A Compassionate Use/Expanded Access Protocol Using 131I-MIBG Therapy for Patients With Refractory Neuroblastoma and Metastatic Pheochromocytoma
Primary Purpose
Neuroblastoma, Pheochromocytoma
Status
Available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
131-I-meta-iodobenzylguanidine
Sponsored by
About this trial
This is an expanded access trial for Neuroblastoma focused on measuring MIBG, 131I-MIBG, relapsed, neuroblastoma, pheochromocytoma
Eligibility Criteria
Inclusion Criteria:
- Diagnosis: Relapsed/Refractory neuroblastoma, with an original diagnosis made histologically or from elevated urine catecholamines with abnormal tumor cells in bone marrow OR relapsed/refractory pheochromocytoma.
- Disease status: Progressive disease at any time (defined as any new lesion or an increase in size by >25% of pre-existing disease), or a failure to respond to standard therapy. Patients must have evidence of MIBG uptake into tumor at ≥ one site within 6 weeks prior to entry on study and subsequent to any intervening therapy
- Prior therapy: A minimum of two weeks since should have elapsed since any chemotherapy causing myelosuppression. It must be a minimum of three months since receiving radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation. For any other sites of radiation, at least 2 weeks should have elapsed. For patients who received radiation to the only site of MIBG-avid disease within two months of study entry, biopsy confirmation of residual active disease is required, with positive bone marrow being sufficient. At least 7 days should have elapsed since completion of therapy with a biologic agent and at least 3 half-lives should have elapsed since therapy with a monoclonal antibody. No cytokine therapy may be given within 24 hours of receiving 131I-MIBG. Patients may have received prior MIBG therapy, provided they demonstrated a response or stable disease initially, with progressive disease occurring at least 35 days following treatment.
- Organ function: ANC >500/uL, platelets >20,000/uL with transfusion allowed. Bilirubin ≤2x ULN, AST/ALT ≤10x ULN.Serum Creatinine ≤2x ULN OR 24-hour creatinine clearance OR GFR ≥60ml/min/1.73m2. Normal lung function demonstrated by no dyspnea, exercise intolerance or oxygen requirement. Oxygen saturation ≥94% on room air. No clinically significant cardiac dysfunction and ejection fraction ≥45% on echocardiogram.
- Stem cells: Patients must have a minimum of 2.0 x106/kg viable CD34+ peripheral blood stem cells for re-infusion following 131I-MIBG. An additional back-up of 2.0x106/kg CD34+ cells is recommended but not required.
- Life expectancy longer than 8 weeks,Karnofsky or Lansky performance status of ≥ 50%
Exclusion Criteria:
- Pregnant or lactating patients
- Disease of any organ system that would compromise the patient's ability to participate in the study, including hemodialysis. Significant organ impairment should be discussed with the Principal Investigator prior to study entry
- Patients with active grade 3-4 infection, as defined by the NCI CTCAE V4.0.
- Patients with known MBIG-avid brain parenchymal disease (leptomeningeal or skull based metastases are eligible).
- In patients with metastatic pheochromocytoma, a urinalysis must be preformed prior to study enrollment. If proteinuria is present, a 24 hour urine must be collected and total protein determined. If the 24-hour urine protein is above the institutional upper limit of normal, the patient is excluded.
Sites / Locations
- Cohen Children's Medical Center
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT03015844
First Posted
January 5, 2017
Last Updated
April 11, 2023
Sponsor
Northwell Health
Collaborators
Cohen Children's Medical Center
1. Study Identification
Unique Protocol Identification Number
NCT03015844
Brief Title
A Compassionate Use/Expanded Access Protocol Using 131I-MIBG Therapy for Patients With Refractory Neuroblastoma and Metastatic Pheochromocytoma
Official Title
A Compassionate Use/Expanded Access Protocol Using 131I-MIBG Therapy for Patients With Refractory Neuroblastoma and Metastatic Pheochromocytoma
Study Type
Expanded Access
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Available
Study Start Date
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Primary Completion Date
undefined (undefined)
Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Northwell Health
Collaborators
Cohen Children's Medical Center
4. Oversight
5. Study Description
Brief Summary
This is an expanded access protocol/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. Response rate, toxicity, and time to progression and death will be evaluated.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuroblastoma, Pheochromocytoma
Keywords
MIBG, 131I-MIBG, relapsed, neuroblastoma, pheochromocytoma
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
131-I-meta-iodobenzylguanidine
Other Intervention Name(s)
131I-MIBG, Iobenguane
10. Eligibility
Sex
All
Minimum Age & Unit of Time
365 Days
Maximum Age & Unit of Time
29 Years
Eligibility Criteria
Inclusion Criteria:
Diagnosis: Relapsed/Refractory neuroblastoma, with an original diagnosis made histologically or from elevated urine catecholamines with abnormal tumor cells in bone marrow OR relapsed/refractory pheochromocytoma.
