Antineoplaston Therapy in Treating Patients With Stage IV Melanoma

• Sponsor: Burzynski Research Institute (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00003509
• Collaborator: (none)
• Enrollment: 13
• Location: 1
• Arm: 1
• Actual Duration (Months): 106.6
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

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

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

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

Detailed Description

Stage IV Melanoma 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 Stage IV Melanoma, 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 Stage IV Melanoma.

• 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: 13 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 Malignant Melanoma
• Actual Study Start Date: Mar 27, 1996
• Actual Primary Completion Date: Feb 14, 2005
• Actual Study Completion Date: Feb 14, 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 Stage IV Melanoma 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)

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Objective Response [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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 99 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

DISEASE CHARACTERISTICS:

• Histologically confirmed stage IV melanoma that is recurrent or progressing and unlikely to respond to existing therapy

• Measurable disease by MRI or CT scan

• Tumor must be at least 2 cm in the lymph nodes in the head, neck, axillary, inguinal, or femoral areas and at least 0.5 cm in other locations

PATIENT CHARACTERISTICS:

Age:

• 18 and over

Performance status:

• Karnofsky 60-100%

Life expectancy:

• At least 2 months

Hematopoietic:

• WBC at least 2,000/mm3

• Platelet count at least 50,000/mm3

Hepatic:

• Bilirubin no greater than 2.5 mg/dL

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

• No hepatic insufficiency

Renal:

• Creatinine no greater than 2.5 mg/dL

• No renal insufficiency

• No renal conditions that contraindicate high dosages of sodium

Cardiovascular:

• No chronic heart failure

• No uncontrolled hypertension

• No history of congestive heart failure

• No 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

• No serious medical or psychiatric disorders

• No active infection

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 (6 weeks for nitrosoureas) and recovered

• No concurrent antineoplastic agents

Endocrine therapy:

• Concurrent corticosteroids allowed

Radiotherapy:

• At least 8 weeks since prior radiotherapy and recovered (patients with multiple tumors who have received radiotherapy to some, but not all, tumors may be admitted earlier than 8 weeks)

Surgery:

• Recovered from prior surgery

Other:

• Prior cytodifferentiating agent allowed

• 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: NCT00003509
• Other Study ID Numbers: CDR0000066552; BC-ME-2
• First Posted: Jan 27, 2003
• Last Update Posted: Dec 19, 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
• Keywords provided by Burzynski Research Institute: Stage IV melanoma of the skin
• Additional relevant MeSH terms: Melanoma

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 Stage IV Melanoma 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	13
COMPLETED	8
NOT COMPLETED	5

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 Stage IV Melanoma 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	13
Age (years) [Median (Full Range) ]
Median (Full Range) [years]	44.0
Sex: Female, Male (Count of Participants)
Female	5 38.5%
Male	8 61.5%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Objective Response
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.
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 Stage IV Melanoma 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	8
Stable Disease	2 15.4%
Progressive Disease	6 46.2%

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 Stage IV Melanoma 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	4/13 (30.8%)
Blood and lymphatic system disorders
Hemorrhage-Other	1/13 (7.7%)
Cardiac disorders
Supraventricular and nodal arrhythmia: Sinus tachycardia	1/13 (7.7%)
Hypotension	1/13 (7.7%)
Infections and infestations
Central Venous Catheter Infection	1/13 (7.7%)
Nervous system disorders
Seizure	1/13 (7.7%)
Other (Not Including Serious) Adverse Events
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	13/13 (100%)
Blood and lymphatic system disorders
Hemoglobin	3/13 (23.1%)
Leukocytes (total WBC)	1/13 (7.7%)
Lymphopenia	2/13 (15.4%)
Neutrophils/granulocytes (ANC/AGC)	2/13 (15.4%)
Platelets	1/13 (7.7%)
Hemorrhage-Other	1/13 (7.7%)
Cardiac disorders
Supraventricular and nodal arrhythmia: Sinus tachycardia	1/13 (7.7%)
Hypertension	1/13 (7.7%)
Hypotension	1/13 (7.7%)
Eye disorders
Vision-blurred vision	1/13 (7.7%)
Gastrointestinal disorders
Diarrhea	2/13 (15.4%)
Dry mouth/salivary gland (xerostomia)	2/13 (15.4%)
Nausea	8/13 (61.5%)
Vomiting	6/13 (46.2%)
Liver dysfunction/failure (clinical)	1/13 (7.7%)
Pain: Abdomen NOS	1/13 (7.7%)
General disorders
Allergic reaction/hypersensitivity (including drug fever)	2/13 (15.4%)
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)	1/13 (7.7%)
Central Venous Catheter - Other	1/13 (7.7%)
Fatigue (asthenia, lethargy, malaise)	7/13 (53.8%)
Fever	3/13 (23.1%)
Insomnia	2/13 (15.4%)
Heartburn/dyspepsia	1/13 (7.7%)
Pain: Head/headache	6/13 (46.2%)
Infections and infestations
Central Venous Catheter	1/13 (7.7%)
Infection - Other	1/13 (7.7%)
Infection (documented clinically): Mucosa	1/13 (7.7%)
Infection (documented clinically): Sinus	1/13 (7.7%)
Infection (documented clinically): Upper airway NOS	1/13 (7.7%)
Lung (pneumonia)	1/13 (7.7%)
Opportunistic infection	1/13 (7.7%)
Investigations
Albumin, serum-low (hypoalbuminemia)	1/13 (7.7%)
Alkaline phosphatase	2/13 (15.4%)
Hyperbilirubinemia	1/13 (7.7%)
Hyperglycemia	6/13 (46.2%)
Hypernatremia	6/13 (46.2%)
Hypertriglyceridemia	1/13 (7.7%)
Hypocalcemia	1/13 (7.7%)
Hypoglycemia	2/13 (15.4%)
Hypokalemia	10/13 (76.9%)
Hypophosphatemia	1/13 (7.7%)
SGOT	4/13 (30.8%)
SGPT	4/13 (30.8%)
Musculoskeletal and connective tissue disorders
Pain: Back	1/13 (7.7%)
Pain: Joint	4/13 (30.8%)
Pain: Muscle	3/13 (23.1%)
Nervous system disorders
Hyponatremia	2/13 (15.4%)
Confusion	2/13 (15.4%)
Dizziness	3/13 (23.1%)
Mood alteration: Depression	1/13 (7.7%)
Neuropathy: sensory	1/13 (7.7%)
Seizure	4/13 (30.8%)
Somnolence/depressed level of consciousness	3/13 (23.1%)
Speech impairment	2/13 (15.4%)
Syncope (fainting)	1/13 (7.7%)
Renal and urinary disorders
Hemorrhage, GU	1/13 (7.7%)
Hemorrhage, GU: Bladder	2/13 (15.4%)
Infection (documented clinically): Bladder (urinary)	1/13 (7.7%)
Urinary frequency/urgency	1/13 (7.7%)
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)	3/13 (23.1%)
Skin and subcutaneous tissue disorders
Edema/Fluid retention	2/13 (15.4%)

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.