MIBG for Refractory Neuroblastoma and Pheochromocytoma
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|ClinicalTrials.gov Identifier: NCT01850888|
Recruitment Status : Recruiting
First Posted : May 10, 2013
Last Update Posted : January 15, 2019
|Condition or disease||Intervention/treatment||Phase|
|Relapsed Neuroblastoma Metastatic Pheochromocytoma||Drug: 131 I-Metaiodobenzylguanidine (131I-MIBG) Drug: Potassium iodide solution Drug: G-CSF Procedure: hematopoietic stem cell infusion||Not Applicable|
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:
- 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.
- Dose of 12 mCi/kg for patients without a stem cell source with normal renal function and meets other eligibility criteria.
- 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 without signs of recovery for >2 weeks and any patient requiring platelet transfusion more than two times weekly for 4 consecutive weeks.
- Follow-up will be done until disease progression, death or other therapies are initiated.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||100 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Refractory Neuroblastoma and Pheochromocytoma|
|Study Start Date :||December 2013|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2019|
Experimental: 131 I-MIBG Treatment Arm
Therapeutic 131 I-Metaiodobenzylguanidine (131I-MIBG) will be infused intravenously, intravenous fluids will be administered to help maintain urine flow and isotope excretion. Potassium iodide solution will be administered to protect thyroid function. G-CSF will be used if necessary for neutrophil recovery. Hematopoietic stem cell infusion if meets the criteria.
Drug: 131 I-Metaiodobenzylguanidine (131I-MIBG)
Minimum dose of 10 mCi/kg and up to 18 mCi/kg maximum will be diluted in 25 ml of normal saline, and will be infused intravenously over 90-120 minutes.
Other Name: 131I-MIBG
Drug: Potassium iodide solution
For the therapeutic MIBG administration, potassium iodide solution will be administered in a loading dose of 6mg/kg orally at least 8 hours prior to the MIBG injection, and then will be given at 1mg/kg/dose every 4 hours on days 0-6, then 1 mg/kg/day through day 45 post injection. The minimum dose of potassium iodide to be given is one drop, which equals 50 mg.
Other Name: KI solution
It is recommended that patients with ANC less than 750 after MIBG infusion begin G-CSF 5 mcg/kg/day subcutaneously (or receive equivalent single dose of Neulasta every 14 days while neutropenic) until neutrophil recovery (generally >5000).
Other Name: Neulasta
Procedure: hematopoietic stem cell infusion
The majority of patients on this protocol will have autologous PBSCs available. Allogeneic stem cells may be utilized in patients who has received a prior allogeneic transplant. The minimum quantity for peripheral blood stem cells is 1.5 x 106 CD34+ cells/kg (optimum > 2 x 106 CD34+ cells/kg). The minimum dose for bone marrow is 1.0 x 108 mononuclear cells/kg (optimum >2.0 x 108 mononuclear cells/kg). Infusion will be performed according to institutional guidelines.
Other Name: HSCT
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01850888
|Contact: Emily Greengard, MDfirstname.lastname@example.org|
|United States, Minnesota|
|University of Minnesota Masonic Cancer Center||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Emily G Greengard, MD 612-626-2378 email@example.com|
|Principal Investigator: Emily G Greengard, MD|
|Principal Investigator:||Emily Greengard, MD||University of Minnesota Department of Pediatrics|