<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT02988726</url>
  </required_header>
  <id_info>
    <org_study_id>BC-BT-19</org_study_id>
    <nct_id>NCT02988726</nct_id>
  </id_info>
  <brief_title>Antineoplaston Therapy in Treating Patients With Neurofibroma and Schwannoma</brief_title>
  <official_title>Phase II Study of Antineoplastons A19 and AS2-1 in Patients With Neurofibroma and Schwannoma</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Burzynski Research Institute</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Burzynski Research Institute</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      RATIONALE: Current therapies for adults with a recurrent/residual Neurofibroma or Schwannoma&#xD;
      provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy&#xD;
      suggest that it may prove beneficial in the treatment of adults with a recurrent/residual&#xD;
      Neurofibroma or Schwannoma.&#xD;
&#xD;
      PURPOSE: This study is being performed to determine the effects (good and bad) that&#xD;
      Antineoplaston therapy has on adults with a recurrent/residual Neurofibroma or Schwannoma.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      OVERVIEW: This is a single arm, open-label study in which adults with a recurrent/residual&#xD;
      Neurofibroma or Schwannoma receive gradually escalating doses of intravenous Antineoplaston&#xD;
      therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment&#xD;
      continues for at least 12 months in the absence of disease progression or unacceptable&#xD;
      toxicity.&#xD;
&#xD;
      OBJECTIVES:&#xD;
&#xD;
        -  To determine the efficacy of Antineoplaston therapy in adults with a recurrent/residual&#xD;
           Neurofibroma or Schwannoma, as measured by an objective response to therapy (complete&#xD;
           response, partial response or stable disease).&#xD;
&#xD;
        -  To determine the safety and tolerance of Antineoplaston therapy in adults with a&#xD;
           recurrent/residual Neurofibroma or Schwannoma.&#xD;
&#xD;
        -  To determine objective response, tumor size is measured utilizing MRI scans, which are&#xD;
           performed every 8 weeks for the first two years, every 3 months for the third and fourth&#xD;
           years, every 6 months for the 5th and sixth years, and annually thereafter.&#xD;
&#xD;
      PROJECTED ACCRUAL: A total of 20-40 patients will be accrued to this study.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Slow accrual&#xD;
  </why_stopped>
  <start_date>March 1999</start_date>
  <completion_date type="Actual">August 2001</completion_date>
  <primary_completion_date type="Actual">August 2001</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants with Objective Response</measure>
    <time_frame>12 months</time_frame>
    <description>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), &gt;=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.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage of Participants who Survived</measure>
    <time_frame>6 months, 12 months, 24 months, 36 months, 48 months, 60 months</time_frame>
    <description>6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">3</enrollment>
  <condition>CNS Tumor</condition>
  <arm_group>
    <arm_group_label>Antineoplaston therapy</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <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.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Antineoplaston therapy (Atengenal + Astugenal)</intervention_name>
    <description>Adults with a recurrent/residual Neurofibroma or Schwannoma will receive Antineoplaston therapy (Atengenal + Astugenal).</description>
    <arm_group_label>Antineoplaston therapy</arm_group_label>
    <other_name>A10 (Atengenal); AS2-1 (Astugenal)</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Histologically confirmed incurable Neurofibroma or Schwannoma. Histological&#xD;
             confirmation is waived for tumor location where biopsy cannot be safely performed.&#xD;
&#xD;
          -  There will be no exclusion based on multifocality, or leptomeningeal or systemic&#xD;
             metastases.&#xD;
&#xD;
          -  The tumor must be at least 5 mm in size.&#xD;
&#xD;
          -  Radiologic evidence of residual or recurrent tumor by gadolinium-enhanced MRI&#xD;
             performed no more than 14 days before the beginning of therapy.&#xD;
&#xD;
          -  Patients who have previously received radiation therapy, chemotherapy, immunotherapy&#xD;
             or cytodifferentiating agents are eligible.&#xD;
&#xD;
          -  Patients who did not receive standard therapy are eligible.&#xD;
&#xD;
          -  Patients may be male or female. If female, the patient must not be pregnant or&#xD;
             breast-feeding an infant, and either incapable of becoming pregnant or currently using&#xD;
             contraceptive methods. Acceptable methods include the birth control pill, use of a&#xD;
             diaphragm, intrauterine device, or condom by the patient's sexual partner. If male,&#xD;
             the patient should use appropriate contraception, such as condoms, during the study&#xD;
             and at least 4 weeks following completion of the study.&#xD;
&#xD;
          -  Patient must sign the Informed Consent Document indicting an awareness of the&#xD;
             experimental nature of this study. In the event the patient has impairment of higher&#xD;
             intellectual function, for example, aphasia, the the patient's legal next of kin or&#xD;
             legal guardian must sign the Informed Consent Document indicating an awareness of the&#xD;
             experimental nature of the study.&#xD;
&#xD;
          -  Patient must fully recover from the operative procedure and have life expectancy of&#xD;
             two months or more. The patient's performance status should be consistent with&#xD;
             outpatient therapy, i.e., 60% - 100% Karnofsky. The use of corticosteroids is&#xD;
             permitted to reduce symptoms attributed to cerebral edema. It is recommended that the&#xD;
             smallest dose be used compatible with the preservation of optimal neurological&#xD;
             function.&#xD;
&#xD;
          -  Minimal hematological parameters include a hemoglobin of at least 9 g/dL, a white&#xD;
             blood count of at least 1500, and a platelet count of at least 50,000.&#xD;
&#xD;
          -  Patients with no evidence of hepatic or renal insufficiency; a total serum bilirubin&#xD;
             and creatinine not higher than 2.5 mg/dL; and a SGOT and SGPT not higher than 5 times&#xD;
             the upper limit of normal.&#xD;
&#xD;
          -  Patients must recover from the adverse effect of previous therapy. At least eight&#xD;
             weeks must have lapsed since the last dose of radiation therapy and at least four&#xD;
             weeks must have lapsed since the last dose of chemotherapy (six weeks for&#xD;
             nitrosoureas) or immunotherapy.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Failure to meet all inclusion criteria.&#xD;
&#xD;
          -  Patient either pregnant or breast feeding an infant.&#xD;
&#xD;
          -  Patient is a high medical or psychiatric risk, having non-malignant systemic disease,&#xD;
             which would, in the opinion of the investigator, make therapy with an investigational&#xD;
             agent unwise.&#xD;
&#xD;
          -  Patient is incompetent to give informed consent to treatment. However, the patient may&#xD;
             be admitted if a legally appointed guardian gives consent.&#xD;
&#xD;
          -  Presence of an active infection.&#xD;
&#xD;
          -  Patients with known chronic heart failure and serious lung disease, such as severe&#xD;
             COPD.&#xD;
&#xD;
          -  Patients with hypertension are excluded unless the hypertension is adequately&#xD;
             controlled.&#xD;
&#xD;
          -  Patients who have had prior Antineoplaston treatment should be excluded from this&#xD;
             protocol.&#xD;
&#xD;
          -  Patients who do not have adequate hepatic function.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>99 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Stanislaw R. Burzynski, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Burzynski Research Institute</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Burzynski Clinic</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77055-6330</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.burzynskiresearch.com</url>
    <description>Burzynski Research Institute</description>
  </link>
  <link>
    <url>http://www.burzynskiclinic.com</url>
    <description>Burzynski Clinic</description>
  </link>
  <verification_date>July 2017</verification_date>
  <study_first_submitted>November 22, 2016</study_first_submitted>
  <study_first_submitted_qc>December 7, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 9, 2016</study_first_posted>
  <last_update_submitted>July 24, 2017</last_update_submitted>
  <last_update_submitted_qc>July 24, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">July 26, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Neurofibroma</keyword>
  <keyword>Schwannoma</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neurofibroma</mesh_term>
    <mesh_term>Neurofibromatoses</mesh_term>
    <mesh_term>Neurilemmoma</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT02988726
org study id: BC-BT-19
nct id: NCT02988726
lead sponsor: 
has dmc: No
RATIONALE: Current therapies for adults with a recurrent/residual Neurofibroma or Schwannoma
      provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy
      suggest that it may prove beneficial in the treatment of adults with a recurrent/residual
      Neurofibroma or Schwannoma.

