Trial Outcomes & Findings for Antineoplaston Therapy in Treating Children With Rhabdoid Tumor of the Central Nervous System (NCT NCT00003469)

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

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

12 months

Results posted on

2017-08-24

Participant Flow

Four patients were recruited between February 1996 and May 2003. 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 Rhabdoid tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Antineoplaston Therapy in Treating Children With Rhabdoid Tumor of the Central Nervous System

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Antineoplaston Therapy n=4 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 Rhabdoid tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Age, Continuous	3.7 Years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=4 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 Rhabdoid 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	2 Participants

SECONDARY outcome

Timeframe: 6 months, 12 months, 24 months

Population: All study subjects receiving any Antineoplaston therapy

6 months, 12 months, 24 months overall survival

Outcome measures

Outcome measures
Measure	Antineoplaston Therapy n=4 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 Rhabdoid tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Percentage of Participants Who Survived 6 months overall survival	75.0 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	0.0 Percentage of participants

Adverse Events

Antineoplaston Therapy

Serious events: 3 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Antineoplaston Therapy n=4 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 Rhabdoid tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
General disorders Central Venous Catheter Infection	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Vomiting	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypernatremia	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypokalemia	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Pain: Head/headache	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX

Other adverse events

Other adverse events
Measure	Antineoplaston Therapy n=4 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 Rhabdoid tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Hemoglobin	75.0% 3/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Leukocytes (total WBC)	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Lymphopenia	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Cardiac disorders Hypotension	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Central venous catheter infection	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Non-functional central venous catheter	50.0% 2/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fatigue (asthenia, lethargy, malaise	50.0% 2/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Fever	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Constipation	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Diarrhea	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Nausea	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Gastrointestinal disorders Vomiting	75.0% 3/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Sinus	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Infections and infestations Infection (documented clinically): Upper airway NOS	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypernatremia	75.0% 3/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hyperuricemia	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypoglycemia	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypokalemia	100.0% 4/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Hypomagnesemia	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Investigations Uric acid, serum-high (hyperuricemia)	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Neuropathy: motor	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Speech impairment	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Eye disorders Vision-blurred vision	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Nervous system disorders Pain: Head/headache	75.0% 3/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. All study subjects were seen at the Burzynski Clinic in Houston TX
General disorders Flu-like syndrome	25.0% 1/4 • 7 years, 2 months Four patients were recruited between February 1996 and October 2010. 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