Diffuse, Intrinsic Pontine GliomaPhase 2NCT02742883
A Study of Atengenal and Astugenal in Diffuse, Intrinsic Pontine Glioma (DIPG)
A Phase 2 study testing Antineoplaston therapy (Atengenal + Astugenal) in people with Diffuse, Intrinsic Pontine Glioma, run by Burzynski Research Institute at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 3 months to 99 years
- Conditions the study is for
- Diffuse, Intrinsic Pontine Glioma
- What is being tested?
- Antineoplaston therapy (Atengenal + Astugenal)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Apr 2016to Mar 2028
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Conditions
- Diffuse, Intrinsic Pontine Glioma
- Being tested
- Antineoplaston therapy (Atengenal + Astugenal)
- Medical subject headings
- Diffuse Intrinsic Pontine Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Antineoplaston therapy (Atengenal + Astugenal)Drug
Also called A10 (Atengenal); AS2-1 (Astugenal)
Patients with diffuse intrinsic pontine glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
| Study group | Role | What they receive |
|---|---|---|
| Antineoplaston therapy | Experimental | 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. Receives: Drug: Antineoplaston therapy (Atengenal + Astugenal) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy Measured by Objective Response Rate
Objective Response Rate
Measured: 104 weeks
Secondary outcomes (1)
Efficacy Measured by Overall Survival
6 months, 12 months, and 24 months overall survival
Measured: 6 months, 12 months, and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2016 (estimated)
- Study start13 Apr 2016
- Primary completionMar 2028 (estimated)
- Study completionMar 2028 (estimated)
Why it stopped: Clinical Hold
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diffuse, Intrinsic Pontine GliomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Burzynski Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Stanislaw R. Burzynski, MD, PhD, Principal investigator, Burzynski Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02742883
- Sponsor's protocol ID
- BRI-BT-55
- Status last verified
- Sep 2025
- First submitted
- 12 Apr 2016
- Last updated
- 16 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of Atengenal (A-10) and Astugenal (AS2-1) in Diffuse, Intrinsic Pontine Glioma (DIPG)
- ClinicalTrials.gov ID
- NCT02742883
- Acronym / sponsor ID
- BRI-BT-55
- Conditions
- Diffuse, Intrinsic Pontine Glioma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Clinical Hold
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Burzynski Research InstituteUniversity, hospital or other
- Study start
- 13 Apr 2016 (actual)
- Primary completion
- Mar 2028 (estimated)
- Study completion
- Mar 2028 (estimated)
- First posted
- 19 Apr 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Antineoplaston therapy | Experimental | Drug: Antineoplaston therapy (Atengenal + Astugenal)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. |
Who may qualify
- Ages
- 3 months to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Antineoplaston therapy (Atengenal + Astugenal)Also: A10 (Atengenal); AS2-1 (Astugenal)Patients with diffuse intrinsic pontine glioma will receive Antineoplaston therapy (Atengenal + Astugenal). | Drug | Antineoplaston therapy |
Primary outcome
Efficacy Measured by Objective Response Rate
Time frame: 104 weeks
Objective Response Rate
1 secondary outcome
Efficacy Measured by Overall Survival
Time frame: 6 months, 12 months, and 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2025.
- Burzynski Research InstituteLink provided by the sponsor.
- Burzynski ClinicLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stanislaw R. Burzynski, MD, PhD principal investigator
Burzynski Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Antineoplaston therapy (Atengenal + Astugenal) in people with Diffuse, Intrinsic Pontine Glioma, run by Burzynski Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy Measured by Objective Response Rate (time frame: 104 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2025.