Clinical Trial: MIBG for Refractory Neuroblastoma and Pheochromocytoma

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Refractory Neuroblastoma and Pheochromocytoma

Brief Summary: This is a best available therapy/compassionate use single institution study designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with progressive neuroblastoma and metastatic pheochromocytoma who are not eligible for therapies of higher priority. Patients may receive a range of doses depending on stem cell availability and tumor involvement of bone marrow. Response rate, toxicity, and time to progression and death will be evaluated.

Detailed Summary:

Primary Objective is to provide access to therapy with 131I-MIBG for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma.

Secondary Objective is to assess disease response to 131I-MIBG therapy for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma.

Tertiary Objectives are to 1) gain more information about the toxicities of 131I-MIBG therapy; 2) assess improvement of symptoms, including pain and fatigue, for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who are receiving 131I-MIBG therapy.

  • The therapeutic dose of 131I-MIBG will be based on the following:

    1. Minimum dose of 10 mCi/kg for patients without a stem cell source whose renal function is above the upper limit of normal but still meets eligibility criteria.
    2. Dose of 12 mCi/kg for patients without a stem cell source with normal renal function and meets other eligibility criteria.
    3. Dose of > 12 mCi/kg to 18 mCi/kg maximum at investigator's discretion for patients meeting eligibility criteria with stem cells available.
  • A urinary catheter and intravenous fluids will be used for bladder protection, and potassium iodide solution for thyroid Protection.
  • G-CSF is recommended for patients with ANC less than 750 after MIBG infusion.
  • hematopoietic stem cell infusion is recommended for patients with grade 4 hematologic toxicity following 131I-MIBG therapy that continues to have an ANC <200 on G-CSF wi
    Sponsor: Masonic Cancer Center, University of Minnesota

    Current Primary Outcome: Number of patients who receive 131 I-MIBG. [ Time Frame: 2 hours ]

    The number of patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who receive access to 131 I-MIBG.


    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Disease response [ Time Frame: 1 year ]
      Disease response to 131 I-MIBG therapy in patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma. Disease response will be measured by Curie Score for patients with MIBG avid disease only or by both Currie Score and RECIST criteria for patients who have measureable disease in addition to MIBG avid disease. Disease response will be assessed at day 56 (+/- 14 days) and then every 3 months until 1-year post treatment, then every 6 months until progression, death or other therapy.
    • Incidence of hematologic toxicities [ Time Frame: 1 year ]
      Evaluation of hematologic toxicities of 131I MIBG therapy. CBC with differential and platelet count will be obtained prior to study enrollment, on day 0 and then twice weekly until ANC>500/mm3 and platelet count >20,000 x 3 days without transfusion. Once that is achieved CBC will be then obtained on day 56 and then every 3 months until 1 year post treatment, then every 6 months until progression, death or other therapy. In addition, we will be looking at the percent of patient that require infusion of stem cell product for cytopenias.
    • Incidence of hepatic toxicities [ Time Frame: 1 year ]
      ALT, AST, bilirubin will be obtained prior to study enrollment and then weekly until day 42, again on day 56 and then every 3 months until 1 year post treatment, then every 6 months until progression, death or other therapy
    • Incidence of Thyroid Toxicity [ Time Frame: 1 year ]
      T4 and TSH will be obtained prior to study enrollment and again on day 56 and then every 3 months until 1 year post treatment, then every 6 months until progression, death or other therapy.
    • Improvement of pain symptoms [ Time Frame: 56 days ]
      Assessment of pain will occur on day 0 of therapy, on the day of discharge and then weekly until day 42 and then again on day 56. The PedsQL Pediatric Pain Questionnaire will be used for assessment of pain in all patients. These questionnaires include both patient report and parent report, when appropriate.
    • Improvement of fatigue [ Time Frame: 56 days ]
      Assessment of fatigue will occur on day 0 of therapy, on the day of discharge and then weekly until day 42 and then again on day 56. The PedsQL Multidimensional Fatigue Scale will be used for assessment of fatigue in all patients. These scales include both patient report and parent report, when appropriate.


    Original Secondary Outcome: Same as current

    Information By: Masonic Cancer Center, University of Minnesota

    Dates:
    Date Received: March 26, 2013
    Date Started: December 2013
    Date Completion: December 2018
    Last Updated: August 15, 2016
    Last Verified: August 2016