Antineoplaston Therapy in Treating Patients With Cancer of Unknown Primary Origin

• Sponsor: Burzynski Research Institute (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00003526
• Collaborator: (none)
• Enrollment: 8
• Location: 1
• Arm: 1
• Actual Duration (Months): 106.5
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

Current therapies for Cancer of Unknown Primary Origin provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of Cancer of Unknown Primary Origin.

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

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

Detailed Description

Cancer of Unknown Primary Origin 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 Cancer of Unknown Primary Origin, 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 Cancer of Unknown Primary Origin.

• 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.

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 Adenocarcinoma of Unknown Primary
• Actual Study Start Date: Mar 18, 1996
• Actual Primary Completion Date: Feb 2, 2005
• Actual Study Completion Date: Feb 2, 2005

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 Cancer of Unknown Primary Origin 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.; Other Names: A10 (Atengenal); AS2-1 (Astugenal)ANP

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Objective Response or Stable Disease [5 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: 6 Months to 99 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

DISEASE CHARACTERISTICS:

• Histologically confirmed adenocarcinoma of unknown primary that is unlikely to respond to existing therapy, including surgery, radiotherapy, and chemotherapy

• Evidence of tumor by MRI, CT scan, chest x-ray, or radionuclide scan

PATIENT CHARACTERISTICS:

Age:

• 6 months 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:

• No hepatic insufficiency

• Bilirubin no greater than 2.5 mg/dL

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

Renal:

• No renal insufficiency

• Creatinine no greater than 2.5 mg/dL

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

Cardiovascular:

• 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, such as chronic obstructive pulmonary disease

Other:

• Not pregnant or nursing

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

• No active infection

• No nonmalignant systemic disease

• Not a high medical or psychiatric risk

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• At least 4 weeks since prior immunotherapy

• No concurrent immunomodulating agents

Chemotherapy:

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

Endocrine therapy:

• Concurrent corticosteroids allowed

Radiotherapy:

• At least 8 weeks since prior radiotherapy

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

• 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: NCT00003526
• Other Study ID Numbers: CDR0000066570; BC-UP-2
• First Posted: Jan 27, 2003
• Last Update Posted: Dec 17, 2020
• Last Verified: Dec 1, 2020

• 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

Study Results

Participant Flow

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 Cancer of Unknown Primary Origin 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.
Period Title: Overall Study
STARTED	8
COMPLETED	2
NOT COMPLETED	6

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 Cancer of Unknown Primary Origin 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.
Overall Participants	8
Age (years) [Median (Full Range) ]
Median (Full Range) [years]	61.6
Sex: Female, Male (Count of Participants)
Female	4 50%
Male	4 50%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Objective Response or Stable Disease
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	5 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 Cancer of Unknown Primary Origin 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.
Measure Participants	2
Stable Disease	1 12.5%
Progressive Disease	1 12.5%

Adverse Events

Time Frame	8 years, 11 months
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 Cancer of Unknown Primary Origin 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.
All Cause Mortality
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	2/8 (25%)
Gastrointestinal disorders
Hemorrhage, GI: Abdomen NOS	1/8 (12.5%)
Infections and infestations
Infection (documented clinically): Lung (pneumonia)	1/8 (12.5%)
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)	1/8 (12.5%)
Other (Not Including Serious) Adverse Events
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	8/8 (100%)
Blood and lymphatic system disorders
Hemoglobin	2/8 (25%)
Lymphopenia	1/8 (12.5%)
Ear and labyrinth disorders
Tinnitus	1/8 (12.5%)
Gastrointestinal disorders
Diarrhea	2/8 (25%)
Dysphagia (difficulty swallowing)	1/8 (12.5%)
Nausea	2/8 (25%)
Vomiting	2/8 (25%)
Hemorrhage, GI	2/8 (25%)
General disorders
Allergic reaction/hypersensitivity (including drug fever)	1/8 (12.5%)
Non-functional Central Venous Catheter	1/8 (12.5%)
Fatigue (asthenia, lethargy, malaise)	4/8 (50%)
Fever	2/8 (25%)
Pain: Head/headache	2/8 (25%)
Infections and infestations
Infection (documented clinically): Bladder (urinary)	1/8 (12.5%)
Lung (pneumonia)	2/8 (25%)
Infection (documented clinically): Upper airway NOS	1/8 (12.5%)
Infection: Other	1/8 (12.5%)
Hyponatremia	1/8 (12.5%)
Investigations
Albumin, serum-low (hypoalbuminemia)	1/8 (12.5%)
Alkaline phosphatase	1/8 (12.5%)
Hyperbilirubinemia	1/8 (12.5%)
Hyperglycemia	3/8 (37.5%)
Hypernatremia	5/8 (62.5%)
Hypocalcemia	1/8 (12.5%)
Hypochloremia	1/8 (12.5%)
Hypoglycemia	1/8 (12.5%)
Hypokalemia	6/8 (75%)
Metabolic/Laboratory - Other	1/8 (12.5%)
SGOT	2/8 (25%)
SGPT	1/8 (12.5%)
Musculoskeletal and connective tissue disorders
Pain: Joint	1/8 (12.5%)
Nervous system disorders
Confusion	1/8 (12.5%)
Dizziness	2/8 (25%)
Mood alteration	1/8 (12.5%)
Neuropathy: cranial: CN VII Motor-face; Sensory-taste	1/8 (12.5%)
Seizure	1/8 (12.5%)
Somnolence/depressed level of consciousness	2/8 (25%)
Speech impairment	1/8 (12.5%)
Respiratory, thoracic and mediastinal disorders
Cough	1/8 (12.5%)
Dyspnea (shortness of breath)	3/8 (37.5%)
Skin and subcutaneous tissue disorders
Edema/Fluid retention	4/8 (50%)
Vascular disorders
Thrombosis/thrombus/embolism	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.