PheochromocytomaNCT01377532
Compassionate Use of 131I-MIBG for Patients With Malignant Pheochromocytoma
A study testing 131 I-Metaiodobenzylguanidine (131 I-MIBG) in people with Pheochromocytoma and Paraganglioma, run by University of California, San Francisco at 1 site in United States.
Trial summary
This is a compassionate use protocol to allow palliative therapy for patients with malignant pheochromocytoma and paragangliomas. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Pheochromocytoma, Paraganglioma
- What is being tested?
- 131 I-Metaiodobenzylguanidine (131 I-MIBG)
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis: Refractory or relapsed PHEO/PGL with original diagnosis based on tumor histopathology or the finding of PHEO/PGL tumor cells in the bone marrow. The diagnosis may also be based upon the presence of high plasma fractionated metanephrines or high urine catecholamines/metanephrines with diagnostic MIBG uptake.
- Age: > 2 years and able to cooperate with radiation safety restrictions during therapy period.
- Disease status: It must be determined that either the PHEO/PGL tumors are not amenable to safe surgical resection or are metastatic. Disease evaluable by MIBG scan must be present within 6 weeks of study entry and subsequent to any intervening therapy.
- Life Expectancy: greater than 3 months.
- Lanksy and Karnofsky Performance Status: 70% or higher.
- Prior Therapy: Patients may enter this study with or without having had other therapy for recurrent tumor. Patients may be treated who have not had chemotherapy or radiation therapy. Patients may also be treated who have failed to respond to standard chemotherapy or radiation therapy. Patients must have fully recovered from the toxic effects of any prior therapy. At least 2 weeks should have elapsed since any anti-tumor therapy and the patient must meet hematologic criteria below. Three months should have elapsed in the case of completing radiation to any of the following fields: total craniospinal, total abdominal, whole lung, total body irradiation). Cytokine therapy (eg G-CSF, GM-CSF, IL-6, erythropoietin) must be discontinued a minimum of 24 hours prior to MIBG therapy. Prior 131I-MIBG therapy is allowed if > 8 weeks from previous treatment. Cumulative lifetime dose of 131I-MIBG, including the planned treatment, should not exceed 36 mCi/kg.
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- Liver function: bilirubin < 2x upper limit of normal (ULN). Exception: Gilbert syndrome); AST < 10x ULN.
- Kidney function: Creatinine =< 2x ULN.
- Hematopoietic Criteria Patients must have adequate hematopoietic function (without transfusion): ANC >750 x 10E9/L; Platelets >50 x 10E9/L if stem cells are not available; if stem cells are available, the patient should be independent of platelet transfusions with a platelet count of at least 20 x 10E9/L. Hemoglobin >10g/dl at time of treatment (transfusion allowed). Patients with granulocytopenia and/or thrombocytopenia due to tumor metastatic to the bone marrow may be eligible after discussion with Dr. Fitzgerald or designee.
- Normal lung function as manifested by no dyspnea at rest or exercise intolerance, no oxygen requirement.
- No clinically significant cardiac dysfunction, normal EKG and EJ >50%
Who cannot join
- Patients with disease of any major organ system that would compromise their ability to withstand therapy. Any significant organ impairment should be discussed with the Study Chair or Vice Chair prior to patient entry.
- Patients with proteinuria in the absence of urinary infection 4 weeks prior to the planned treatment date.
- Because of the teratogenic potential of the study medications, no patients who are pregnant or breast feeding will be allowed. Patients of childbearing potential must practice an effective method of birth control while participating on this study, to avoid possible pregnancy.
- Patients who are on hemodialysis.
- Patients with active infections that meet grade 3-4 toxicity criteria.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Francisco
San Francisco, California, 94143
About this study
This is a compassionate use protocol to allow palliative therapy for patients with malignant pheochromocytoma and paragangliomas.
Read the detailed description
Pheochromocytomas (PHEO) and paragangliomas (PGL) are rare tumors, with an incidence of 2-8 cases per million annually. These tumors develop in both children and adults. About 15-20% metastasize. Chemotherapy for this tumor usually consists of a combination of cyclophosphamide, vincristine, and dacarbazine delivered over two days and repeated every 3 weeks. Such combined chemotherapy is ineffective for the majority of patients with metastatic PHEO/PGL. A few patients with malignant PHEO have experienced remissions with sunitinib, but the drug may produce severe toxicity and the experience with that drug is limited. Those patients who do experience a remission with chemotherapy must continue it indefinitely to stay in remission. However, most such patients experience such severe side effects from the chemotherapy (marrow suppression, neuropathy, etc) that their chemotherapy must be discontinued. Thus, chemotherapy is either ineffective or intolerable for the vast majority of patients with metastatic PHEO/PGL.
