Antineoplaston Therapy in Treating Patients With Advanced Mesothelioma

• Sponsor: Burzynski Research Institute (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00003508
• Collaborator: (none)
• Enrollment: 8
• Location: 1
• Arm: 1
• Actual Duration (Months): 109.6
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

Current therapies for advanced Mesothelioma provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of advanced Mesothelioma.

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

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

Detailed Description

Advanced Mesothelioma patients receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues up to 12 months in the absence of disease progression or unacceptable toxicity.

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in patients with advanced Mesothelioma, 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 advanced Mesothelioma.

• To determine objective response, tumor size is measured utilizing physical examination and radiologic studies, 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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 8 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 in Patients With Mesothelioma
• Actual Study Start Date: Mar 8, 1996
• Actual Primary Completion Date: Apr 26, 2005
• Actual Study Completion Date: Apr 26, 2005

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Arm: 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 advanced mesothelioma will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.hourly interEach infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.; Other Names: A10 (Atengenal); AS2-1 (Astugenal)

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Objective Response, Stable Disease, Progressive Disease or Not Evaluable [109 months]

   Objective response rate per The International Working Group response criteria (1999): 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 lesions, sustained for at least four weeks. Stable Disease (SD), < 50% change in the sum of the products of of the greatest perpendicular diameters of all measurable lesions, sustained for at least twelve weeks.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 1 Year to 120 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

DISEASE CHARACTERISTICS:

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

• Evidence of disease by CT scan or MRI

PATIENT CHARACTERISTICS:

Age:

• 1 and over

Performance status:

• Karnofsky 60-100%

Life expectancy:

• At least 2 months

Hematopoietic:

• WBC at least 2,000/mm^3

• Platelet count at least 50,000/mm^3

Hepatic:

• Bilirubin no greater than 2.5 mg/dL

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

• Hepatic function adequate

Renal:

• Creatinine no greater than 2.5 mg/dL

• No renal insufficiency

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

Cardiovascular:

• No uncontrolled hypertension

• No history of congestive heart failure

• No cardiovascular conditions that contraindicate high dosages of sodium

Pulmonary:

• No serious lung disease (e.g., chronic obstructive pulmonary disease)

Other:

• Not pregnant or nursing

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

• Not at high medical or psychiatric risk

• No nonmalignant systemic disease

• No active infection

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• At least 4 weeks since prior immunotherapy and recovered

• No concurrent immunomodulatory agents

Chemotherapy:

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

Endocrine therapy:

• Concurrent corticosteroids allowed

Radiotherapy:

• At least 8 weeks since prior radiotherapy (or less if multiple tumors) and recovered

Surgery:

• Recovered from prior surgery

Other:

• Prior cytodifferentiating agents allowed

• No prior antineoplastons

• No other concurrent antineoplastic agents

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

• Study Chair: Stanislaw R. Burzynski, MD, PhD, Burzynski Research Institute

Study Documents (Full-Text)

None provided.

More Information

Publications

• Burzynski SR, Lewy RI, Axler M, Janicki TJ.Treatment of malignant mesothelioma (MM) with sodium phenylbutyrate.Presented at the 15th International Congress on Anti-Cancer Treatment; February 9-12, 2004, Paris, France.
• Preliminary findings on the use of targeted therapy in combination with sodium phenylbutyrate in advanced malignant mesothelioma: A strategy for improved survival. Journal of Cancer Therapy. 2014; 5:1127-1144.

• Responsible Party: Burzynski Research Institute
• ClinicalTrials.gov Identifier: NCT00003508
• Other Study ID Numbers: CDR0000066551; BC-MA-2
• First Posted: Jan 27, 2003
• Last Update Posted: Apr 27, 2021
• Last Verified: Jan 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Burzynski Research Institute: advanced malignant mesothelioma; recurrent malignant mesothelioma
• Additional relevant MeSH terms: Mesothelioma; Mesothelioma, Malignant

Study Results

Participant Flow

Arm/Group Title	Arm: Experimental: 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 advanced mesothelioma will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.hourly interEach infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.
Period Title: Overall Study
STARTED	8
COMPLETED	3
NOT COMPLETED	5

