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Trial record 28 of 78 for:    DIPG

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

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ClinicalTrials.gov Identifier: NCT02742883
Recruitment Status : Active, not recruiting
First Posted : April 19, 2016
Last Update Posted : August 9, 2019
Sponsor:
Information provided by (Responsible Party):
Burzynski Research Institute

Tracking Information
First Submitted Date  ICMJE April 12, 2016
First Posted Date  ICMJE April 19, 2016
Last Update Posted Date August 9, 2019
Study Start Date  ICMJE April 2016
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 14, 2016)
Efficacy Measured by Objective Response Rate [ Time Frame: 104 weeks ]
Objective Response Rate
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02742883 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 14, 2016)
Efficacy Measured by Overall Survival [ Time Frame: 6 months, 12 months, and 24 months ]
6 months, 12 months, and 24 months overall survival
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of Atengenal and Astugenal in Diffuse, Intrinsic Pontine Glioma (DIPG)
Official Title  ICMJE A Phase 2 Study of Atengenal (A-10) and Astugenal (AS2-1) in Diffuse, Intrinsic Pontine Glioma (DIPG)
Brief 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 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Diffuse, Intrinsic Pontine Glioma
Intervention  ICMJE Drug: Antineoplaston therapy (Atengenal + Astugenal)
Patients with diffuse intrinsic pontine glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Other Name: A10 (Atengenal); AS2-1 (Astugenal)
Study Arms  ICMJE 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.
Intervention: Drug: Antineoplaston therapy (Atengenal + Astugenal)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: May 10, 2016)
1
Original Estimated Enrollment  ICMJE
 (submitted: April 14, 2016)
50
Estimated Study Completion Date  ICMJE February 2022
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Months to 99 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02742883
Other Study ID Numbers  ICMJE BRI-BT-55
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Burzynski Research Institute
Study Sponsor  ICMJE Burzynski Research Institute
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Stanislaw R. Burzynski, MD, PhD Burzynski Research Institute
PRS Account Burzynski Research Institute
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP