Trial Outcomes & Findings for Antineoplaston Therapy in Treating Children With Visual Pathway Glioma (NCT NCT00003477)

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. Stable Disease (SD): \<50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions and no Progressive Disease, sustained for at least four weeks. Progressive Disease (PD): \>=25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

12 months

Results posted on

2017-08-24

Participant Flow

Twelve patients were recruited between June1996 and May 2004. 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 visual pathway glioma, which is not amenable to standard therapy or has not responded to standard therapy, will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study STARTED	12
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	4

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 visual pathway glioma, which is not amenable to standard therapy or has not responded to standard therapy, will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study Not evalauable	4

Baseline Characteristics

Antineoplaston Therapy in Treating Children With Visual Pathway Glioma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Antineoplaston Therapy n=12 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 visual pathway glioma, which is not amenable to standard therapy or has not responded to standard therapy, will receive Antineoplaston therapy (Atengenal + Astugenal).
Age, Continuous	4.5 Years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=8 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.
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	3 Participants
Number of Participants With Objective Response Progressive Disease	1 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=12 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.
Percentage of Participants Who Survived 6 months overall survival	91.7 Percentage of participants
Percentage of Participants Who Survived 12 months overall survival	83.3 Percentage of participants
Percentage of Participants Who Survived 24 months overall survival	75.0 Percentage of participants
Percentage of Participants Who Survived 36 months overall survival	58.3 Percentage of participants
Percentage of Participants Who Survived 48 months overall survival	50.0 Percentage of participants
Percentage of Participants Who Survived 60 months overall survival	50.0 Percentage of participants

Adverse Events

Antineoplaston Therapy

Serious events: 9 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Antineoplaston Therapy n=12 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): Patients with a visual pathway glioma will receive Antineoplaston therapy (Atengenal + Astugenal)
Nervous system disorders Seizure	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Somnolence/depressed level of consciousness	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Central venous catheter infection	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Fever	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Vomiting	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Hemorrhage, CNS	8.3% 1/12 • 8 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=12 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): Patients with a visual pathway glioma will receive Antineoplaston therapy (Atengenal + Astugenal)
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	33.3% 4/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Immune system disorders Allergic rhinitis (including sneezing, nasal stuffiness)	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Hemoglobin	41.7% 5/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Leukocytes (total WBC)	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Lymphopenia	25.0% 3/12 • 8 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)	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Central Venous Catheter Infection	41.7% 5/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Central Venous Catheter Non-functional	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Central Venous Catheter - Other	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Fatigue (asthenia, lethargy, malaise)	50.0% 6/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Fever	41.7% 5/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Rigors/chills	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Endocrine disorders Cushingoid appearance	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Anorexia	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Ear and labyrinth disorders Auditory/Ear - Other	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Blood and lymphatic system disorders Platelets	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
General disorders Central Venous Catheter Pain	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Skin and subcutaneous tissue disorders Rash/desquamation	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Endocrine disorders Hot flashes/flushes	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Dehydration	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Diarrhea	33.3% 4/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Distension/bloating, abdominal	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Dry mouth/salivary gland (xerostomia)	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Heartburn/dyspepsia	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Nausea	58.3% 7/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Vomiting	66.7% 8/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Hemorrhage, CNS	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Hemorrhage, GU: Urinary NOS	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Abdomen NOS	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Bladder (urinary)	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Lung (pneumonia)	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Middle ear (otitis	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Pharynx	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Sinus	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Soft tissue NOS	8.3% 1/12 • 8 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	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Infection (documented clinically): Urinary tract NOS	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Opportunistic infection	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Infections and infestations Viral hepatitis	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Albumin, serum-low (hypoalbuminemia)	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Alkaline phosphatase	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Bicarbonate, serum-low	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypercholesteremia	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hyperglycemia	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypernatremia	58.3% 7/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypertriglyceridemia	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypocalcemia	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypokalemia	83.3% 10/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypomagnesemia	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hypoglycemia	33.3% 4/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Hyponatremia	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Metabolic/Laboratory - Other	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Proteinuria	33.3% 4/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations SGOT	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations SGPT	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Investigations Uric acid, serum-high (hyperuricemia)	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Muscle weakness: Left-sided	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Ataxia (incoordination)	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Dizziness	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Mood alteration	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Neuropathy: motor	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Seizure	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Somnolence/depressed level of consciousness	66.7% 8/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Tremor	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Eye disorders Nystagmus	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Eye disorders Ocular/Visual - Other	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Gastrointestinal disorders Pain: Abdomen NOS	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Back	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Respiratory, thoracic and mediastinal disorders Pain: Chest/thorax NOS	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Nervous system disorders Pain: Head/headache	50.0% 6/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Joint	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Muscle	16.7% 2/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Musculoskeletal and connective tissue disorders Pain: Neck	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Respiratory, thoracic and mediastinal disorders Pain: Throat/pharynx/larynx	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Respiratory, thoracic and mediastinal disorders Cough	25.0% 3/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Respiratory, thoracic and mediastinal disorders Pulmonary/Upper Respiratory - Other	8.3% 1/12 • 8 years, 2 months Adverse event data was collected through regular patient assessment and regular laboratory testing
Renal and urinary disorders Urinary frequency/urgency	33.3% 4/12 • 8 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