Antineoplaston Therapy in Treating Patients With Ependymoma

• Sponsor: Burzynski Research Institute (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00003479
• Collaborator: (none)
• Enrollment: 9
• Location: 1
• Arm: 1
• Duration (Months): 411
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

RATIONALE: Current therapies for patients with ependymoma provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of patients with ependymoma .

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on patients with ependymoma.

Condition or Disease	Intervention/Treatment	Phase
Ependymoma	Drug: Antineoplaston therapy (Atengenal + Astugenal)	Phase 2

Detailed Description

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in patients with ependymoma as measured by an objective response to therapy (complete response, partial response) or stable disease.

• To determine the safety and tolerance of Antineoplaston therapy in patients with ependymoma

OVERVIEW: This is a single arm, open-label study in which patients with ependymoma receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues for at least 12 months in the absence of disease progression or unacceptable toxicity. After 12 months, patients with a complete or partial response or with stable disease may continue treatment.

To determine objective response, tumor size is measured utilizing MRI scans, which are performed every 8 weeks for the first two years, every 3 months for the third and fourth years, every 6 months for the 5th and sixth years, and annually thereafter.

PROJECTED ACCRUAL: A total of 20-40 patients will be accrued to this study

Study Design

• Study Type: Interventional
• Actual Enrollment: 9 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Phase II Study of Antineoplastons A10 and AS2-1 Infusions in Patients With Ependymoma
• Study Start Date: Jul 1, 1966
• Actual Primary Completion Date: Oct 1, 2000
• Actual Study Completion Date: Oct 1, 2000

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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.	Drug: Antineoplaston therapy (Atengenal + Astugenal); Patients with an ependymoma will receive Antineoplaston therapy (Atengenal + Astugenal).; Other Names: A10 (Atengenal); AS2-1 (Astugenal)

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Objective Response [12 months]

   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.

Secondary Outcome Measures

1. Percentage of Participants Who Survived [6 months, 12 months, 24 months, 36 months, 48 months, 60 months]

   6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival

Eligibility Criteria

Criteria

• Ages Eligible for Study: 6 Months to 99 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

DISEASE CHARACTERISTICS:

• Histologically confirmed ependymoma that is unlikely to respond to existing therapy and for which no curative therapy exists

• Evidence of residual tumor (>= 5mm) by MRI scan performed within two weeks prior to study entry

• No brain stem tumors

PATIENT CHARACTERISTICS:

Age:

• 6 months and over

Performance status:

• Karnofsky 60-100%

Life expectancy:

• At least 2 months

Hematopoietic:

• WBC at least 2,000/mm3

• Platelet count greater than 50,000/mm3

Hepatic:

• Bilirubin no greater than 2.5 mg/dL

• SGOT/SGPT no greater than 5 times upper limit of normal

• No hepatic failure

Renal:

• Creatinine no greater than 2.5 mg/dL

• No renal failure

• No history of renal conditions that contraindicate high dosages of sodium

Cardiovascular:

• No severe heart disease

• No uncontrolled hypertension

• No history of congestive heart failure

• No history of other cardiovascular conditions that contraindicate high dosages of sodium

Pulmonary:

• No severe lung disease

Other:

• Not pregnant or nursing

• Fertile patients must use effective contraception during and for 4 weeks after study

• No serious active infections or fever

• No other serious concurrent disease

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• At least 4 weeks since prior immunotherapy and recovered

• No concurrent immunomodulating agents

Chemotherapy:

• At least 4 weeks since prior chemotherapy and recovered (6 weeks for nitrosoureas)

• No concurrent antineoplastic agents

Endocrine therapy:

• Concurrent corticosteroids for cerebral edema allowed (must be on stable dose for at least 1 week prior to study)

Radiotherapy:

• At least 8 weeks since prior radiotherapy and recovered

Surgery:

• Not specified

Other:

• No prior antineoplaston therapy

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Burzynski Clinic	Houston	Texas	United States	77055-6330

Sponsors and Collaborators

• Burzynski Research Institute

Investigators

• Principal Investigator: Stanislaw R. Burzynski, MD, PhD, Burzynski Research Institute

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Burzynski Research Institute
• Burzynski Clinic

Publications

None provided.

• Responsible Party: Burzynski Research Institute
• ClinicalTrials.gov Identifier: NCT00003479
• Other Study ID Numbers: CDR0000066516; BC-BT-24
• First Posted: Jan 27, 2003
• Last Update Posted: Feb 23, 2022
• Last Verified: Feb 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Burzynski Research Institute: Ependymoma; Anaplastic Ependymoma
• Additional relevant MeSH terms: Ependymoma

Study Results

Participant Flow

Recruitment Details	Nine patients were recruited between July 1996 and October 2000. All study subjects were seen at the Burzynski Clinic in Houston TX.

