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A Study of Atengenal and Astugenal in Diffuse, Intrinsic Pontine Glioma (DIPG)

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2016 by Burzynski Research Institute
Sponsor:
Information provided by (Responsible Party):
Burzynski Research Institute
ClinicalTrials.gov Identifier:
NCT02742883
First received: April 12, 2016
Last updated: April 28, 2016
Last verified: April 2016
  Purpose

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.


Condition Intervention Phase
Diffuse, Intrinsic Pontine Glioma
Drug: Antineoplaston therapy (Atengenal + Astugenal)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2 Study of Atengenal (A-10) and Astugenal (AS2-1) in Diffuse, Intrinsic Pontine Glioma (DIPG)

Resource links provided by NLM:


Further study details as provided by Burzynski Research Institute:

Primary Outcome Measures:
  • Efficacy Measured by Objective Response Rate [ Time Frame: 104 weeks ] [ Designated as safety issue: Yes ]
    Objective Response Rate


Secondary Outcome Measures:
  • Efficacy Measured by Overall Survival [ Time Frame: 6 months, 12 months, and 24 months ] [ Designated as safety issue: Yes ]
    6 months, 12 months, and 24 months overall survival


Estimated Enrollment: 50
Study Start Date: April 2016
Estimated Study Completion Date: February 2021
Estimated Primary Completion Date: March 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Drug: Antineoplaston therapy (Atengenal + Astugenal)
Patients with diffuse intrinsic pontine glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Other Name: A10 (Atengenal); AS2-1 (Astugenal)

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.

  Eligibility

Ages Eligible for Study:   3 Months to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Five cohorts of patients with diffuse, intrinsic pontine glioma will be studied:

  1. Patients age 3 months to < 3 years;
  2. Patients age 3-21 years with progressive disease (PD) following radiation therapy (RT) ± chemotherapy and/or other therapies;
  3. Patients age 3-21 years with newly diagnosed DIPG who (or whose parents / guardians) have refused RT;
  4. Patients age > 21 years with PD following RT ± chemotherapy and/or other therapies; and
  5. Patients age > 21 years with newly diagnosed DIPG who have refused RT.

Exclusion Criteria include:

  1. Disseminated disease, multicentric tumors, or leptomeningeal disease;
  2. Uncontrolled intercurrent illness;
  3. A history of New York Heart Association Class II congestive heart failure or above;
  4. Pregnancy.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02742883

Contacts
Contact: Stanislaw R. Burzynski, MD, PhD 713-335-5664 srb@burzynskiclinic.com

Locations
United States, Texas
Burzynski Clinic Recruiting
Houston, Texas, United States, 77055-6330
Contact: Stanislaw R. Burzynski, MD, PhD    713-335-5697      
Sponsors and Collaborators
Burzynski Research Institute
Investigators
Principal Investigator: Stanislaw R. Burzynski, MD, PhD Burzynski Research Institute
  More Information

Additional Information:
Responsible Party: Burzynski Research Institute
ClinicalTrials.gov Identifier: NCT02742883     History of Changes
Other Study ID Numbers: BRI-BT-55 
Study First Received: April 12, 2016
Last Updated: April 28, 2016
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Glioma
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Neuroectodermal Tumors

ClinicalTrials.gov processed this record on May 03, 2016