NeuroblastomaNCT01590680
Expanded Access Protocol Using 131I-MIBG
A study testing I-131 MIBG in people with Neuroblastoma and Pheochromocytoma, run by Jubilant DraxImage Inc. at 19 sites in United States.
Trial summary
Protocol JDI2007-01 is an Expanded Access Protocol with therapeutic 131I-MIBG for patients with neuroblastoma or pheochromocytoma / paraganglioma, who otherwise do not qualify for available treatments, or where approved treatment is not commercially available. Read more about this study
- Who is it for?
- Ages 12 months and older
- Conditions the study is for
- Neuroblastoma, Pheochromocytoma, Paraganglioma
- What is being tested?
- I-131 MIBG
- Type of treatment
- Injection or infusion, Surgery or procedure
- How many people
- Not given
- Where
- 19 sitesUnited 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 12 months and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Diagnosis: Refractory or relapsed neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical tumor cells in the bone marrow, OR pheochromocytoma or paraganglioma (less than 12 years of age) not amenable to curative surgery.
- Age ≥12 months and able to cooperate with radiation safety restrictions during therapy period with/without pharmacologic anxiolysis.
- Disease status: Failure to respond to standard therapy (usually combination chemotherapy with or without radiation and surgery) or development of progressive disease at any time (any new lesion or an increase in size of >25% of a pre-existing lesion). Disease evaluation must be completed within 8 weeks of study entry. If possible, the disease evaluation should take place subsequent to any intervening therapy; if intervening therapy does occur, evaluations should be done as clinically indicated. If patient has received prior treatment with MIBG, they must have a response or stable disease after the most recent MIBG infusion. Patient may have PD after showing an initial response to MIBG therapy (at [or around] the day 35-63 post-MIBG therapy evaluation).
- Stem cells: Patients must have a hematopoietic stem cell product available for re-infusion after 131I-MIBG treatment at doses of 12 mCi/kg. If no stem cells are available, then the dose of 131I-MIBG should be <12 mCi/kg.
- Prior Therapy: Patients may enter this study with or without re-induction therapy for recurrent tumor. Patients must have fully recovered from the toxic effects of any prior therapy, meeting the following criteria:
- At least 2 weeks should have elapsed since any anti-tumor therapy and the patient must meet certain hematologic criteria.
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- 3 months should have elapsed in the case of completing external beam radiation for total abdominal, whole lung, total body irradiation (spot irradiation to skull-based metastases is NOT a contraindication). Patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis are not contraindicated for treatment on this protocol.
- Cytokine therapy (e.g. G-CSF, GM-CSF, IL-6, erythropoietin) must be discontinued a minimum of 24 hours prior to 131I-MIBG therapy.
- Minimum of six weeks from previous 131I-MIBG therapy.
- The lifetime cumulative injected activity should be evaluated by the Investigator on a case-by-case basis with special attention to any recovery from past 131I-MIBG dose(s).
- For patients who received a stem cell infusion for a previous 131I-MIBG therapy but do NOT have remaining stored stem cells:
- If the stem cell reinfusion was protocol driven but not based upon the development of profound cytopenias (e.g. automatic stem cell reinfusion on Day 14), the patient is eligible for retreatment with MIBG at a dose <12 mCi/kg at the investigators discretion; ii. If the stem cell reinfusion was given based upon the development of profound cytopenias, decisions for re-treatment with 131I-MIBG will require a case-by-case evaluation by the Investigator.
- Organ Function:
- Liver function: Bilirubin ≤ 2x upper limit of normal; AST/ALT ≤ 10x upper limit of normal.
- Kidney function:
- Serum Creatinine ≤ 2x upper limit of normal OR ii. 24-hr creatinine clearance OR GFR ≥ 60 ml/min/1.73m2.
- Hematologic Criteria: ANC ≥750/uL; Platelets ≥ 50,000/uL without transfusion if stem cells are not available (ANC ≥ 500 and any platelet count allowed if stem cells available). Patient must be off myeloid growth factors for at least 24 hours. If the patient has received prior treatment with MIBG, they may be thrombocytopenic, but requiring no more than 2 platelet transfusions per week to maintain counts above 20,000/uL. Hemoglobin must be ≥ 10gm/dL (transfusion allowed) regardless of stored stem cell availability.
