Trial Outcomes & Findings for Antineoplaston Therapy in Treating Patients With Primary Malignant Brain Tumors (NCT NCT00003475)

Last Updated: 2017-08-24

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

COMPLETED

Study phase

PHASE2

Target enrollment

40 participants

Primary outcome timeframe

12 months

Results posted on

2017-08-24

Participant Flow

Fourty patients were recruited between February 1996 and February 2011. All study subjects were seen at the Burzynski Clinic in Houston TX

Participant milestones

Participant milestones
Measure	Antineoplaston Therpay 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 primary malignant brain tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study STARTED	40
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Antineoplaston Therpay 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 primary malignant brain tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study Not evaluable	13

Baseline Characteristics

Antineoplaston Therapy in Treating Patients With Primary Malignant Brain Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Antineoplaston Therpay 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. Adults with a primary malignant brain tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
Age, Continuous	48.2 Years n=99 Participants
Sex: Female, Male Female	14 Participants n=99 Participants
Sex: Female, Male Male	26 Participants n=99 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Antineoplaston Therpay n=27 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 primary malignant brain tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.
Number of Participants With Objective Response Complete Response	2 Participants
Number of Participants With Objective Response Partial Response	2 Participants
Number of Participants With Objective Response Stable Disease	4 Participants
Number of Participants With Objective Response Progressive Disease	19 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 Therpay 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 primary malignant brain tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.
Percentage of Participants Who Survived 6 months overall survival	47.5 Percentage of participants
Percentage of Participants Who Survived 12 months overall survival	27.5 Percentage of participants
Percentage of Participants Who Survived 24 months overall survival	2.5 Percentage of participants
Percentage of Participants Who Survived 36 months overall survival	2.5 Percentage of participants
Percentage of Participants Who Survived 48 months overall survival	2.5 Percentage of participants
Percentage of Participants Who Survived 60 months overall survival	2.5 Percentage of participants

Adverse Events

Antineoplaston Therapy

Serious events: 21 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 primary malignant brain tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.
Blood and lymphatic system disorders Hemoglobin	2.5% 1/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Central venous catheter infection	5.0% 2/40 • 15 years, 2 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, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Perforation, GI: Colon	2.5% 1/40 • 15 years, 2 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, 2 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, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Skin (cellulitis)	2.5% 1/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Mood alteration: Agitation	2.5% 1/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Seizure	12.5% 5/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Somnolence/depressed level of consciousness	12.5% 5/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Back	2.5% 1/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Pain: Head/headache	5.0% 2/40 • 15 years, 2 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, 2 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, 2 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 primary malignant brain tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	17.5% 7/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Hemoglobin	20.0% 8/40 • 15 years, 2 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, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Lymphopenia	25.0% 10/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Platelets	17.5% 7/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Cardiac disorders Hypertension	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Central venous catheter infection	12.5% 5/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Non-functional central venous catheter	17.5% 7/40 • 15 years, 2 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, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders PT	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Fatigue (asthenia, lethargy, malaise)	50.0% 20/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Fever	15.0% 6/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Rigors/chills	7.5% 3/40 • 15 years, 2 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, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Diarrhea	12.5% 5/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Dry mouth/salivary gland (xerostomia)	12.5% 5/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Heartburn/dyspepsia	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Nausea	27.5% 11/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Taste alteration (dysgeusia)	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Vomiting	30.0% 12/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Hemorrhage, GU	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Hemorrhage, GU: Urinary NOS	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection - Other	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Lung (pneumonia)	15.0% 6/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Mucosa	12.5% 5/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Upper airway NOS	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Opportunistic infection	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Albumin, serum-low (hypoalbuminemia)	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypercholesteremia	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hyperglycemia	42.5% 17/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypernatremia	60.0% 24/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypocalcemia	30.0% 12/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypoglycemia	17.5% 7/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypokalemia	75.0% 30/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypophosphatemia	15.0% 6/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Metabolic/Laboratory - Other	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Proteinuria	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations SGOT	15.0% 6/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations SGPT	20.0% 8/40 • 15 years, 2 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, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Ataxia (incoordination)	15.0% 6/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Confusion	32.5% 13/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Dizziness	25.0% 10/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Memory impairment	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Mood alteration: Depression	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Neuropathy: motor	7.5% 3/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Seizure	27.5% 11/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Somnolence/depressed level of consciousness	50.0% 20/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Speech impairment	12.5% 5/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Eye disorders Vision-blurred vision	7.5% 3/40 • 15 years, 2 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, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Extremity-limb	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Pain: Head/headache	37.5% 15/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Joint	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Muscle	10.0% 4/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	20.0% 8/40 • 15 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Urinary frequency/urgency	12.5% 5/40 • 15 years, 2 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, 2 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