NeuroblastomaPhase 2N2011-01NCT02035137
131I-MIBG Alone VS. 131I-MIBG With Vincristine and Irinotecan VS131I-MIBG With Vorinostat
A Phase 2 study testing 131I-MIBG, Vincristine and Irinotecan in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 14 sites in 2 countries.
Trial summary
This study will compare three treatment regimens containing metaiodobenzylguanidine (MIBG) and compare their effects on tumor response and associated side effects, to determine if one therapy is better than the other for people diagnosed with relapsed or persistent neuroblastoma. Read more about this study
- Who is it for?
- Ages 2 to 30 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- 131I-MIBG, Vincristine and Irinotecan
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 114 participants
- Where
- 14 sitesin 2 countries
- Study dates
- Jul 2014to 26 Feb 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be > 24 months and < 30 years of age when registered on study.
- Patients must have relapsed neuroblastoma, refractory neuroblastoma that had less than a partial response to standard treatment or persistent neuroblastoma that had at least a partial response to frontline therapy frontline therapy with > 3 residual lesions on end-induction MIBG scan.
- Patients must have evidence of MIBG uptake into tumor at ≥ one site within 4 weeks prior to entry on study and subsequent to any intervening therapy.
- Patients must have adequate heart, kidney, liver and bone marrow function. Patients who have bone marrow disease must still have adequate bone marrow function to enter the study.
- Patients must have a dose of unpurged peripheral blood stem cells is 2.0 x 106 viable CD34+ cells/kg available.
Who cannot join
- They have had previous I-131 MIBG therapy
- They have other medical problems that could get much worse with this treatment.
- They are pregnant or breast feeding.
- They have a history of a venous or arterial thrombosis that was not associated to a central line.
- They have active infections such as hepatitis or fungal infections.
- They have active diarrhea.
- They have had an allogeneic stem cell transplant (received stem cell from someone else)
- They can't cooperate with the special precautions that are needed for this trial.
Trial locations and contacts
14 sites in 2 countries.
14 sites in 2 countries
United States13 sites
C.S Mott Children's Hospital
Ann Arbor, Michigan, 48109
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus
Atlanta, Georgia, 30322
Children Hospital of Colorado
Aurora, Colorado, 80045
Children's Hospital Boston
Boston, Massachusetts, 02115
University of North Carolina
Chapel Hill, North Carolina, 27599
University of Chicago, Comer Children's Hospital
Chicago, Illinois, 60637
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039
Cook Children's Healthcare System
Fort Worth, Texas, 76104
Childrens Hospital Los Angeles
Los Angeles, California, 90027-0700
Lucile Salter Packer Children's Hospital
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4318
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115
Seattle Children's Hospital
Seattle, Washington, 98105
About this study
Official title: NANT 2011- 01: Randomized Phase II Pick the Winner Study of 131I-MIBG, 131I-MIBG With Vincristine and Irinotecan, or 131I-MIBG With Vorinostat for Resistant/Relapsed Neuroblastoma
This study will compare three treatment regimens containing metaiodobenzylguanidine (MIBG) and compare their effects on tumor response and associated side effects, to determine if one therapy is better than the other for people diagnosed with relapsed or persistent neuroblastoma.
- Conditions
- Neuroblastoma
- Being tested
- 131I-MIBG, Vincristine, Irinotecan, Vorinostat
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
131I-MIBGRadiation
Also called 131I-Metaiodobenzylguanidine, Iobenguane sulfate, m-Iodobenzylguanidine sulfate, MIBG
VincristineDrug
IrinotecanDrug
VorinostatDrug
Also called Zolinza
| Study group | Role | What they receive |
|---|---|---|
| Single-Agent 131I-MIBG | Active comparator | Single-agent 131I-MIBG (Arm A) 18 mCi/kg 131I-MIBG on Day 1 and autologous stem cell infusion on Day 15. Receives: Radiation: 131I-MIBG |
| 131I-MIBG with Vincristine/Irinotecan | Active comparator | Vincristine / irinotecan / 131I-MIBG (Arm B): vincristine 2 mg/m2 (maximum dose 2 mg) intravenously on Day 0; irinotecan 50 mg/m2 (maximum dose 100 mg) intravenously on Days 0 to 4. Patients will also receive diarrhea prophylaxis with cefixime 8 mg/kg/day orally on Days -1 to +6. 131I-MIBG, 18 mCi/kg on Day 1 and autologous stem cell infusion on Day 15. Receives: Radiation: 131I-MIBG; Drug: Vincristine; Drug: Irinotecan |
| 131I-MIBG with Vorinostat | Active comparator | Vorinostat / 131I-MIBG (Arm C); vorinostat 180 mg/m2 (maximum dose 400 mg) orally once daily on Days -1 to +12 (14 total doses). 131I-MIBG, 18 mCi/kg on Day 1 and autologous stem cell infusion on Day 15. Receives: Radiation: 131I-MIBG; Drug: Vorinostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Tumor Response After One Course of Therapy
To identify the MIBG treatment regimen associated with the highest overall response rate after one course of treatment on the three arms. The response evaluation was based on central review (intent to treat analysis). Responders defined as meeting CR/MRD/PR criteria. Response was based on NANT response criteria v1.2 (https://doi.org/10.1002/pbc.26940). RECST 1.1 criteria was used for measurable tumors with PR criteria > 30% decrease in target tumor size. Curie score was used with PR criteria > 50% decrease in Curie score. Complete Response- disappearance of all target lesions, Curie score of 0 and no detectable bone marrow disease. Overall Response (OR)=CR+PR.
