Trial Outcomes & Findings for Antineoplaston Therapy in Treating Patients With Brain Tumors (NCT NCT00003457)

Last Updated: 2017-08-22

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; Stable Disease (SD), \< 50% decrease and \< 25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least 8 weeks; Progressive Disease (PD), \>=25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions compared to the lowest sum recorded.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

40 participants

Primary outcome timeframe

12 months

Results posted on

2017-08-22

Participant Flow

Fourty patients were recruited between July 1996 and March 2011. 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): Adults with a persistent or recurrent brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study STARTED	40
Overall Study COMPLETED	31
Overall Study NOT COMPLETED	9

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): Adults with a persistent or recurrent brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study Not evaluable	9

Baseline Characteristics

Antineoplaston Therapy in Treating Patients With Brain Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Antineoplaston Therapy n=40 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): Adults with a persistent or recurrent brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Age, Continuous	36.0 Years n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=31 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): Adults with a persistent or recurrent brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Number of Participants With Objective Response Complete Response	4 Participants
Number of Participants With Objective Response Partial Response	5 Participants
Number of Participants With Objective Response Stable Disease	12 Participants
Number of Participants With Objective Response Progressive Disease	10 Participants

SECONDARY outcome

Timeframe: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months

Population: All study subjects receiving any Antineoplaston therapy

6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=40 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): Adults with a persistent or recurrent brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Percentage of Participants Who Survived 6 months overall survival	70.0 Percentage of participants
Percentage of Participants Who Survived 12 months overall survival	50.0 Percentage of participants
Percentage of Participants Who Survived 24 months overall survival	40.0 Percentage of participants
Percentage of Participants Who Survived 36 months overall survival	35.0 Percentage of participants
Percentage of Participants Who Survived 48 months overall survival	30.0 Percentage of participants
Percentage of Participants Who Survived 60 months overall survival	22.5 Percentage of participants

Adverse Events

Antineoplaston Therapy

Serious events: 27 serious events

Other events: 40 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Antineoplaston Therapy n=40 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): Adults with a persistent or recurrent brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Blood and lymphatic system disorders Hemoglobin	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Cardiac disorders Cardiac ischemia/infarction	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Cardiac disorders Pericardial effusion (non-malignant)	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Central venous catheter infection	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders INR (International Normalized Ratio of prothrombin time)	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Fatigue (asthenia, lethargy, malaise) Fatigue (asthenia, lethargy, malaise)	5.0% 2/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Skin and subcutaneous tissue disorders Bruising (in absence of Grade 3 or 4 thrombocytopenia)	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Endocrine disorders Pancreatic endocrine: glucose intolerance	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Obstruction, GI: Small bowel NOS	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Perforation, GI: Duodenum	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Hemorrhage, CNS	5.0% 2/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Hemorrhage, GI: Abdomen NOS	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection - Other	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Infection (documented clinically): Bladder (urinary)	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Blood	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Colon	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Lung (pneumonia)	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Skin (cellulitis)	5.0% 2/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Lung (pneumonia)	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypernatremia	5.0% 2/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Confusion	5.0% 2/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Neuropathy: motor	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Seizure	27.5% 11/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Somnolence/depressed level of consciousness	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Pain: Abdomen NOS	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Pain: Pain NOS	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Flu-like syndrome	2.5% 1/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Vascular disorders Thrombosis/thrombus/embolism	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing

Other adverse events

Other adverse events
Measure	Antineoplaston Therapy n=40 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): Adults with a persistent or recurrent brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	25.0% 10/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Ear and labyrinth disorders Tinnitus	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Hemoglobin	32.5% 13/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Leukocytes (total WBC)	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Lymphopenia	35.0% 14/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	17.5% 7/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Platelets	22.5% 9/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Cardiac disorders Hypertension	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Central venous catheter infection	32.5% 13/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Non-functional central venous catheter	32.5% 13/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Central venous catheter - Other	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Fatigue (asthenia, lethargy, malaise)	70.0% 28/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Fever	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Insomnia	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Rigors/chills	17.5% 7/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Skin and subcutaneous tissue disorders Bruising (in absence of Grade 3 or 4 thrombocytopenia)	10.0% 4/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Edema/Fluid retention	37.5% 15/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Anorexia	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Dehydration	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Diarrhea	15.0% 6/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Dry mouth/salivary gland (xerostomia)	25.0% 10/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Nausea	45.0% 18/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Vomiting	40.0% 16/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Hemorrhage, CNS	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Hemorrhage, GU: Urinary NOS	10.0% 4/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Bladder (urinary)	17.5% 7/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Blood	15.0% 6/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Lung (pneumonia)	10.0% 4/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Mucosa	15.0% 6/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Sinus	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Soft tissue NOS	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Upper airway NOS	17.5% 7/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Urinary tract NOS	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Opportunistic infection	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Albumin, serum-low (hypoalbuminemia)	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Alkaline phosphatase	20.0% 8/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Bicarbonate, serum-low	10.0% 4/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations GGT (gamma-Glutamyl transpeptidase)	17.5% 7/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hyperbilirubinemia	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypercholesteremia	15.0% 6/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hyperglycemia	47.5% 19/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypernatremia	65.0% 26/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypertriglyceridemia	10.0% 4/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypocalcemia	47.5% 19/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypoglycemia	17.5% 7/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypokalemia	92.5% 37/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypomagnesemia	22.5% 9/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hyponatremia	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypophosphatemia	20.0% 8/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Metabolic/Laboratory - Other	10.0% 4/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Proteinuria	30.0% 12/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations SGOT	32.5% 13/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations SGPT	35.0% 14/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Uric acid, serum-high (hyperuricemia)	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Ataxia (incoordination)	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Confusion	35.0% 14/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Dizziness	32.5% 13/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Memory impairment	15.0% 6/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Mood alteration: Agitation	10.0% 4/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Mood alteration: Anxiety	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Mood alteration: Depression	10.0% 4/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Neuropathy: motor	17.5% 7/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Neuropathy: sensory	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Seizure	37.5% 15/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Somnolence/depressed level of consciousness	40.0% 16/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Speech impairment	25.0% 10/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Tremor	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Eye disorders Vision-blurred vision	17.5% 7/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Pain: Abdomen NOS	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Back	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Chest wall	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Pain: Head/headache	45.0% 18/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Joint	37.5% 15/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Muscle	27.5% 11/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Respiratory, thoracic and mediastinal disorders Cough	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	15.0% 6/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Incontinence, urinary	7.5% 3/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Urinary frequency/urgency	25.0% 10/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Flu-like syndrome	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Vascular disorders Thrombosis/thrombus/embolism	12.5% 5/40 • 15 years, 7 months Adverse event data was collected through regular patient assessment and regular laboratory testing

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