Trial Outcomes & Findings for Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors (NCT NCT00003476)

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 and \<25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least eight 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

TERMINATED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

12 months

Results posted on

2017-08-24

Participant Flow

Eight patients were recruited between March 1966 and November 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): Children with a primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study STARTED	8
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	1

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 primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study Not evaluable	1

Baseline Characteristics

Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
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. Antineoplaston therapy (Atengenal + Astugenal): Children with a primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Age, Continuous	9.1 Years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=7 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 primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Number of Participants With Objective Response Partial Response	1 Participants
Number of Participants With Objective Response Stable Disease	1 Participants
Number of Participants With Objective Response Progressive Disease	5 Participants

SECONDARY outcome

Timeframe: 6 months, 12 months

Population: All study subjects receiving any Antineoplaston therapy

Six months and Twelve months overall survival

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. Antineoplaston therapy (Atengenal + Astugenal): Children with a primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Percentage of Participants Who Survived 6 months overall survival	50.0 Percentage of participants
Percentage of Participants Who Survived 12 months overall survival	0 Percentage of participants

Adverse Events

Antineoplaston Therapy

Serious events: 2 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Antineoplaston Therapy n=8 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 primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
General disorders Central venous catheter: Thrombosis/embolism	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fever	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Vomiting	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Lung (pneumonia)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Hydrocephalus	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Seizure	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Respiratory, thoracic and mediastinal disorders Aspiration	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX

Other adverse events

Other adverse events
Measure	Antineoplaston Therapy n=8 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 primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Ear and labyrinth disorders Hearing (without monitoring program)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Hemoglobin	25.0% 2/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Leukocytes (total WBC)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Lymphopenia	37.5% 3/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Central venous catheter: Thrombosis/embolism	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fatigue (asthenia, lethargy, malaise)	37.5% 3/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fever	50.0% 4/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Rigors/chills	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Weight gain	25.0% 2/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Skin and subcutaneous tissue disorders Flushing	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Skin and subcutaneous tissue disorders Rash/desquamation	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Endocrine disorders Cushingoid appearance	25.0% 2/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Anorexia	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Dry mouth/salivary gland (xerostomia)	25.0% 2/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Dysphagia (difficulty swallowing)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Nausea	37.5% 3/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Vomiting	62.5% 5/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Conjunctiva	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Lung (pneumonia)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Mucosa	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Sinus	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Upper airway NOS	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Sinus	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Alkaline phosphatase	25.0% 2/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Bicarbonate, serum-low	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations GGT (gamma-Glutamyl transpeptidase)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hyperchloremia	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Hypercholesteremia	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hyperglycemia	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypernatremia	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypocalcemia	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypoglycemia	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypokalemia	100.0% 8/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypomagnesemia	25.0% 2/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Metabolic/Laboratory - Other	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Uric acid, serum-high (hyperuricemia)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Apnea	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Ataxia (incoordination)	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Hydrocephalus	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy - motor	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy: CN III Pupil, upper eyelid, extra ocular movements	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy: cranial: CN V Motor-jaw muscles; Sensory-facial	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy: cranial: CN VII Motor-face; Sensory-taste	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy: cranial: CN VIII Hearing and balance	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy: motor	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Seizure	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Somnolence/depressed level of consciousness	75.0% 6/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Speech impairment	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Eye disorders Diplopia	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Eye disorders Vision-blurred vision	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Pain: Head/headache	37.5% 3/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Respiratory, thoracic and mediastinal disorders Aspiration	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	25.0% 2/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX
Respiratory, thoracic and mediastinal disorders Nasal cavity/paranasal sinus reactions	12.5% 1/8 • 15 years, 10 months Eight patients were recruited between March 1996 and November 2011. 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