Arm/Group Title	Antineoplaston Therapy
Arm/Group Description	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).
Period Title: Overall Study
STARTED	9
COMPLETED	6
NOT COMPLETED	3

Baseline Characteristics

Arm/Group Title	Antineoplaston Therapy
Arm/Group Description	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 Participants	9
Age (Years) [Median (Full Range) ]
Median (Full Range) [Years]	7.3
Sex: Female, Male (Count of Participants)
Female	5 55.6%
Male	4 44.4%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Objective Response
Description	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.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Antineoplaston Therapy
Arm/Group Description	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).
Measure Participants	6
Partial Response	1 11.1%
Stable Disease	2 22.2%
Progressive Disease	3 33.3%

2. Secondary Outcome

Title	Percentage of Participants Who Survived
Description	6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival
Time Frame	6 months, 12 months, 24 months, 36 months, 48 months, 60 months

Outcome Measure Data

Analysis Population Description

All study subjects receiving any Antineoplaston therapy

Arm/Group Title	Antineoplaston Therapy
Arm/Group Description	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).
Measure Participants	9
6 months overall survival	66.7 741.1%
12 months overall survival	44.4 493.3%
24 months overall survival	33.3 370%
36 months overall survival	22.2 246.7%
48 months overall survival	22.2 246.7%
60 months overall survival	11.1 123.3%

Adverse Events

Time Frame	4 years, 2 months
Adverse Event Reporting Description	Nine patients were recruited between July 1996 and March 2000. All study subjects were seen at the Burzynski Clinic in Houston TX
Arm/Group Title	Antineoplaston Therapy
Arm/Group Description	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).
All Cause Mortality
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	5/9 (55.6%)
Blood and lymphatic system disorders
Hemoglobin	1/9 (11.1%)
Gastrointestinal disorders
Pancreatitis	1/9 (11.1%)
Pain: Stomach	1/9 (11.1%)
Infections and infestations
Central venous catheter infection	1/9 (11.1%)
Infection (documented clinically): Blood	1/9 (11.1%)
Investigations
Hypokalemia	1/9 (11.1%)
Musculoskeletal and connective tissue disorders
Pain: Joint	1/9 (11.1%)
Nervous system disorders
Confusion	1/9 (11.1%)
Seizure	1/9 (11.1%)
Somnolence/depressed level of consciousness	1/9 (11.1%)
Skin and subcutaneous tissue disorders
Rash: erythema multiforme	1/9 (11.1%)
Other (Not Including Serious) Adverse Events
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	9/9 (100%)
Blood and lymphatic system disorders
Hemoglobin	5/9 (55.6%)
Leukocytes (total WBC)	1/9 (11.1%)
Lymphopenia	1/9 (11.1%)
Neutrophils/granulocytes (ANC/AGC)	1/9 (11.1%)
Gastrointestinal disorders
Diarrhea	3/9 (33.3%)
Nausea	5/9 (55.6%)
Vomiting	4/9 (44.4%)
Pancreatitis	1/9 (11.1%)
Pain: Dental/teeth/peridontal	1/9 (11.1%)
Pain: Stomach	1/9 (11.1%)
General disorders
Non-functional central venous catheter	3/9 (33.3%)
Fatigue (asthenia, lethargy, malaise)	4/9 (44.4%)
Fever	2/9 (22.2%)
Insomnia	1/9 (11.1%)
Rigors/chills	1/9 (11.1%)
Weight gain	1/9 (11.1%)
Pruritus/itching	1/9 (11.1%)
Edema/Fluid retention	1/9 (11.1%)
Immune system disorders
Allergic reaction/hypersensitivity (including drug fever)	2/9 (22.2%)
Infections and infestations
Central venous catheter infection	3/9 (33.3%)
Infection (documented clinically): Bladder (urinary)	2/9 (22.2%)
Infection (documented clinically): Blood	1/9 (11.1%)
Infection (documented clinically): Mucosa	3/9 (33.3%)
Infection (documented clinically): Pharynx	1/9 (11.1%)
Infection (documented clinically): Sinus	1/9 (11.1%)
Infection (documented clinically): Soft tissue NOS	1/9 (11.1%)
Infection (documented clinically): Urinary tract NOS	1/9 (11.1%)
Middle ear (otitis media)	1/9 (11.1%)
Skin	1/9 (11.1%)
Upper airway	1/9 (11.1%)
Investigations
Hyperglycemia	2/9 (22.2%)
Hypernatremia	3/9 (33.3%)
Hyperuricemia	1/9 (11.1%)
Hypocalcemia	1/9 (11.1%)
Hypochloremia	1/9 (11.1%)
Hypoglycemia	1/9 (11.1%)
Hypokalemia	7/9 (77.8%)
Metabolic/Laboratory - Other	1/9 (11.1%)
Proteinuria	1/9 (11.1%)
SGOT	1/9 (11.1%)
SGPT	3/9 (33.3%)
Musculoskeletal and connective tissue disorders
Pain: Joint	3/9 (33.3%)
Nervous system disorders
Apnea	1/9 (11.1%)
Ataxia (incoordination)	1/9 (11.1%)
Confusion	3/9 (33.3%)
Dizziness	2/9 (22.2%)
Neuropathy: cranial: CN VIII Hearing and balance	1/9 (11.1%)
Neuropathy: sensory	1/9 (11.1%)
Seizure	3/9 (33.3%)
Somnolence/depressed level of consciousness	5/9 (55.6%)
Speech impairment	2/9 (22.2%)
Syncope (fainting)	1/9 (11.1%)
Pain: Head/headache	4/9 (44.4%)
Renal and urinary disorders
Hemorrhage, GU: Bladder	1/9 (11.1%)
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)	1/9 (11.1%)
Skin and subcutaneous tissue disorders
Rash: erythema multiforme	1/9 (11.1%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.