- Normal lung function, as manifested by no dyspnea at rest or exercise intolerance, no oxygen requirement.
- No clinically significant cardiac dysfunction.
- Signed informed consent/assent has been obtained.
Who cannot join
- Patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma and marketed product is available.
- Patients eligible for the Phase II (OPTIMUM) trial.
- 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 Principal Investigator prior to patient entry.
- Because of the teratogenic potential of the study medications, no patients who are pregnant or lactating will be allowed. Patients of childbearing potential, who are sexually active, must practice an effective method of birth control while participating on this study, to avoid possible damage to the fetus . [e.g. intrauterine device, double-barrier method (i.e., diaphragm, or a cervical cap) with intravaginal spermicidal foam, cream or gel], or male partner sterilization throughout the study].
- Patients who are on hemodialysis
- Proteinuria, in the absence of urinary infection, within 4 weeks prior to the planned treatment date is a relative contraindication to receiving therapy for patients with pheochromocytoma/paraganglioma. Patients with pheochromocytoma/paraganglioma with any clinically significant proteinuria must have a 24-hr urine protein determination. If proteinuria is confirmed as being above the institutional upper limit of normal, the patient is ineligible for MIBG therapy.
- Patients with active infections that meet grade 3-4 according to the current version of the NCI CTCAE.
- Patients with known MIBG-avid parenchymal brain metastases are not eligible. (Patients with leptomeningeal or skull-based metastases are eligible.)
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
- Available
Michigan Medicine, University of Michigan
Ann Arbor, Michigan, 48105
Anne M Ellis, BS, 734-936-5388, elanne@med.umich.edu
Gregory A Yanik, MD
- Available
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Sarah G Carlin, RN,BSN,APHON, 404-785-0083, sarah.carlin@choa.org
Kelly Goldsmith, MD
- Available
Children's Hospital Colorado
Aurora, Colorado, 80045
Suzanne B Smolik, RN,MSN, 720-777-6823, Suzanne.Smolik@childrenscolorado.org
Margaret E Macy, MD
- Available
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Nora Hussey, 617-632-5419, Nora_Hussey@dfci.harvard.edu
Suzanne Shusterman, MD
- Available
North Carolina Children's Hospital
Chapel Hill, North Carolina, 27599
Juanita Ramirez, BA, 919-966-5785, juanita_ramirez@med.unc.edu
Stuart H Gold, MD
- Available
Carolinas Medical Center/ Levine Children's Hospital
Charlotte, North Carolina, 28203
Jontyce Green, RN, 980-442-2356, Jontyce.Green@atriumhealth.org
Javier E Oesterheld, MD
- Available
University of Chicago Medical Center
Chicago, Illinois, 60637
Melissa Z Marx, MPH, 773-702-2927, lzeilner@peds.bsd.uchicago.edu
Susan L Cohn, MD
- Available
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229-3039
Cancer Referral Line, 513-626-2799, cancer@cchmc.org
Brian D Weiss, MD
- Available
Children's Medical Center Dallas
Dallas, Texas, 75235
Tanya C Watt, MD, 214-456-2382, Tanya.Watt@UTSouthwestern.edu
Tanya C Watt, MD
- Available
Duke University Medical Center
Durham, North Carolina, 27710
Tyler M Ray, BA, 919-681-9186, tyler.ray@duke.edu
Jessica M Sun, MD
- Available
Cook Children's Medical Center
Fort Worth, Texas, 76104
Tracey Easley, RN,BSN,CPHON, 682-885-4017, Tracey.Easley@cookchildrens.org
Meaghan M Granger, MD
- Available
Baylor College of Medicine, Texas Children's Hospital
Houston, Texas, 77035
Akudo A Anyanwu, BSN,RN, 832-824-3480, aaanyanw@texaschildrens.org
Jennifer H Foster, MD
- Available
Children's Hospital Los Angeles
Los Angeles, California, 90027
Araz Marachelian, MD, MS, 323-361-4624, AMarachelian@chla.usc.edu
Araz Marachelian, MD
- Available
University of Wisconsin Hospital and Clinics, American Family Children's Hospital
Madison, Wisconsin, 53792
Jenny L Weiland, 608-890-8070, PedsHemOncResearch@lists.wisc.edu
Kenneth B De Santes, MD
- Available
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
Eugenia DC Owens, BS, 615-343-6169, eugenia.owens@vumc.org
Mykala L Heuer, BSN, 615-875-2122, mykala.l.heuer@vumc.org
- Available
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Maria Gemino-Borromeo, CCRP, 267-425-1987, geminoborm@email.chop.edu
Yael P Mosse, MD
- Available
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
WeiPing DeBlasio, MBA,BSN,RN, 412-692-5485, WeipingXu.DeBlasio@chp.edu
Jean M Tersak, MD
- Available
Seattle Children's Hospital
Seattle, Washington, 98105
Christine Goetz, BA,CCRC, 206-884-1149, christine.goetz@seattlechildrens.org
Navin R Pinto, MD
- Available
Washington University School of Medicine
St Louis, Missouri, 63110
Sally Jones, jones_s@wustl.edu
Fredrick Huang, MD
About this study
Official title: An Open Label, Expanded Access Protocol Using 131I-metaiodobenzylguanidine (131I-MIBG) Therapy in Patients With Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma (Not Eligible for Approved Treatment)
Protocol JDI2007-01 is an Expanded Access Protocol with therapeutic 131I-MIBG for patients with neuroblastoma or pheochromocytoma / paraganglioma, who otherwise do not qualify for available treatments, or where approved treatment is not commercially available.
