- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02742883
A Study of Atengenal and Astugenal in Diffuse, Intrinsic Pontine Glioma (DIPG)
A Phase 2 Study of Atengenal (A-10) and Astugenal (AS2-1) in Diffuse, Intrinsic Pontine Glioma (DIPG)
Current therapies for diffuse, intrinsic pontine glioma (DIPG) provide very limited benefit to the patient. The rationale for the use of Antineoplaston therapy in this protocol study derives from experience with subjects from prior Phase 2 studies and Compassionate Exemption patients treated with Antineoplaston therapy at the Burzynski Clinic.
This study is designed to analyze the efficacy and safety of Antineoplaston therapy in five separate DIPG patient cohorts, which are defined by age and prior therapy. This is a two stage study with 20 patients in each cohort being enrolled in the first stage and an additional 20 patients being enrolled in the second stage, if pre-determined efficacy endpoints in the first stage are realized.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
This study is designed to analyze the response of diffuse, intrinsic brainstem glioma (DIPG) to Antineoplaston therapy in 5 separate patient cohorts, which are defined by age and prior therapy. The primary endpoint is objective response (OR), but the determination of OR in DIPG is problematic. Determination of OR using the Macdonald, RANO, and RECIST criteria relies on postgadolinium TI-weighted MRI images of enhancing disease. However, DIPG shows variable enhancement and has a non-enhancing component as seen on T2/FLAIR-weighted MRI images. Recent reviews have proposed OR assessment in diffuse low grade gliomas using modified RANO criteria and in recurrent glioblastoma using RECIST + F (T2/FLAIR-weighted images).Neither of these methodologies have been validated.
This single-arm Phase 2 study of the efficacy of Antineoplaston therapy in DIPG will utilize both bidimensional assessment of enhancing DIPG (RANO), a validated primary endpoint, and unidimensional assessment of enhancing + non-enhancing DIPG (RECIST + F), an exploratory endpoint.
This Phase 2 protocol has a strategy for early assessment of response rates with procedures for termination of enrollment for lack of efficacy. A minimax two-stage design is utilized.
Ten patients with enhancing disease will be enrolled in stage 1 for each cohort. If none of the 10 patients in a particular cohort achieves an OR based on bidimensional measurements, no additional patients with enhancing disease will be enrolled into that particular cohort. However, if 1 of the 10 patients with enhancing disease in a particular cohort achieves an OR, then an additional 10 patients with enhancing disease will be enrolled in stage 2 of the minimax design for that cohort, yielding a maximum of 20 patients with enhancing disease for that cohort. As exploratory endpoints, complete response (CR), partial response (PR), and progressive disease (PD) rates, as well as 6-, 12-, and 24-month overall survival (OS) will be analyzed.
Patients with no enhancing disease will also be enrolled into each cohort but will not count against the numbers designated in the two-stage minimax design. An exploratory endpoint for these patients will be 6-, 12-, and 24-month OS.
Other exploratory endpoints will be determination of OR, CR, PR, and PD based on unidimensional measurement of the enhancing + non-enhancing components of DIPG (RECIST + F).
At completion of this study, the efficacy of Antineoplaston therapy in each cohort and the desirability of its further development in any particular cohort will be determined in discussion with the FDA.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Texas
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Houston, Texas, United States, 77055-6330
- Burzynski Clinic
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
Five cohorts of patients with diffuse, intrinsic pontine glioma will be studied:
- Patients age 3 months to < 3 years;
- Patients age 3-21 years with progressive disease (PD) following radiation therapy (RT) ± chemotherapy and/or other therapies;
- Patients age 3-21 years with newly diagnosed DIPG who (or whose parents / guardians) have refused RT;
- Patients age > 21 years with PD following RT ± chemotherapy and/or other therapies; and
- Patients age > 21 years with newly diagnosed DIPG who have refused RT.