Measured: 43-50 days from study day 1
Secondary outcomes (1)
Number of Participants With Grade 3 or Greater Non-hematologic Toxicities
Compare toxicity profiles for grade 3 or greater toxicities associated with each of 131I-MIBG treatment regimens; single-agent 131I-MIBG; Vincristine/Irinotecan/131I-MIBG; or Vorinostat/131I-MIBG
Measured: All toxicities from enrollment through 30 days following end of protocol therapy, an average of 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2014 (estimated)
- Study startJul 2014
- Primary completion26 Feb 2021
- Study completion26 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 114 participants (actual)
Sponsor and investigators
- Lead sponsor
- New Approaches to Neuroblastoma Therapy Consortium
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Steven DuBois, MD, Study chair, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02035137
- Sponsor's protocol ID
- N2011-01
- Acronym
- N2011-01
- Status last verified
- May 2022
- First submitted
- 7 Jan 2014
- Last updated
- 8 Jun 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
DuBois SG, Granger MM, Groshen S, Tsao-Wei D, Ji L, Shamirian A, Czarnecki S, Goodarzian F, Berkovich R, Shimada H, Villablanca JG, Vo KT, Pinto N, Mosse YP, Maris JM, Shusterman S, Cohn SL, Goldsmith KC, Weiss B, Yanik GA, Twist CJ, Irwin MS, Haas-Kogan DA, Park JR, Marachelian A, Matthay KK. Randomized Phase II Trial of MIBG Versus MIBG, Vincristine, and Irinotecan Versus MIBG and Vorinostat for Patients With Relapsed or Refractory Neuroblastoma: A Report From NANT Consortium. J Clin Oncol. 2021 Nov 1;39(31):3506-3514. doi: 10.1200/JCO.21.00703. Epub 2021 Jul 16.
PubMed 34270348
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- NANT 2011- 01: Randomized Phase II Pick the Winner Study of 131I-MIBG, 131I-MIBG With Vincristine and Irinotecan, or 131I-MIBG With Vorinostat for Resistant/Relapsed Neuroblastoma
- ClinicalTrials.gov ID
- NCT02035137
- Acronym / sponsor ID
- N2011-01 / N2011-01
- Conditions
- Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 114 participants (actual)
- Lead sponsor
- New Approaches to Neuroblastoma Therapy ConsortiumUniversity, hospital or other
- Study start
- Jul 2014
- Primary completion
- 26 Feb 2021 (actual)
- Study completion
- 26 Feb 2021 (actual)
- First posted
- 14 Jan 2014 (estimated)
- Results
- Posted 8 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will compare three treatment regimens containing metaiodobenzylguanidine (MIBG) and compare their effects on tumor response and associated side effects, to determine if one therapy is better than the other for people diagnosed with relapsed or persistent neuroblastoma.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Single-Agent 131I-MIBG | Active comparator | Radiation: 131I-MIBGSingle-agent 131I-MIBG (Arm A) 18 mCi/kg 131I-MIBG on Day 1 and autologous stem cell infusion on Day 15. |
| 131I-MIBG with Vincristine/Irinotecan | Active comparator | Radiation: 131I-MIBG, Drug: Vincristine, Drug: IrinotecanVincristine / irinotecan / 131I-MIBG (Arm B): vincristine 2 mg/m2 (maximum dose 2 mg) intravenously on Day 0; irinotecan 50 mg/m2 (maximum dose 100 mg) intravenously on Days 0 to 4. Patients will also receive diarrhea prophylaxis with cefixime 8 mg/kg/day orally on Days -1 to +6. 131I-MIBG, 18 mCi/kg on Day 1 and autologous stem cell infusion on Day 15. |
| 131I-MIBG with Vorinostat | Active comparator | Radiation: 131I-MIBG, Drug: VorinostatVorinostat / 131I-MIBG (Arm C); vorinostat 180 mg/m2 (maximum dose 400 mg) orally once daily on Days -1 to +12 (14 total doses). 131I-MIBG, 18 mCi/kg on Day 1 and autologous stem cell infusion on Day 15. |
Who may qualify
- Ages
- 2 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Patients must be > 24 months and < 30 years of age when registered on study.
