Symptoms and General Pathology

All Conditions

Neoplasms

Nervous System Diseases

Rare Diseases

Recurrence

Glioblastoma

Glioma

Brain Neoplasms

Astrocytoma

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Central Nervous System Neoplasms

Nervous System Neoplasms

Brain Diseases

Central Nervous System Diseases

Brain Tumor, Childhood

Anaplastic Astrocytoma

Neuroepithelioma

Childhood Brain Stem Glioma

Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for at least 12 months. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dose is reached.

Children with a primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).

Antineoplaston therapy (Atengenal + Astugenal)

Stanislaw R. Burzynski, MD, PhD

RATIONALE: Current therapies for children with primary malignant brain tumors provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primary malignant brain tumors.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children with primary malignant brain tumors.

OBJECTIVES:

* To determine the efficacy of Antineoplaston therapy in children with primary malignant brain tumors as measured by an objective response to therapy (complete response, partial response) or stable disease.
* To determine the safety and tolerance of Antineoplaston therapy in children with primary malignant brain tumors.

OVERVIEW: This is a single arm, open-label study in which children with primary malignant brain tumors receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues for at least 12 months in the absence of disease progression or unacceptable toxicity. After 12 months, patients with a complete or partial response or with stable disease may continue treatment.

To determine objective response, tumor size is measured utilizing MRI scans, which are performed every 8 weeks for the first two years, every 3 months for the third and fourth years, every 6 months for the 5th and sixth years, and annually thereafter.

PROJECTED ACCRUAL: A total of 20-40 patients will be accrued to this study

Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors

Phase II Study of Antineoplastons A10 and AS2-1 in Children With Primary Malignant Brain Tumors

Objective response rate per Response Assessment in Neuro-Oncology (RANO) for target lesions and assessed by MRI: Complete Response (CR), disappearance of all disease sustained for at least four weeks; Partial Response (PR), \>=50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least four weeks; Stable Disease (SD), \<50% decrease and \<25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least eight weeks; Progressive Disease (PD), \>=25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions.

Six months and Twelve months overall survival

Burzynski Research Institute

Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX

Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for at least 12 months. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dose is reached.

Antineoplaston therapy (Atengenal + Astugenal): Children with a primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).

Antineoplaston Therapy

Age, Continuous

Sex: Female, Male

S. R. Burzynski, MD, PhD

Partial Response

Stable Disease

Progressive Disease

Number of Participants With Objective Response

6 months overall survival

12 months overall survival

All study subjects receiving any Antineoplaston therapy

Percentage of Participants Who Survived

Overall Study

Blood and Lymph Conditions

Immune System Diseases

Lymphoma

Leukemia

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Hypersensitivity

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Sarcoma

Hodgkin Disease

Ependymoma

Rhabdomyosarcoma

Gliosarcoma

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Hematologic Diseases

Disease Attributes

Neoplasms, Connective and Soft Tissue

Myeloproliferative Disorders

Bone Marrow Diseases

Chronic Disease

Myosarcoma

Neoplasms, Neuroepithelial

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Myeloid Leukemia

Acute Myeloid Leukemia

Lymphosarcoma

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Chronic Graft Versus Host Disease

Acute Non Lymphoblastic Leukemia

Chronic Myeloid Leukemia

Soft Tissue Sarcoma

Hodgkin Lymphoma

Germ Cells Tumors

Acute Graft Versus Host Disease

Childhood Acute Lymphoblastic Leukemia

Malignant Germ Cell Tumor

Chronic Myeloproliferative Disorders

We intend to enroll chemorefractory or relapsed pediatric patients with all types of cancers where tumor tissue would be available for ex vivo drug screening and genomic profiling. The results of the drug sensitivity assay and genetic screening will be used to inform treating physician about patient-specific drug sensitivity or resistance guiding best therapy choices.

Diana Azzam, PhD

Daria Salyakina, PhD

This study is a prospective, non-randomized feasibility study. Freshly isolated tumor cells from patients will be screened using state-of-the-art viability assay designed for ex vivo high-throughput drug sensitivity testing (DST). In addition, genetic information will be obtained from cancer and normal (germline) tissue and correlated with drug response. This study will provide the platform for informing treating physician about individualized treatment options. The main outcome of this study will be the proportions of the patients whose treatment was guided by the personalized medicine approach.

PRIMARY OBJECTIVE: The primary objective of the study is to determine feasibility of providing pediatric cancer patients with access to personalized treatment options and clinical management recommendations based on ex vivo drug sensitivity testing (DST) and genomic profiling.

SECONDARY OBJECTIVE: The secondary objective of the study is to compare individual outcomes (response and disease-free survival) in patients with pediatric cancers treated with DST-guided therapy as compared to non-DST guided (conventional) therapy.

EXPLORATORY OBJECTIVE: To explore associations between genetic abnormalities in malignancies and ex vivo drug response.

Perform molecular and functional drug testing on blood, biopsy, bone marrow and tumor samples at relapse.

Ex Vivo Drug Sensitivity Testing and Mutation Profiling

Personalized Ex Vivo Drug Screening and Genomics Profiling to Guide Individualized Treatments for Children With Relapsed or Refractory Solid Tumors and Leukemias

This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a monotherapy or combination drug regimen based on functional and/or genomics data within 4 weeks in at least 16 out of 25 patients (64%).

To achieve at least 90% power, the null hypothesis will be rejected when at least 16 out of 25 patients receive treatment recommendations through functional and/or genomics data within 4 weeks on the study.

With that outcome, we would have 95% confidence that the true feasibility rate is at least 30% (95% CI: 0.425, 1).

We will assess changes in cohort Objective Response Rate (ORR) by comparing ORR in patients treated with FPM-guided therapy versus ORR in patients treated with non-FPM guided conventional therapy (standard of care)

We will assess changes in cohort PFS by comparing PFS in patients treated with FPM-guided therapy versus PFS in patients treated with non-FPM guided conventional therapy (standard of care)

We will assess changes in PFS from each patient's previous treatment versus their PFS from the treatment assigned during the trial. Assessments will be made both in the FPM-guided cohort and the non-FPM-guided conventional therapy cohort. Analysis will include both the raw ratio as well as the number of incidences of 30% improved PFS on trial versus previous regimen (PFS2/PFS1 \> 1.3x).

Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Florida International University

Assistant Professor

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors	NCT00003476	Childhood Brain...	Antineoplaston ...	6 Months - 18 Years	Burzynski Research Institute
Ex Vivo Drug Sensitivity Testing and Mutation Profiling	NCT03860376	Recurrent Child... Recurrent Child... Recurrent Child... Refractory Chil... Refractory Chil... Refractory Chil... Recurrent Child... Recurrent Child... Recurrent Child... Recurrent Child... Recurrent Child... Recurrent Child... Recurrent Child... Refractory Chro...		1 Day - 21 Years	Florida International University

Spots Global Cancer Trial Database for recurrent childhood brainstem glioma