- Conditions
- Pheochromocytoma, Paraganglioma
- Being tested
- 131 I-Metaiodobenzylguanidine (131 I-MIBG)
- Keywords
- Pheochromocytoma, Paraganglioma, MIBG, 131I-MIBG, Resistant, Relapsed, Treatment, UCSF, Pediatric, Adult, Oncology
- Medical subject headings
- Pheochromocytoma, Paraganglioma, Recurrence, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
131 I-Metaiodobenzylguanidine (131 I-MIBG)Drug
Also called 131I-MIBG
Therapeutic 131I-MIBG will be synthesized at Nuclear Diagnostic Products (NDP; Rockaway, New Jersey) with specific activities of 9-18 Ci/mmole. The therapeutic dose: 8-12 mCi/kg (maximum 500 mCi ± 10% at investigator's discretion) will be diluted in 25 ml of normal saline, and will be infused intravenously through a patient's peripheral or central line over 120 minutes. The patient will remain in a radiation protected isolation room until radiation emissions are ≤ 2 mr/hr at a 1 meter distance or meets institutional and state guidelines. This usually takes 4-6 days. In all cases, special shielding will be equipped in the room to minimize exposure to the outside environment and personnel will observe institutional radiation safety precautions. A radiation safety officer will instruct the patient, family, and caretakers about specific UCSF radiation safety guidelines for both hospitalization and discharge.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2011 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PheochromocytomaStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Paul Fitzgerald, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01377532
- Sponsor's protocol ID
- CompUse MIBG Pheo
- Status last verified
- May 2020
- First submitted
- 13 Jun 2011
- Last updated
- 6 May 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use of 131I-MIBG for Patients With Malignant Pheochromocytoma
- ClinicalTrials.gov ID
- NCT01377532
- Acronym / sponsor ID
- CompUse MIBG Pheo
- Conditions
- Pheochromocytoma, Paraganglioma
- Keywords
- Pheochromocytoma, Paraganglioma, MIBG, 131I-MIBG, Resistant, Relapsed, Treatment, UCSF
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- First posted
- 21 Jun 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a compassionate use protocol to allow palliative therapy for patients with malignant pheochromocytoma and paragangliomas.
Detailed description
Pheochromocytomas (PHEO) and paragangliomas (PGL) are rare tumors, with an incidence of 2-8 cases per million annually. These tumors develop in both children and adults. About 15-20% metastasize. Chemotherapy for this tumor usually consists of a combination of cyclophosphamide, vincristine, and dacarbazine delivered over two days and repeated every 3 weeks. Such combined chemotherapy is ineffective for the majority of patients with metastatic PHEO/PGL. A few patients with malignant PHEO have experienced remissions with sunitinib, but the drug may produce severe toxicity and the experience with that drug is limited. Those patients who do experience a remission with chemotherapy must continue it indefinitely to stay in remission. However, most such patients experience such severe side effects from the chemotherapy (marrow suppression, neuropathy, etc) that their chemotherapy must be discontinued. Thus, chemotherapy is either ineffective or intolerable for the vast majority of patients with metastatic PHEO/PGL.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Diagnosis: Refractory or relapsed PHEO/PGL with original diagnosis based on tumor histopathology or the finding of PHEO/PGL tumor cells in the bone marrow. The diagnosis may also be based upon the presence of high plasma fractionated metanephrines or high urine catecholamines/metanephrines with diagnostic MIBG uptake.
- Age: > 2 years and able to cooperate with radiation safety restrictions during therapy period.
- Disease status: It must be determined that either the PHEO/PGL tumors are not amenable to safe surgical resection or are metastatic. Disease evaluable by MIBG scan must be present within 6 weeks of study entry and subsequent to any intervening therapy.
- Life Expectancy: greater than 3 months.
- Lanksy and Karnofsky Performance Status: 70% or higher.
Key exclusion criteria (5 listed)
- Patients with disease of any major organ system that would compromise their ability to withstand therapy. Any significant organ impairment should be discussed with the Study Chair or Vice Chair prior to patient entry.
- Patients with proteinuria in the absence of urinary infection 4 weeks prior to the planned treatment date.
- Because of the teratogenic potential of the study medications, no patients who are pregnant or breast feeding will be allowed. Patients of childbearing potential must practice an effective method of birth control while participating on this study, to avoid possible pregnancy.
- Patients who are on hemodialysis.
- Patients with active infections that meet grade 3-4 toxicity criteria.
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 |
|---|---|---|
| 131 I-Metaiodobenzylguanidine (131 I-MIBG)Also: 131I-MIBGTherapeutic 131I-MIBG will be synthesized at Nuclear Diagnostic Products (NDP; Rockaway, New Jersey) with specific activities of 9-18 Ci/mmole. The therapeutic dose: 8-12 mCi/kg (maximum 500 mCi ± 10% at investigator's discretion) will be diluted in 25 ml of normal saline, and will be infused intravenously through a patient's peripheral or central line over 120 minutes. The patient will remain in a radiation protected isolation room until radiation emissions are ≤ 2 mr/hr at a 1 meter distance or meets institutional and state guidelines. This usually takes 4-6 days. In all cases, special shielding will be equipped in the room to minimize exposure to the outside environment and personnel will observe institutional radiation safety precautions. A radiation safety officer will instruct the patient, family, and caretakers about specific UCSF radiation safety guidelines for both hospitalization and discharge. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2020.
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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.
Paul Fitzgerald, MD principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing 131 I-Metaiodobenzylguanidine (131 I-MIBG) in people with Pheochromocytoma and Paraganglioma, run by University of California, San Francisco 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?
The sponsor lists 11 inclusion and 5 exclusion criteria. 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: status not confirmed, as of the sponsor's last update on 6 May 2020. 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.
Are results available for this study?
Results are not available in the source record.
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.
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 6 May 2020.