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NeuroblastomaPhase 2NCT07261241

NANT 2021-02: Randomized MIBG With Vorinostat/Dinutuximab/Vorinostat + Dinutuximab

A Phase 2 study testing Radiation: 131I-MIBG, Dinutuximab and Vorinostat in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 13 sites in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial summary

Patients will then be randomized at study entry to one of three treatment arms. Patients on Arm A will receive a single treatment course with 131I-MIBG with vorinostat. Patients on Arm B will receive a single treatment course with 131I-MIBG and dinutuximab. Read more about this study

Who is it for?
Ages 1 year to 30 years
Conditions the study is for
Neuroblastoma
What is being tested?
Radiation: 131I-MIBG, Dinutuximab and Vorinostat
Could I receive a placebo?
No, everyone receives an active treatment
How many people
118 participants
Where
13 sitesUnited States
Study dates
31 Dec 2026to 31 Dec 2031
Phase
Phase 2
Are there results?
Not yet: the study is under way

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 1 year to 30 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age Patients must be ≥ 1 year and ≤31 years of age at the time of enrollment on the study.
  • Diagnosis Patients must have a diagnosis of neuroblastoma by histologic verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
  • Disease Risk Group Patients must have high risk neuroblastoma according to COG risk classification at the time of study registration. Patients whose disease was initially considered low or intermediate risk but then reclassified as high risk neuroblastoma prior to enrollment also meet this criteria.
  • Response to Prior Therapy (using INRC definitions)
  • Patients must have at least ONE of the following:
  • Recurrent/progressive disease after the diagnosis of high-risk neuroblastoma at any time prior to enrollment - regardless of response to frontline therapy. (Note that this excludes patients initially considered low or intermediate-risk that progressed to high-risk disease but have not progressed after the diagnosis of high-risk neuroblastoma).
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  • If no prior history of recurrent/progressive disease since the diagnosis of high-risk neuroblastoma,
  • Refractory disease: A best overall response of no response/stable disease since diagnosis of high-risk neuroblastoma AND after at least 4 courses of induction therapy.
  • Persistent disease: A best overall response of partial response since diagnosis of high-risk neuroblastoma AND after at least 4 courses of induction therapy:
  • If a patient with persistent disease has 3 or more MIBG avid sites (including all soft tissue and/or bone lesions) OR a Curie Score of ≥ 3, then no biopsy is required for eligibility.
  • If a patient with persistent disease has only 1 or 2 MIBG avid sites (including all soft tissue and/or bone lesions) then biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma in at least one MIBG avid site (bone marrow, bone, or soft tissue) present at the time of registration is required. Bone and/or soft tissue lesions may be biopsied at any time point prior to study registration; bone marrow must be done at the time of study registration.
  • Sites of Disease: MIBG Uptake Patients must have evidence of MIBG uptake by planar imaging into tumor at ≥ 1 site (bone or soft tissue) within 21 days prior to study entry and subsequent to any intervening therapy. See exclusion criteria.
  • Autologous peripheral blood stem cells (PBSC)
  • The minimum dose for peripheral blood stem cells is 1.5 x 10^6 viable CD34+ cells/kg. Patients who do not meet this minimum requirement for available PBSCs are not eligible.
  • For patients whose body weight exceeds ideal body weight (IBW) by more than 20%, adjusted body weight may be used for the calculation of PBSC dose.
  • Performance Status Patients must have a Lansky (≤ 16 years) or Karnofsky (> 16 years) score of ≥ 50.
  • Note: Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
  • Prior Therapy
  • Patients must have fully recovered from the acute toxic effects and beyond the washout period of all prior chemotherapy, immunotherapy (monoclonal antibodies [MOAB], bi-/tri-specific antibody T-cell engagers, and cellular therapy) or radiotherapy prior to study registration.
  • Organ Function Requirements
  • Hematologic Function:
  • Patients must meet the following hematologic criteria for enrollment regardless of bone marrow disease involvement:
  • ANC ≥750/uL (no short-acting granulocyte growth factors ≤ 7 days of blood draw documenting eligibility and no long-acting granulocyte growth factors ≤ 14 days of blood draw documenting eligibility); and
  • Platelet count ≥ 50,0000/µl, transfusion independent (no platelet transfusions or platelet growth factors ≤ 7 days of blood draw documenting eligibility) Renal Function
  • Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age:
  • Liver Function
  • Total bilirubin ≤ 1.5 x ULN for age; and,
  • SGPT (ALT) ≤ 135 U/L (≤ 3x ULN). Note that for ALT, the upper limit of normal for all sites is defined as 45 U/L.
  • Central Nervous System (CNS) Function:
  • Patients with a history of intraparenchymal or leptomeningeal based CNS disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment.
  • Patients with skull-based tumors with direct intracranial extension are eligible as long as there are no neurologic signs or symptoms related to the lesion.
  • Cardiac Function
  • Normal ejection fraction (≥ 55%) documented by either echocardiogram or radionuclide MUGA evaluation OR normal fractional shortening (≥ 27%) documented by echocardiogram.
  • Corrected QT (QTcF) interval ≤ 480 msec.
  • Pulmonary Function No evidence of dyspnea at rest, no exercise intolerance, or oxygen requirement.
  • Reproductive Function
  • All females of childbearing potential (female patients 10 and older without documented ovarian failure) must have a negative serum or urine beta-HCG ≤ 7 days prior to registration.
  • Male and female subjects of reproductive age and childbearing potential must agree to use two acceptable methods of birth control (i.e., intra-uterine device, hormonal contraception, diaphragm with spermicide, condom with spermicide, or abstinence) or to abstain from heterosexual intercourse for the duration of their participation in the study, or for 3 months after last dose of protocol therapy, whichever is longer.