Read the detailed description
Primary Objectives:
- To provide 131I-MIBG for compassionate use in patients with neuroblastoma, who otherwise do not qualify for inclusion or cannot participate in the sponsor's pivotal Phase II, FDA-approved, clinical trial.
- To provide 131I-MIBG for compassionate use in patients with neuroblastoma in the absence of a commercially available FDA approved product for the indication.
- Provide palliative therapy with 131I-MIBG for patients with advanced neuroblastoma.
- To provide alternative therapeutic 131I-MIBG options for patients with pheochromocytoma / paraganglioma, not qualifying for FDA-approved MIBG treatment.
- To provide alternative therapeutic 131I-MIBG options for patients with pheochromocytoma / paraganglioma, in the absence of a commercially available FDA-approved product for that indication.
- Gain more information about acute and late toxicity of 131I-MIBG therapy for patients with refractory neuroblastoma, pheochromocytoma, or paraganglioma.
Patients will receive a therapeutic dose at the investigator's discretion (5-18 mCi/kg). However, a dose of 12 mCi/kg or higher requires stored stem cells. Patients may be eligible for additional 131I-MIBG treatments (up to a cumulative total of 3 treatments) if they meet certain criteria.
Treatments with 131I-MIBG must be separated by a minimum of six weeks from previous 131I-MIBG therapy. Post-treatment evaluation will be performed 5-9 weeks (35-63 days) post treatment, and patients will be followed every 6 months until 2 years from therapy. All patients will have toxicity monitoring for 2 years following 131I-MIBG therapy, or until going off study.
- Conditions
- Neuroblastoma, Pheochromocytoma, Paraganglioma
- Being tested
- I-131 MIBG
- Medical subject headings
- Neuroblastoma, Pheochromocytoma, Paraganglioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
I-131 MIBGRadiation
Also called I-131 Iobenguane, I-131 meta-iodobenzylguanidine
The therapeutic dose (5-18 mCi/kg at investigator's discretion; any dose ≥12 mCi/kg requires stored stem cells) will be diluted in normal saline, and will be infused intravenously over 90-120 minutes.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- 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
- Jubilant DraxImage Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01590680
- Sponsor's protocol ID
- JDI2007-01
- Status last verified
- Feb 2023
- First submitted
- 1 May 2012
- Last updated
- 28 Feb 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Expanded Access Protocol Using 131I-metaiodobenzylguanidine (131I-MIBG) Therapy in Patients With Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma (Not Eligible for Approved Treatment)
- ClinicalTrials.gov ID
- NCT01590680
- Acronym / sponsor ID
- JDI2007-01
- Conditions
- Neuroblastoma, Pheochromocytoma, Paraganglioma
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Jubilant DraxImage Inc.Industry (pharma, biotech, devices)
- First posted
- 3 May 2012 (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.
Protocol JDI2007-01 is an Expanded Access Protocol with therapeutic 131I-MIBG for patients with neuroblastoma or pheochromocytoma / paraganglioma, who otherwise do not qualify for available treatments, or where approved treatment is not commercially available.
