Trial Outcomes & Findings for Antineoplaston Therapy in Treating Patients With Ependymoma (NCT NCT00003479)

Last Updated: 2022-02-23

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

9 participants

Primary outcome timeframe

12 months

Results posted on

2022-02-23

Participant Flow

Nine patients were recruited between July 1996 and October 2000. 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): Patients with an ependymoma will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study STARTED	9
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	3

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): Patients with an ependymoma will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study Not evaluable	3

Baseline Characteristics

Antineoplaston Therapy in Treating Patients With Ependymoma

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): Patients with an ependymoma will receive Antineoplaston therapy (Atengenal + Astugenal).
Age, Continuous	7.3 Years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=6 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): Patients with an ependymoma will receive Antineoplaston therapy (Atengenal + Astugenal).
Number of Participants With Objective Response Partial Response	1 Participants
Number of Participants With Objective Response Stable Disease	2 Participants
Number of Participants With Objective Response Progressive Disease	3 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=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): Patients with an ependymoma will receive Antineoplaston therapy (Atengenal + Astugenal).
Percentage of Participants Who Survived 6 months overall survival	66.7 Percentage of Participants
Percentage of Participants Who Survived 12 months overall survival	44.4 Percentage of Participants
Percentage of Participants Who Survived 24 months overall survival	33.3 Percentage of Participants
Percentage of Participants Who Survived 36 months overall survival	22.2 Percentage of Participants
Percentage of Participants Who Survived 48 months overall survival	22.2 Percentage of Participants
Percentage of Participants Who Survived 60 months overall survival	11.1 Percentage of Participants

Adverse Events

Antineoplaston Therapy

Serious events: 5 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): Patients with an ependymoma will receive Antineoplaston therapy (Atengenal + Astugenal).
Blood and lymphatic system disorders Hemoglobin	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Central venous catheter infection	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Skin and subcutaneous tissue disorders Rash: erythema multiforme	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Pancreatitis	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Blood	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypokalemia	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Confusion	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Seizure	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Somnolence/depressed level of consciousness	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Musculoskeletal and connective tissue disorders Pain: Joint	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Pain: Stomach	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. 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): Patients with an ependymoma will receive Antineoplaston therapy (Atengenal + Astugenal).
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	22.2% 2/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Hemoglobin	55.6% 5/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Leukocytes (total WBC)	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Lymphopenia	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Central venous catheter infection	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Non-functional central venous catheter	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fatigue (asthenia, lethargy, malaise)	44.4% 4/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fever	22.2% 2/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Insomnia	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Rigors/chills	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Weight gain	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Pruritus/itching	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Skin and subcutaneous tissue disorders Rash: erythema multiforme	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Edema/Fluid retention	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Diarrhea	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Nausea	55.6% 5/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Vomiting	44.4% 4/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Renal and urinary disorders Hemorrhage, GU: Bladder	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Pancreatitis	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Bladder (urinary)	22.2% 2/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Blood	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Mucosa	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Pharynx	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Sinus	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Soft tissue NOS	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Urinary tract NOS	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Middle ear (otitis media)	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Skin	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Upper airway	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hyperglycemia	22.2% 2/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypernatremia	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hyperuricemia	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypocalcemia	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypochloremia	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypoglycemia	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypokalemia	77.8% 7/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Metabolic/Laboratory - Other	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Proteinuria	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations SGOT	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations SGPT	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Apnea	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Ataxia (incoordination)	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Confusion	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Dizziness	22.2% 2/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy: cranial: CN VIII Hearing and balance	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy: sensory	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Seizure	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Somnolence/depressed level of consciousness	55.6% 5/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Speech impairment	22.2% 2/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Syncope (fainting)	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Pain: Dental/teeth/peridontal	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Pain: Head/headache	44.4% 4/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Musculoskeletal and connective tissue disorders Pain: Joint	33.3% 3/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Pain: Stomach	11.1% 1/9 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. 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 • 4 years, 2 months Nine patients were recruited between July 1996 and March 2000. 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