Trial Outcomes & Findings for Antineoplaston Therapy in Treating Children With Recurrent or Refractory High-Grade Glioma (NCT NCT00003535)

Last Updated: 2018-03-21

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

12 months

Results posted on

2018-03-21

Participant Flow

Nine patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX

Participant milestones

Participant milestones
Measure	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. Antineoplaston therapy (Atengenal + Astugenal): Children with a recurrent/progressive high grade glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study STARTED	9
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	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. Antineoplaston therapy (Atengenal + Astugenal): Children with a recurrent/progressive high grade glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study Not evaluable	5

Baseline Characteristics

Antineoplaston Therapy in Treating Children With Recurrent or Refractory High-Grade Glioma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Antineoplaston Therapy n=9 Participants 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. Antineoplaston therapy (Atengenal + Astugenal): Children with a recurrent/progressive high grade glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Age, Continuous	9.7 Years n=93 Participants
Sex: Female, Male Female	1 Participants n=93 Participants
Sex: Female, Male Male	8 Participants n=93 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=4 Participants 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. Antineoplaston therapy (Atengenal + Astugenal): Children with a recurrent/progressive high grade glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Number of Participants With Objective Response Complete Response	1 Participants
Number of Participants With Objective Response Partial Response	1 Participants
Number of Participants With Objective Response Progressive Disease	2 Participants

SECONDARY outcome

Timeframe: 6 months, 12 months, 24 months

Population: All study subjects receiving any Antineoplaston therapy

6 months, 12 months, 24 months overall survival

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=9 Participants 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. Antineoplaston therapy (Atengenal + Astugenal): Children with a recurrent/progressive high grade glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Percentage of Participants Who Survived 6 months overall survival	22.2 Percentage of Participants
Percentage of Participants Who Survived 12 months overall survival	11.1 Percentage of Participants
Percentage of Participants Who Survived 24 months overall survival	0.0 Percentage of Participants

Adverse Events

Antineoplaston Therapy

Serious events: 7 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Antineoplaston Therapy n=9 participants at risk 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. Antineoplaston therapy (Atengenal + Astugenal): Children with a recurrent/progressive high grade glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Blood and lymphatic system disorders Platelets	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Central Venous Catheter: Infection	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Dehydration	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Vomiting	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection: Other	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Lung (pneumonia)	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypernatremia	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Seizure	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Somnolence/depressed level of consciousness	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Pain: Head/headache	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Respiratory, thoracic and mediastinal disorders Pleural effusion (non-malignant)	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX

Other adverse events

Other adverse events
Measure	Antineoplaston Therapy n=9 participants at risk 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. Antineoplaston therapy (Atengenal + Astugenal): Children with a recurrent/progressive high grade glioma will receive Antineoplaston therapy (Atengenal + Astugenal).
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Hemoglobin	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Leukocytes (total WBC)	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Lymphopenia	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	22.2% 2/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Platelets	33.3% 3/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Cardiac disorders Supraventricular and nodal arrhythmia: Sinus tachycardia	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Cardiac disorders Hypertension	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Central Venous Catheter: Infection	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fatigue (asthenia, lethargy, malaise)	33.3% 3/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fever	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Edema/Fluid retention	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Dehydration	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Diarrhea	22.2% 2/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Nausea	22.2% 2/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Vomiting	44.4% 4/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Renal and urinary disorders Hemorrhage, GU: Bladder	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection: Other	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Lung (pneumonia)	22.2% 2/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Mucosa	22.2% 2/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Upper airway NOS	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypercholesteremia	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hyperglycemia	22.2% 2/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypokalemia	88.9% 8/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypomagnesemia	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypophosphatemia	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Proteinuria	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypernatremia	55.6% 5/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Confusion	22.2% 2/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Seizure	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Somnolence/depressed level of consciousness	44.4% 4/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Speech impairment	22.2% 2/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Pain: Head/headache	11.1% 1/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	33.3% 3/9 • 3 years, 9 months Thirteen patients were recruited between April 1994 and August 1997. All study subjects were seen at the Burzynski Clinic in Houston TX

Additional Information

S. R. Burzynski, MD, PhD

Burzynski Research Institute, Inc.

Phone: 713-335-5664

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place