Detailed description
Primary Objectives:
- To provide 131I-MIBG for compassionate use in patients with neuroblastoma, who otherwise do not qualify for inclusion or cannot participate in the sponsor's pivotal Phase II, FDA-approved, clinical trial.
- To provide 131I-MIBG for compassionate use in patients with neuroblastoma in the absence of a commercially available FDA approved product for the indication.
- Provide palliative therapy with 131I-MIBG for patients with advanced neuroblastoma.
- To provide alternative therapeutic 131I-MIBG options for patients with pheochromocytoma / paraganglioma, not qualifying for FDA-approved MIBG treatment.
- To provide alternative therapeutic 131I-MIBG options for patients with pheochromocytoma / paraganglioma, in the absence of a commercially available FDA-approved product for that indication.
- Gain more information about acute and late toxicity of 131I-MIBG therapy for patients with refractory neuroblastoma, pheochromocytoma, or paraganglioma.
Patients will receive a therapeutic dose at the investigator's discretion (5-18 mCi/kg). However, a dose of 12 mCi/kg or higher requires stored stem cells. Patients may be eligible for additional 131I-MIBG treatments (up to a cumulative total of 3 treatments) if they meet certain criteria.
Treatments with 131I-MIBG must be separated by a minimum of six weeks from previous 131I-MIBG therapy. Post-treatment evaluation will be performed 5-9 weeks (35-63 days) post treatment, and patients will be followed every 6 months until 2 years from therapy. All patients will have toxicity monitoring for 2 years following 131I-MIBG therapy, or until going off study.
Study design
Who may qualify
- Ages
- 12 months and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (20 listed)
- Diagnosis: Refractory or relapsed neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical tumor cells in the bone marrow, OR pheochromocytoma or paraganglioma (less than 12 years of age) not amenable to curative surgery.
- Age ≥12 months and able to cooperate with radiation safety restrictions during therapy period with/without pharmacologic anxiolysis.
- Disease status: Failure to respond to standard therapy (usually combination chemotherapy with or without radiation and surgery) or development of progressive disease at any time (any new lesion or an increase in size of >25% of a pre-existing lesion). Disease evaluation must be completed within 8 weeks of study entry. If possible, the disease evaluation should take place subsequent to any intervening therapy; if intervening therapy does occur, evaluations should be done as clinically indicated. If patient has received prior treatment with MIBG, they must have a response or stable disease after the most recent MIBG infusion. Patient may have PD after showing an initial response to MIBG therapy (at [or around] the day 35-63 post-MIBG therapy evaluation).
- Stem cells: Patients must have a hematopoietic stem cell product available for re-infusion after 131I-MIBG treatment at doses of 12 mCi/kg. If no stem cells are available, then the dose of 131I-MIBG should be <12 mCi/kg.
- Prior Therapy: Patients may enter this study with or without re-induction therapy for recurrent tumor. Patients must have fully recovered from the toxic effects of any prior therapy, meeting the following criteria:
Key exclusion criteria (8 listed)
- Patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma and marketed product is available.
- Patients eligible for the Phase II (OPTIMUM) trial.
- 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 Principal Investigator prior to patient entry.
- Because of the teratogenic potential of the study medications, no patients who are pregnant or lactating will be allowed. Patients of childbearing potential, who are sexually active, must practice an effective method of birth control while participating on this study, to avoid possible damage to the fetus . [e.g. intrauterine device, double-barrier method (i.e., diaphragm, or a cervical cap) with intravaginal spermicidal foam, cream or gel], or male partner sterilization throughout the study].
- Patients who are on hemodialysis
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 |
|---|---|---|
| I-131 MIBGAlso: I-131 Iobenguane, I-131 meta-iodobenzylguanidineThe therapeutic dose (5-18 mCi/kg at investigator's discretion; any dose ≥12 mCi/kg requires stored stem cells) will be diluted in normal saline, and will be infused intravenously over 90-120 minutes. | Radiation | – |
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 28 Feb 2023.
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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.
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing I-131 MIBG in people with Neuroblastoma and Pheochromocytoma, run by Jubilant DraxImage Inc. at 19 sites 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 20 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 28 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites 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?
Contact Melda Dolan, MD (+1-215-930-4550, meldadolan@jubl.com), or a site near the patient. 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 28 Feb 2023.