      PURPOSE: This study is being performed to determine the effects (good and bad) that
      Antineoplaston therapy has on adults with a recurrent/residual Neurofibroma or Schwannoma.
OVERVIEW: This is a single arm, open-label study in which adults with a recurrent/residual
      Neurofibroma or Schwannoma 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.

      OBJECTIVES:

        -  To determine the efficacy of Antineoplaston therapy in adults with a recurrent/residual
           Neurofibroma or Schwannoma, 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 adults with a
           recurrent/residual Neurofibroma or Schwannoma.

        -  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.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of Participants with Objective Response
time frame: 12 months
description: 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.
measure: Percentage of Participants who Survived
time frame: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months
description: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival
arm group label: Antineoplaston therapy
arm group type: Experimental
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.
intervention type: Drug
intervention name: Antineoplaston therapy (Atengenal + Astugenal)
description: Adults with a recurrent/residual Neurofibroma or Schwannoma will receive Antineoplaston therapy (Atengenal + Astugenal).
arm group label: Antineoplaston therapy
other name: A10 (Atengenal); AS2-1 (Astugenal)
criteria: 
gender: All
minimum age: 18 Years
maximum age: 99 Years
healthy volunteers: No
last name: Stanislaw R. Burzynski, MD, PhD
role: Principal Investigator
affiliation: Burzynski Research Institute
facility: 
country: United States
url: http://www.burzynskiresearch.com
description: Burzynski Research Institute
url: http://www.burzynskiclinic.com
description: Burzynski Clinic
responsible party type: Sponsor
mesh term: Neurofibroma
mesh term: Neurofibromatoses
mesh term: Neurilemmoma
sharing ipd: No