Who cannot join

  • Pregnancy, breast feeding, or unwillingness to use effective contraception during the study will not be entered on this study due to risks of fetal and teratogenic adverse events.
  • Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring or radiation isolation requirements of the study.
  • Patients with disease of any major organ system that would compromise their ability to withstand therapy.
  • Patients must not have received prior allogeneic stem cell transplant.
  • Patients who have received prior solid organ transplantation.
  • Patients must not have received prior total body irradiation.
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  • Patients who are on hemodialysis.
  • Patients with an active or uncontrolled infection. Patients on prolonged antifungal therapy are still eligible if they are culture negative, afebrile, and meet other organ function criteria.
  • Known history of active human immunodeficiency virus (HIV) infection, hepatitis B, or hepatitis C. Testing is not required in the absence of clinical findings or suspicion.
  • Patients with a history of having to permanently discontinue anti-GD2 antibody therapy, GM-CSF, or vorinostat due to toxicity are not eligible.
  • Patients who have received prior MIBG in combination with anti-GD2 monoclonal antibody and/or histone deacetylase inhibitor
  • The maximum total allowable dose of 131I-MIBG that can be given per institutional guidelines must be at least 90% of the calculated or protocol maximum 131I-MIBG dose or the patient is not eligible.
  • Patients with a history of deep venous thrombosis that was not associated with the presence of a central venous catheter.
  • Only for institutions that have not been granted a waiver by NANT:
  • Patient declines participation in NANT 2004-05, Biology Study
  • Patient declines participation in NANT 2023-03, MIGHTY Study
  • Patients with evidence of active MIBG non-avid disease; patients with previously treated and stable disease that is not MIBG avid are still eligible.
  • Patients whose best response post previous MIBG therapy was progressive disease.
  • Patients with a cumulative lifetime dose of 131I-MIBG greater than 20 mCi/kg.

Trial locations and contacts

13 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Araz Marachelian, MD contact

323-361-5687

nantops@chla.usc.edu

13 sites in 1 country

United States13 sites

  • C.S Mott Children's Hospital

    Ann Arbor, Michigan, 48109

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322

  • Children Hospital of Colorado

    Aurora, Colorado, 80045

  • Childrens Hospital Boston, Dana-Farber Cancer Institute.

