Antineoplaston Therapy in Treating Children With Primitive Neuroectodermal Tumors (PNET)
RATIONALE: Current therapies for children with primitive neuroectodermal tumors that have not responded to standard therapy provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primitive neuroectodermal tumors that have not responded to standard therapy.
PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) with primitive neuroectodermal tumors that has not responded to standard therapy.
|Childhood CNS Primitive Neuroectodermal Tumor||Drug: Antineoplaston therapy (Atengenal + Astugenal)||Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Study of Antineoplastons A10 and AS2-1 In Children With Primitive Neuroectodermal Tumors|
- Number of Participants With Objective Response [ Time Frame: 12 months ]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.
- Percentage of Participants Who Survived [ Time Frame: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months ]6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival
|Study Start Date:||April 1996|
|Study Completion Date:||February 2005|
|Primary Completion Date:||February 2005 (Final data collection date for primary outcome measure)|
Experimental: Antineoplaston therapy
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.
Drug: Antineoplaston therapy (Atengenal + Astugenal)
Children with a primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
Other Name: A10 (Atengenal); AS2-1 (Astugenal)
- To determine the efficacy of Antineoplaston therapy in children with primitive neuroectodermal tumors that has not responded to standard therapy, 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 a brain tumor.
OVERVIEW: This is a single arm, open-label study in which children with primitive neuroectodermal tumors that have not responded to standard therapy 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00003460
|United States, Texas|
|Houston, Texas, United States, 77055-6330|
|Principal Investigator:||Stanislaw R Burzynski, MD, PhD||Burzynski Research Institute|