Relapsed NeuroblastomaNCT03649438
131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Relapsed/Refractory Neuroblastoma
A study testing 131 I-Metaiodobenzylguanidine, Potassium Iodide and G-csf in people with Relapsed Neuroblastoma and Metastatic Pheochromocytoma, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
This expanded access is the best available therapy/compassionate use designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who are not eligible for therapies of higher priority. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Relapsed Neuroblastoma, Metastatic Pheochromocytoma
- What is being tested?
- 131 I-Metaiodobenzylguanidine, Potassium Iodide and G-csf
- Type of treatment
- Pill or oral medicine, 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis:
- Relapsed/refractory neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical neuroblastoma cells in the bone marrow
- Metastatic pheochromocytoma
- Age >1 year and able to cooperate with radiation safety restrictions during therapy period
- Karnofsky or Lansky performance status of ≥ 50%
- Life expectancy: patients must have a life expectancy of at least 8 weeks
Show all 24Show fewer
- Disease status: Failure to respond to standard therapy (usually combination chemotherapy with or without radiation and surgery) or development of progressive disease at any phase of standard therapy (any new lesion or an increase in size >25% of a pre-existing lesion). Patients may enter this study with or without re-induction therapy for recurrent tumor.
- Disease must be evaluable by MIBG scan. A positive MIBG scan must be present within 8 weeks prior to study entry and subsequent to any intervening therapy. If the patient has only one MIBG positive lesion and that lesion was radiated, a biopsy must be done at least 4 weeks after radiation was completed and must show viable neuroblastoma.
- Stem Cells: If a patient does not have a hematopoietic stem cell product available for re-infusion after MIBG treatment, they may not receive a 131IMIBG dose >12 mCi/kg. Patients must have a hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of > 12 mCi/kg. 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).
- Stem cell source: The patients on this protocol will have autologous PBSCs available.
- Have acceptable organ function as defined below within 7 days of enrollment:
- Bone Marrow: ANC ≥750 X 109 /L, platelets ≥50,000 X 109 /L, and hemoglobin ≥ 8.0 g/dL without transfusion if stem cells are not available. ANC ≥500 X 109 /L, platelets ≥20,000 X 109 /L, and hemoglobin ≥ 8.0 g/dL with transfusions allowed if stem cells are available. Patients with stem cells available are excluded if they require two platelet transfusions per week to maintain the minimum required platelet count. A bone marrow examination is not medically indicated for patients diagnosed with metastatic pheochromocytoma.
- Renal: Creatinine ≤ 2 times upper limit of normal
- Hepatic: Bilirubin ≤2x upper limit of normal; AST/ALT ≤10x upper limit of normal
- Cardiac: Ejection fraction ≥ 45% on echocardiogram or shortening fraction
- >/=27%
- Pulmonary: normal lung function as manifested by no dyspnea and/or oxygen saturation ≥ 94% on room air.
- Prior Therapy: Patients must have recovered from all acute toxicities (defined as CTCAE 5.0 ≤ grade 1) associated with any prior therapy, and:
- Myelosuppressive chemotherapy: At least 2 weeks should have elapsed since any chemotherapy causing myelosuppression
- Biologic (anti-neoplastic agent): At least 7 days should have elapsed since the completion of therapy with a biologic agent.
- Monoclonal antibodies: At least 3 half-lives should have elapsed since therapy with a monoclonal antibody
- Radiation therapy: Three-months should have elapsed in the case of completing radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation). For all other sites of radiation, at least 2 weeks should have elapsed.
- Cytokine therapy (e.g. G-CSF, GM-CSF, IL-6, IL-2): must be discontinued a minimum of 24 hours prior to MIBG therapy.
- Voluntary written informed consent must be obtained before performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care.
Who cannot join
- Patients with disease of any major organ system that would compromise their ability to withstand therapy.
- Because of the teratogenic potential of the study medication, no patients who are pregnant or lactating will be allowed. Patients of childbearing potential must practice an effective method of birth control while participating on this study, to avoid possible damage to the fetus.
- Known allergy to any of the agents or their ingredients used in this study.