    Boston, Massachusetts, 02115

  • Children's Memorial Hospital - Chicago

    Chicago, Illinois, 60614

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039

  • University of Texas Southwestern

    Dalls, Texas, 75235

    Tanya Watt, MD, tanya.watt@utsouthwestern.edu

  • Cook Children's Medical Center - Fort Worth

    Fort Worth, Texas, 76104

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027-0700

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105

    Sara Federico, MD, sara.federico@stjude.org

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104-4318

  • UCSF Helen Diller Family Comprehensive Cancer Center

    San Francisco, California, 94143

  • Children's Hospital and Regional Medical Center - Seattle

    Seattle, Washington, 98105

Other trials in United States

About this study

Official title: NANT 2021-02: A Randomized Phase 2 Study of 131I-MIBG With Vorinostat VS. 131I-MIBG With Dinutuximab vs. 131I-MIBG With Dinutuximab and Vorinostat for Relapsed or Refractory Neuroblastoma

Patients will then be randomized at study entry to one of three treatment arms. Patients on Arm A will receive a single treatment course with 131I-MIBG with vorinostat. Patients on Arm B will receive a single treatment course with 131I-MIBG and dinutuximab. Patients on Arm C will receive a single treatment course with 131I-MIBG with dinutuximab + vorinostat. After this course of treatment, we will check to see your response and then check to see how you are doing over time. All patients may choose to proceed to a second course of the same treatment if they and their physician feel healthy enough to do so. Approximately 118 patients will be receiving therapy on this trial.

Read the detailed description

The proposed study is a 3-arm randomized, pick-the-winner, phase 2 trial designed to identify the optimal combination 131I-MIBG treatment regimen for further study. The three treatment arms are 131I-MIBG + vorinostat; 131I-MIBG + dinutuximab; and 131I-MIBG + dinutuximab + vorinostat. Objective response rate following a single course of therapy will be the primary endpoint driving selection of the regimen to move forward into future studies.

Conditions
Neuroblastoma
Being tested
Radiation: 131I-MIBG, Dinutuximab, Vorinostat
Medical subject headings
Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Radiation: 131I-MIBGDrug

    Also called Iobenguane I 131

    Patients will receive 131I-MIBG 18 mCi/kg (maximum dose 1200 mCi) on Day 1

  • DinutuximabDrug

    Also called Chimeric Monoclonal Antibody 14.18, Chimeric MOAB 14.18, human/murine anti-GD2 monoclonal antibody, chimeric anti-GD2

    Dinutuximab 17.5 mg/m2/day is given intravenously on Days 8-11 and 29-32 of therapy

  • VorinostatDrug

    Also called Zolinza

    Vorinostat will be given on days 0-13 at a dose of 180 mg/m2/dose (maximum dose 400 mg).

