Childhood CNS Primitive Neuroectodermal TumorPhase 2PNETNCT00003460
Antineoplaston Therapy in Treating Children With Primitive Neuroectodermal Tumors
A Phase 2 study testing Antineoplaston therapy (Atengenal + Astugenal) in people with Childhood CNS Primitive Neuroectodermal Tumor, run by Burzynski Research Institute at 1 site in United States.
Trial summary
RATIONALE: Current therapies for children with primitive neuroectodermal tumors that have not responded to standard therapy provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primitive neuroectodermal… Read more about this study
- Who is it for?
- Ages 6 months to 17 years
- Conditions the study is for
- Childhood CNS Primitive Neuroectodermal Tumor
- What is being tested?
- Antineoplaston therapy (Atengenal + Astugenal)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 1996to Feb 2005
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 17 years
- All sexes
- No healthy volunteers
- Child
DISEASE CHARACTERISTICS:
- Histologically confirmed (except if medically contraindicated) incurable primitive neuroectodermal tumor
- Evidence of progressive or recurrent tumor by MRI scan performed within 2 weeks prior to study entry
- Must have received and failed prior standard therapy
- Tumor must be at least 5 mm
PATIENT CHARACTERISTICS:
Age:
- 6 months to 17 years
Performance status:
- Karnofsky 60-100%
Life expectancy:
- At least 2 months
Hematopoietic:
- WBC at least 2000/mm^3
- Platelet count greater than 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
- No hepatic insufficiency
Renal:
- Creatinine no greater than 2.5 mg/dL
- No history of renal conditions that contraindicate high dosages of sodium
Cardiovascular:
- No uncontrolled hypertension
- No severe heart disease
- No history of congestive heart failure
- No 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 participation
- No serious active infections or fever
- No other serious concomitant 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 (6 weeks for nitrosoureas) and recovered
- No concurrent antineoplastic agents
Endocrine therapy:
- Concurrent corticosteroids for cerebral edema allowed (must be on a stable dose for at least 1 week prior to study entry)
Radiotherapy:
- At least 8 weeks since prior radiotherapy and recovered
Surgery:
- Not specified
Other:
- No prior antineoplaston treatment
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Study of Antineoplastons A10 and AS2-1 In Children With Primitive Neuroectodermal Tumors
RATIONALE: Current therapies for children with primitive neuroectodermal tumors that have not responded to standard therapy provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primitive neuroectodermal tumors that have not responded to standard therapy.
PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) with primitive neuroectodermal tumors that has not responded to standard therapy.
Read the detailed description
OBJECTIVES:
- To determine the efficacy of Antineoplaston therapy in children with primitive neuroectodermal tumors that has not responded to standard therapy, 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 children with a brain tumor.
OVERVIEW: This is a single arm, open-label study in which children with primitive neuroectodermal tumors that have not responded to standard therapy 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.
- Being tested
- Antineoplaston therapy (Atengenal + Astugenal)
- Keywords
- Recurrent primitive neuroectodermal tumor
- Medical subject headings
- Neuroectodermal Tumors, Primitive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Antineoplaston therapy (Atengenal + Astugenal)Drug
Also called A10 (Atengenal); AS2-1 (Astugenal)
Children with a primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
| Study group | Role | What they receive |
|---|---|---|
| Antineoplaston therapy | Experimental | 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. Receives: Drug: Antineoplaston therapy (Atengenal + Astugenal) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Objective Response
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.
Measured: 12 months
Secondary outcomes (1)
Percentage of Participants Who Survived
6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival
Measured: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2003 (estimated)
- Study startApr 1996
- Primary completionFeb 2005
- Study completionFeb 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Childhood CNS Primitive Neuroectodermal TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Burzynski Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Stanislaw R Burzynski, MD, PhD, Principal investigator, Burzynski Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003460
- Sponsor's protocol ID
- CDR0000066492
- Other IDs
- BC-BT-12
- Acronym
- PNET
- Status last verified
- Jul 2017
- First submitted
- 1 Nov 1999
- Last updated
- 24 Aug 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Antineoplastons A10 and AS2-1 In Children With Primitive Neuroectodermal Tumors
- ClinicalTrials.gov ID
- NCT00003460
- Acronym / sponsor ID
- PNET / CDR0000066492
- Keywords
- Recurrent primitive neuroectodermal tumor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Burzynski Research InstituteUniversity, hospital or other
- Study start
- Apr 1996
- Primary completion
- Feb 2005 (actual)
- Study completion
- Feb 2005 (actual)
- First posted
- 27 Jan 2003 (estimated)
- Results
- Posted 10 Nov 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Current therapies for children with primitive neuroectodermal tumors that have not responded to standard therapy provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primitive neuroectodermal tumors that have not responded to standard therapy.
PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) with primitive neuroectodermal tumors that has not responded to standard therapy.
Detailed description
OBJECTIVES:
- To determine the efficacy of Antineoplaston therapy in children with primitive neuroectodermal tumors that has not responded to standard therapy, 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 children with a brain tumor.
OVERVIEW: This is a single arm, open-label study in which children with primitive neuroectodermal tumors that have not responded to standard therapy 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Antineoplaston therapy | Experimental | Drug: Antineoplaston therapy (Atengenal + Astugenal)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. |
Who may qualify
- Ages
- 6 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Antineoplaston therapy (Atengenal + Astugenal)Also: A10 (Atengenal); AS2-1 (Astugenal)Children with a primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal). | Drug | Antineoplaston therapy |
Primary outcome
Number of Participants With Objective Response
Time frame: 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.
1 secondary outcome
Percentage of Participants Who Survived
Time frame: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Burzynski Research InstituteLink provided by the sponsor.
- Burzynski ClinicLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stanislaw R Burzynski, MD, PhD principal investigator
Burzynski Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Antineoplaston therapy (Atengenal + Astugenal) in people with Childhood CNS Primitive Neuroectodermal Tumor, run by Burzynski Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Nov 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Objective Response (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2017.