Disease status: Progressive disease at any time (defined as any new lesion or an increase in size by >25% of pre-existing disease), or a failure to respond to standard therapy. Patients must have evidence of MIBG uptake into tumor at ≥ one site within 6 weeks prior to entry on study and subsequent to any intervening therapy
Prior therapy: A minimum of two weeks since should have elapsed since any chemotherapy causing myelosuppression. It must be a minimum of three months since receiving radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation. For any other sites of radiation, at least 2 weeks should have elapsed. For patients who received radiation to the only site of MIBG-avid disease within two months of study entry, biopsy confirmation of residual active disease is required, with positive bone marrow being sufficient. At least 7 days should have elapsed since completion of therapy with a biologic agent and at least 3 half-lives should have elapsed since therapy with a monoclonal antibody. No cytokine therapy may be given within 24 hours of receiving 131I-MIBG. Patients may have received prior MIBG therapy, provided they demonstrated a response or stable disease initially, with progressive disease occurring at least 35 days following treatment.
Organ function: ANC >500/uL, platelets >20,000/uL with transfusion allowed. Bilirubin ≤2x ULN, AST/ALT ≤10x ULN.Serum Creatinine ≤2x ULN OR 24-hour creatinine clearance OR GFR ≥60ml/min/1.73m2. Normal lung function demonstrated by no dyspnea, exercise intolerance or oxygen requirement. Oxygen saturation ≥94% on room air. No clinically significant cardiac dysfunction and ejection fraction ≥45% on echocardiogram.
Stem cells: Patients must have a minimum of 2.0 x106/kg viable CD34+ peripheral blood stem cells for re-infusion following 131I-MIBG. An additional back-up of 2.0x106/kg CD34+ cells is recommended but not required.
Life expectancy longer than 8 weeks,Karnofsky or Lansky performance status of ≥ 50%
Exclusion Criteria:
Pregnant or lactating patients
Disease of any organ system that would compromise the patient's ability to participate in the study, including hemodialysis. Significant organ impairment should be discussed with the Principal Investigator prior to study entry
Patients with active grade 3-4 infection, as defined by the NCI CTCAE V4.0.
Patients with known MBIG-avid brain parenchymal disease (leptomeningeal or skull based metastases are eligible).
In patients with metastatic pheochromocytoma, a urinalysis must be preformed prior to study enrollment. If proteinuria is present, a 24 hour urine must be collected and total protein determined. If the 24-hour urine protein is above the institutional upper limit of normal, the patient is excluded.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Julie Krystal, MD, MPH
Phone
718-470-3460
Email
jkrystal12@northwell.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Jonathan Fish, MD
Phone
718-470-3460
Email
Jfish1@northwell.edu
Facility Information:
Facility Name
Cohen Children's Medical Center
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Individual Site Status
Available
12. IPD Sharing Statement
Citations:
PubMed Identifier
19171714
Citation
Matthay KK, Quach A, Huberty J, Franc BL, Hawkins RA, Jackson H, Groshen S, Shusterman S, Yanik G, Veatch J, Brophy P, Villablanca JG, Maris JM. Iodine-131--metaiodobenzylguanidine double infusion with autologous stem-cell rescue for neuroblastoma: a new approaches to neuroblastoma therapy phase I study. J Clin Oncol. 2009 Mar 1;27(7):1020-5. doi: 10.1200/JCO.2007.15.7628. Epub 2009 Jan 26.
Results Reference
background
PubMed Identifier
14528098
Citation
Kang TI, Brophy P, Hickeson M, Heyman S, Evans AE, Charron M, Maris JM. Targeted radiotherapy with submyeloablative doses of 131I-MIBG is effective for disease palliation in highly refractory neuroblastoma. J Pediatr Hematol Oncol. 2003 Oct;25(10):769-73. doi: 10.1097/00043426-200310000-00005.
Results Reference
background
PubMed Identifier
11956276
Citation
Yanik GA, Levine JE, Matthay KK, Sisson JC, Shulkin BL, Shapiro B, Hubers D, Spalding S, Braun T, Ferrara JL, Hutchinson RJ. Pilot study of iodine-131-metaiodobenzylguanidine in combination with myeloablative chemotherapy and autologous stem-cell support for the treatment of neuroblastoma. J Clin Oncol. 2002 Apr 15;20(8):2142-9. doi: 10.1200/JCO.2002.08.124.
Results Reference
background
PubMed Identifier
9440747
Citation
Matthay KK, DeSantes K, Hasegawa B, Huberty J, Hattner RS, Ablin A, Reynolds CP, Seeger RC, Weinberg VK, Price D. Phase I dose escalation of 131I-metaiodobenzylguanidine with autologous bone marrow support in refractory neuroblastoma. J Clin Oncol. 1998 Jan;16(1):229-36. doi: 10.1200/JCO.1998.16.1.229.
Results Reference
background
PubMed Identifier
17369569
Citation
Matthay KK, Yanik G, Messina J, Quach A, Huberty J, Cheng SC, Veatch J, Goldsby R, Brophy P, Kersun LS, Hawkins RA, Maris JM. Phase II study on the effect of disease sites, age, and prior therapy on response to iodine-131-metaiodobenzylguanidine therapy in refractory neuroblastoma. J Clin Oncol. 2007 Mar 20;25(9):1054-60. doi: 10.1200/JCO.2006.09.3484.
Results Reference
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A Compassionate Use/Expanded Access Protocol Using 131I-MIBG Therapy for Patients With Refractory Neuroblastoma and Metastatic Pheochromocytoma
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