Exclusion Criteria include:
- Disseminated disease, multicentric tumors, or leptomeningeal disease;
- Uncontrolled intercurrent illness;
- A history of New York Heart Association Class II congestive heart failure or above;
- Pregnancy.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Antineoplaston therapy
Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for up to 104 days.
Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dosage is reached, but not exceeding 12.0 g/kg/d Atengenal or 0.4 mg/kg/d Astugenal.
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Patients with diffuse intrinsic pontine glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Efficacy Measured by Objective Response Rate
Time Frame: 104 weeks
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Objective Response Rate
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104 weeks
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Efficacy Measured by Overall Survival
Time Frame: 6 months, 12 months, and 24 months
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6 months, 12 months, and 24 months overall survival
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6 months, 12 months, and 24 months
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Collaborators and Investigators
Sponsor
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Anticipated)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- BRI-BT-55
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Diffuse, Intrinsic Pontine Glioma
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National Cancer Institute (NCI)RecruitingRecurrent Malignant Glioma | Recurrent Medulloblastoma | Refractory Malignant Glioma | Refractory Medulloblastoma | Recurrent Diffuse Intrinsic Pontine Glioma | Recurrent Primary Central Nervous System Neoplasm | Refractory Primary Central Nervous System Neoplasm | Refractory Diffuse Intrinsic Pontine...United States, Canada
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Sidney Kimmel Comprehensive Cancer Center at Johns...Solving Kids' CancerCompletedDiffuse Intrinsic Pontine Glioma (DIPG)United States
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University of California, San FranciscoTranslational Genomics Research InstituteCompletedDiffuse Intrinsic Pontine Glioma (DIPG)United States
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University of California, San FranciscoNational Institute of Neurological Disorders and Stroke (NINDS); The Chad-Tough... and other collaboratorsRecruitingDiffuse Intrinsic Pontine Glioma | Diffuse Midline Glioma, H3 K27M-Mutant | Recurrent Diffuse Intrinsic Pontine Glioma | Recurrent Diffuse Midline Glioma, H3 K27M-Mutant | Recurrent WHO Grade III Glioma | WHO Grade III GliomaUnited States, Israel, Australia, Netherlands, Switzerland, New Zealand
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University of FloridaAccelerate Brain Cancer Cure; Lyla Nsouli FoundationActive, not recruitingBrain Stem Glioma | Diffuse Intrinsic Pontine Glioma (DIPG)United States
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Shenzhen Geno-Immune Medical InstituteShenzhen Children's Hospital; Shenzhen Hospital of Southern Medical UniversityUnknownDiffuse Intrinsic Pontine Glioma or GlioblastomaChina
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Hadassah Medical OrganizationCompletedPediatric Malignant Brain Tumor -Diffuse Intrinsic Pontine GliomaIsrael
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Pediatric Brain Tumor ConsortiumNational Cancer Institute (NCI)CompletedMalignant Glioma | Recurrent Childhood Ependymoma | Recurrent Medulloblastoma | Recurrent Diffuse Intrinsic Pontine Glioma | Recurrent Atypical Teratoid/Rhabdoid Tumor | Refractory Diffuse Intrinsic Pontine Glioma | CNS Embryonal Tumor, Not Otherwise SpecifiedUnited States
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Cheng-Chia (Fred) WuFocused Ultrasound FoundationActive, not recruitingDiffuse Intrinsic Pontine Glioma | Diffuse Midline Glioma, H3 K27M-Mutant | Diffuse Pontine and Thalamic GliomasUnited States
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Ralph NULL SalloumChildren's Hospital Medical Center, CincinnatiRecruitingHigh Grade Glioma | Medulloblastoma | Meningioma | Anaplastic Ependymoma | Recurrent Malignant Glioma | Recurrent Medulloblastoma | Refractory Malignant Glioma | Refractory Medulloblastoma | Recurrent Diffuse Intrinsic Pontine Glioma | Recurrent Primary Central Nervous System Neoplasm | Refractory Primary... and other conditionsUnited States
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