Study groups in NCT07261241
Study groupRoleWhat they receive
Arm A: 131I-MIBG + vorinostatExperimentalPatients assigned to Arm A will receive vorinostat orally once daily on Days 0 to 13 at a dose of 180 mg/m2/dose (maximum dose 400 mg). Patients will receive 131I-MIBG 18 mCi/kg (maximum dose 1200 mCi) on Day 1 and autologous stem cell infusion on Day 15 (plus 2 days or minus 1 day, hereafter abbreviated as +2/-1 days). There must be at least 24 hours between the last dose of vorinostat and stem cell infusion. Disease evaluation is to occur between days 43-57. The time interval between performing end of Course 1 disease evaluation and administration of Course 2 131I-MIBG is not to exceed 4 weeks.
Receives: Drug: Radiation: 131I-MIBG; Drug: Vorinostat
Arm B: 131I-MIBG + dinutuximabExperimentalPatients assigned to Arm B will receive 18 mCi/kg (maximum dose 1200 mCi) 131I-MIBG on Day 1 and autologous stem cell infusion on Day 15 (+2/-1 days). Dinutuximab 17.5 mg/m2/day is given intravenously on Days 8-11 and 29-32 of therapy. Disease evaluation is to occur between Days 43-57. In case of treatment delays with dinutuximab during Course 1, the time interval between performing end of Course 1 disease evaluation and administration of Course 2 131I-MIBG is not to exceed 4 weeks.
Receives: Drug: Radiation: 131I-MIBG; Drug: Dinutuximab
Arm C: 131I-MIBG + vorinostat + dinutuximabExperimentalPatients assigned to Arm C will receive vorinostat 180 mg/m2/dose (maximum dose 400 mg) on Days 0 to 13, 131I-MIBG 18 mCi/kg (maximum dose 1200 mCi) on Day 1. Dinutuximab 17.5 mg/m2/day is given intravenously on Days 8-11 and 29-32 of therapy. Disease evaluation is to occur between Days 43-57. In case of treatment delays with dinutuximab during Course 1, the time interval between performing end of Course 1 disease evaluation and administration of Course 2 131I-MIBG is not to exceed 4 weeks.
Receives: Drug: Radiation: 131I-MIBG; Drug: Dinutuximab; Drug: Vorinostat

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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: 43-57 days from study day 1

Secondary outcomes (10)

  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; 131I-MIBG with vorinostat; 131I-MIBG with dinutuximab; or 131I-MIBG with vorinostat and dinutuximab

    Measured: All toxicities from enrollment through 30 days following end of protocol therapy, an average of 6 months

  2. Objective Tumor Response After Two Courses of Therapy

    To identify the MIBG treatment regimen associated with the highest overall response rate after two courses 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: 43-57 days from study day 1

  3. Objective Bone Marrow Tumor Response After One Course of Therapy

    To identify the MIBG treatment regimen associated with the highest bone marrow 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: 43-57 days from study day 1

  4. Objective Bone Marrow Tumor Response After Two Courses of Therapy

    To identify the MIBG treatment regimen associated with the highest bone marrow response rate after two courses 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: 43-57 days from study day 1

  5. Objective Soft Tissue Tumor Response After One Course of Therapy

    To identify the MIBG treatment regimen associated with the highest soft tissue 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: 43-57 days from study day 1

  6. Objective Soft Tissue Tumor Response After Two Courses of Therapy

    To identify the MIBG treatment regimen associated with the highest soft tissue response rate after two courses 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: 43-57 days from study day 1

  7. Objective Bone Tumor Response After One Course of Therapy

    To identify the MIBG treatment regimen associated with the highest bone 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: 43-57 days from study day 1

  8. Objective Bone Tumor Response After Two Courses of Therapy

    To identify the MIBG treatment regimen associated with the highest bone response rate after two courses 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: 43-57 days from study day 1

  9. Objective MIBG Tumor Response After One Course of Therapy

    To identify the MIBG treatment regimen associated with the highest MIBG 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: 43-57 days from study day 1

  10. Objective MIBG Tumor Response After Two Courses of Therapy

    To identify the MIBG treatment regimen associated with the highest MIBG response rate after two courses 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: 43-57 days from study day 1

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Dec 2025
  2. Study start31 Dec 2026 (estimated)
  3. Primary completion31 Dec 2030 (estimated)
  4. Study completion31 Dec 2031 (estimated)

Resources

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
118 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07261241
Sponsor's protocol ID
NANT 2021-02
Status last verified
Aug 2026
First submitted
21 Nov 2025
Last updated
21 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Radiation: 131I-MIBG, Dinutuximab and Vorinostat in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 13 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 38 inclusion and 19 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: opening soon, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 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.

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?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Araz Marachelian, MD (323-361-5687, nantops@chla.usc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Objective Tumor Response After One Course of Therapy (time frame: 43-57 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 NCT07261241.

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 21 Aug 2026.

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