- Patients who are on hemodialysis
- Patients with untreated positive blood cultures or progressive infections as assessed by radiographic studies
- Patients who have had prior treatment with 131I-MIBG who do not meet the re-treatment criteria.
- In patients with metastatic pheochromocytoma, screening urinalysis required prior to study enrollment. If random collection urine specimen is positive for proteinuria, patients must have 24-hour urine protein determination. Patients with metastatic pheochromocytoma are excluded if 24-hour urine protein is above the institutional upper limit of normal.
- Patients with known MIBG-avid parenchymal brain metastases are excluded.
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Texas Southwestern Medical Center
Dallas, Texas, 75235
About this study
This expanded access is the best available therapy/compassionate use designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with relapsed/refractory neuroblastoma or 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.
Read the detailed description
Primary Objective is to provide access to therapy with 131I-MIBG for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma.
Secondary Objectives are to (1) assess disease response to 131I-MIBG therapy for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma; (2) gain more information about the toxicities of 131I-MIBG therapy; and (3) assess improvement of symptoms, including pain and fatigue, for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who are receiving 131I-MIBG therapy.
- Being tested
- 131 I-Metaiodobenzylguanidine, Potassium Iodide, G-csf
- Medical subject headings
- Neuroblastoma, Pheochromocytoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
131 I-MetaiodobenzylguanidineDrug
Also called 131I-MIBG
- 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.
Potassium IodideDrug
Also called KI 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.
G-csfDrug
Also called Neulasta
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). This will start 24 hours after stem cell infusion (if stem cells are to be infused).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed NeuroblastomaStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe 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 Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tanya Watt, University of Texas Southwestern Medical Center
- Investigators
- Tanya Watt, MD, Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03649438
- Sponsor's protocol ID
- STU-2019-0665
- Status last verified
- Dec 2024
- First submitted
- 18 Jul 2018
- Last updated
- 11 Dec 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Relapsed/Refractory Neuroblastoma
- ClinicalTrials.gov ID
- NCT03649438
- Acronym / sponsor ID
- STU-2019-0665
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- First posted
- 28 Aug 2018 (actual)
- 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 expanded access is the best available therapy/compassionate use designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with relapsed/refractory neuroblastoma or 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 description
Primary Objective is to provide access to therapy with 131I-MIBG for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma.
Secondary Objectives are to (1) assess disease response to 131I-MIBG therapy for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma; (2) gain more information about the toxicities of 131I-MIBG therapy; and (3) assess improvement of symptoms, including pain and fatigue, for patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who are receiving 131I-MIBG therapy.
Study design
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (24 listed)
- Diagnosis:
- Relapsed/refractory neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical neuroblastoma cells in the bone marrow
- Metastatic pheochromocytoma
- Age >1 year and able to cooperate with radiation safety restrictions during therapy period
- Karnofsky or Lansky performance status of ≥ 50%
Key exclusion criteria (8 listed)
- Patients with disease of any major organ system that would compromise their ability to withstand therapy.
- Because of the teratogenic potential of the study medication, no patients who are pregnant or lactating will be allowed. Patients of childbearing potential must practice an effective method of birth control while participating on this study, to avoid possible damage to the fetus.
- Known allergy to any of the agents or their ingredients used in this study.
- Patients who are on hemodialysis
- Patients with untreated positive blood cultures or progressive infections as assessed by radiographic studies
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-MetaiodobenzylguanidineAlso: 131I-MIBG* 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. | Drug | – |
| Potassium IodideAlso: KI solutionFor 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. | Drug | – |
| G-csfAlso: NeulastaIt 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). This will start 24 hours after stem cell infusion (if stem cells are to be infused). | 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 11 Dec 2024.
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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.
Tanya Watt, MD principal investigator
University of Texas Southwestern Medical Center
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, Potassium Iodide and G-csf in people with Relapsed Neuroblastoma and Metastatic Pheochromocytoma, run by University of Texas Southwestern Medical Center 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 24 inclusion and 8 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 11 Dec 2024. 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 11 Dec 2024.