Trial Outcomes & Findings for Antineoplaston Therapy in Treating Children With Primitive Neuroectodermal Tumors (NCT NCT00003460)
NCT ID: NCT00003460
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
COMPLETED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
12 months
Results posted on
2017-08-24
Participant Flow
Thirteen patients were recruited between April 1996 and January 2005. All study subjects were seen at the Burzynski Clinic in Houston TX
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 primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Antineoplaston Therapy in Treating Children With Primitive Neuroectodermal Tumors
Baseline characteristics by cohort
| Measure |
Antineoplaston Therapy
n=13 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 primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
|
|---|---|
|
Age, Continuous
|
6.1 Years
n=93 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: 12 monthsObjective 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.
Outcome measures
| Measure |
Antineoplaston Therapy
n=12 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 primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
|
|---|---|
|
Number of Participants With Objective Response
Complete Response
|
3 Participants
|
|
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
|
7 Participants
|
SECONDARY outcome
Timeframe: 6 months, 12 months, 24 months, 36 months, 48 months, 60 monthsPopulation: All study subjects receiving any Antineoplaston therapy
6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival
Outcome measures
| Measure |
Antineoplaston Therapy
n=13 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 primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
|
|---|---|
|
Percentage of Participants Who Survived
6 months overall survival
|
61.5 Percentage of participants
|
|
Percentage of Participants Who Survived
12 months overall survival
|
61.5 Percentage of participants
|
|
Percentage of Participants Who Survived
24 months overall survival
|
53.8 Percentage of participants
|
|
Percentage of Participants Who Survived
36 months overall survival
|
46.2 Percentage of participants
|
|
Percentage of Participants Who Survived
48 months overall survival
|
38.5 Percentage of participants
|
|
Percentage of Participants Who Survived
60 months overall survival
|
38.5 Percentage of participants
|
Adverse Events
Antineoplaston Therapy
Serious events: 10 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Antineoplaston Therapy
n=13 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 primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
|
|---|---|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Edema/Fluid retention
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Blood
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Lung (pneumonia)
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection with normal ANC: Lung (pneumonia)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Respiratory, thoracic and mediastinal disorders
Lung (pneumonia)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Ear and labyrinth disorders
Middle ear (otitis media)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection: NOS
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hypernatremia
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hypokalemia
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Seizure
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Somnolence/depressed level of consciousness
|
23.1%
3/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Speech impairment
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Vascular disorders
Thrombosis/thrombus/embolism
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
Other adverse events
| Measure |
Antineoplaston Therapy
n=13 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 primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
|
|---|---|
|
General disorders
Allergic reaction/hypersensitivity (including drug fever)
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Blood and lymphatic system disorders
Hemoglobin
|
38.5%
5/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Blood and lymphatic system disorders
Leukocytes (total WBC)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Blood and lymphatic system disorders
Lymphopenia
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Blood and lymphatic system disorders
Platelets
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Cardiac disorders
Supraventricular and nodal arrhythmia: Sinus tachycardia
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Central venous catheter infection
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Non-functional central venous catheter
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Central venous catheter: Other
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Central venous catheter: Thrombosis/embolism
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
61.5%
8/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Fever
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Rigors/chills
|
30.8%
4/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Weight gain
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Skin and subcutaneous tissue disorders
Pruritus/itching
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
General disorders
Edema/Fluid retention
|
38.5%
5/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Endocrine disorders
Cushingoid appearance
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Gastrointestinal disorders
Anorexia
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Gastrointestinal disorders
Diarrhea
|
38.5%
5/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Gastrointestinal disorders
Dry mouth/salivary gland (xerostomia)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Gastrointestinal disorders
Dysphagia (difficulty swallowing)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Gastrointestinal disorders
Nausea
|
30.8%
4/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Gastrointestinal disorders
Vomiting
|
53.8%
7/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Bladder (urinary)
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Blood
|
23.1%
3/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Bronchus
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Eye NOS
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Lung (pneumonia)
|
23.1%
3/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Middle ear (otitis)
|
30.8%
4/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Mucosa
|
38.5%
5/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Stomach
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection (documented clinically): Upper airway
|
23.1%
3/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection with normal ANC: Lung (pneumonia)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection: NOS
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Infection: Skin
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Skin and subcutaneous tissue disorders
Hypertrychosis
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Alkaline phosphatase
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Infections and infestations
Hyperglycemia
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hypernatremia
|
30.8%
4/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hypocalcemia
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hypoglycemia
|
23.1%
3/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hypokalemia
|
92.3%
12/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hypomagnesemia
|
23.1%
3/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hyponatremia
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Hypophosphatemia
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
SGOT
|
23.1%
3/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
SGPT
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Investigations
Uric acid, serum-high (hyperuricemia)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Ataxia (incoordination)
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Confusion
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Dizziness
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Hydrocephalus
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Seizure
|
38.5%
5/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Somnolence/depressed level of consciousness
|
53.8%
7/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Speech impairment
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Tremor
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Eye disorders
Diplopia
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Eye disorders
Vision-blurred vision
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Musculoskeletal and connective tissue disorders
Pain: Extremity-limb
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Nervous system disorders
Pain: Head/headache
|
46.2%
6/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Musculoskeletal and connective tissue disorders
Pain: Joint
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Musculoskeletal and connective tissue disorders
Pain: Muscle
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
15.4%
2/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Renal and urinary disorders
Incontinence, urinary
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Renal and urinary disorders
Urinary frequency/urgency
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
|
Vascular disorders
Thrombosis/thrombus/embolism
|
7.7%
1/13 • 8 years, 10 months
Adverse event data was collected through regular patient assessment and regular laboratory testing
|
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