma	NCT04165941	Brain Tumor Adu...	DRI cell therap...	18 Years -	University of Alabama at Birmingham
Health Related Quality of Life in Pediatric Central Nervous System (CNS) Tumors: A Feasibility Study Utilizing PROMIS	NCT02603692	Brain Neoplasms	PROMIS-QOL	5 Years - 35 Years	Children's Hospital Medical Center, Cincinnati

Spots Global Cancer Trial Database for newly diagnosed brain tumor