- Patients must have relapsed neuroblastoma, refractory neuroblastoma that had less than a partial response to standard treatment or persistent neuroblastoma that had at least a partial response to frontline therapy frontline therapy with > 3 residual lesions on end-induction MIBG scan.
- Patients must have evidence of MIBG uptake into tumor at ≥ one site within 4 weeks prior to entry on study and subsequent to any intervening therapy.
- Patients must have adequate heart, kidney, liver and bone marrow function. Patients who have bone marrow disease must still have adequate bone marrow function to enter the study.
- Patients must have a dose of unpurged peripheral blood stem cells is 2.0 x 106 viable CD34+ cells/kg available.
Key exclusion criteria (8 listed)
- They have had previous I-131 MIBG therapy
- They have other medical problems that could get much worse with this treatment.
- They are pregnant or breast feeding.
- They have a history of a venous or arterial thrombosis that was not associated to a central line.
- They have active infections such as hepatitis or fungal infections.
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 |
|---|---|---|
| 131I-MIBGAlso: 131I-Metaiodobenzylguanidine, Iobenguane sulfate, m-Iodobenzylguanidine sulfate, MIBG | Radiation | 131I-MIBG with Vincristine/Irinotecan, 131I-MIBG with Vorinostat, Single-Agent 131I-MIBG |
| Vincristine | Drug | 131I-MIBG with Vincristine/Irinotecan |
| Irinotecan | Drug | 131I-MIBG with Vincristine/Irinotecan |
| VorinostatAlso: Zolinza | Drug | 131I-MIBG with Vorinostat |
Primary outcome
Objective Tumor Response After One Course of Therapy
Time frame: 43-50 days from study day 1
To identify the MIBG treatment regimen associated with the highest overall response rate after one course of treatment on the three arms. The response evaluation was based on central review (intent to treat analysis). Responders defined as meeting CR/MRD/PR criteria. Response was based on NANT response criteria v1.2 (https://doi.org/10.1002/pbc.26940). RECST 1.1 criteria was used for measurable tumors with PR criteria > 30% decrease in target tumor size. Curie score was used with PR criteria > 50% decrease in Curie score. Complete Response- disappearance of all target lesions, Curie score of 0 and no detectable bone marrow disease. Overall Response (OR)=CR+PR.
1 secondary outcome
Number of Participants With Grade 3 or Greater Non-hematologic Toxicities
Time frame: All toxicities from enrollment through 30 days following end of protocol therapy, an average of 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34270348DuBois SG, Granger MM, Groshen S, Tsao-Wei D, Ji L, Shamirian A, Czarnecki S, Goodarzian F, Berkovich R, Shimada H, Villablanca JG, Vo KT, Pinto N, Mosse YP, Maris JM, Shusterman S, Cohn SL, Goldsmith KC, Weiss B, Yanik GA, Twist CJ, Irwin MS, Haas-Kogan DA, Park JR, Marachelian A, Matthay KK. Randomized Phase II Trial of MIBG Versus MIBG, Vincristine, and Irinotecan Versus MIBG and Vorinostat for Patients With Relapsed or Refractory Neuroblastoma: A Report From NANT Consortium. J Clin Oncol. 2021 Nov 1;39(31):3506-3514. doi: 10.1200/JCO.21.00703. Epub 2021 Jul 16.
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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.
Steven DuBois, MD study chair
Dana-Farber Cancer Institute
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 131I-MIBG, Vincristine and Irinotecan in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 14 sites in 2 countries.
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 5 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: finished, as of the sponsor's last update on 8 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Canada. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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.
What does the primary outcome measure?
Objective Tumor Response After One Course of Therapy (time frame: 43-50 days from study day 1). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02035137, which also lists 1 linked publication.
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 8 Jun 2022; AzTrials last refreshed its copy on 8 Nov 2026.