Baseline Characteristics

Arm/Group Title	Arm: Experimental: 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 advanced mesothelioma will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.hourly interEach infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.
Overall Participants	8
Overall Evaluable Patients	3
Age (years) [Median (Standard Deviation) ]
Median (Standard Deviation) [years]	60.0 (8.9527)
Sex: Female, Male (Count of Participants)
Female	5 62.5%
Male	3 37.5%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Objective Response, Stable Disease, Progressive Disease or Not Evaluable
Description	Objective response rate per The International Working Group response criteria (1999): 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 lesions, sustained for at least four weeks. Stable Disease (SD), < 50% change in the sum of the products of of the greatest perpendicular diameters of all measurable lesions, sustained for at least twelve weeks.
Time Frame	109 months

Outcome Measure Data

Analysis Population Description

All patients enrolled in the study.

Arm/Group Title	Arm: Experimental: 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 advanced mesothelioma will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.hourly interEach infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.
Measure Participants	8
Objective Response	0 0%
Stable Disease	2 25%
Progressive Disease	1 12.5%
Not evaluable	5 62.5%

Adverse Events

Time Frame	9 years, 1 month
Adverse Event Reporting Description	Eight patients were recruited between March 1966 and April 2005. All study subjects were seen at the Burzynski Clinic in Houston TX.
Arm/Group Title	Arm: Experimental: 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 advanced mesothelioma will receive Antineoplaston therapy (Atengenal + Astugenal). The daily doses of A10 and AS2-1 are divided into six infusions, which are given at 4-hourly intervals. Each infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.hourly interEach infusion starts with infusion of A10 and is immediately followed by infusion of AS2-1.
All Cause Mortality
	Arm: Experimental: Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Arm: Experimental: Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	2/8 (25%)
Infections and infestations
Infection NOS	1/8 (12.5%)
Nervous system disorders
Seizure	1/8 (12.5%)
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary: Bronchopulmonary NOS	1/8 (12.5%)
Other (Not Including Serious) Adverse Events
	Arm: Experimental: Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	8/8 (100%)
Blood and lymphatic system disorders
Hemoglobin	2/8 (25%)
Lymphopenia	2/8 (25%)
Cardiac disorders
Hypertension	1/8 (12.5%)
Hypotension	1/8 (12.5%)
Endocrine disorders
Cushingoid appearance	1/8 (12.5%)
Gastrointestinal disorders
Constipation	1/8 (12.5%)
Diarrhea	3/8 (37.5%)
Dry mouth/salivary gland (xerostomia)	1/8 (12.5%)
Heartburn/dyspepsia	1/8 (12.5%)
Nausea	3/8 (37.5%)
Taste alteration (dysgeusia)	1/8 (12.5%)
General disorders
Allergic reaction/hypersensitivity (including drug fever)	2/8 (25%)
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)	2/8 (25%)
Fatigue (asthenia, lethargy, malaise)	5/8 (62.5%)
Fever	1/8 (12.5%)
Rigors/chills	1/8 (12.5%)
Edema/Fluid retention	3/8 (37.5%)
Infections and infestations
Central Venous Catheter Infection	1/8 (12.5%)
Infection	1/8 (12.5%)
Infection (documented clinically): Bronchus	1/8 (12.5%)
Infection (documented clinically): Skin (cellulitis)	1/8 (12.5%)
Opportunistic infection	1/8 (12.5%)
Investigations
Albumin, serum-low (hypoalbuminemia)	2/8 (25%)
Alkaline phosphatase	2/8 (25%)
Hyperglycemia	1/8 (12.5%)
Hyperkalemia	1/8 (12.5%)
Hypernatremia	4/8 (50%)
Hypocalcemia	2/8 (25%)
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary: Bronchopulmonary NOS	1/8 (12.5%)
Hemorrhage, pulmonary: Nose	1/8 (12.5%)
Skin and subcutaneous tissue disorders
Flushing	1/8 (12.5%)
Pruritus/itching	1/8 (12.5%)

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.