NCT01041638

Brief Summary

This phase III trial is studying the side effects of giving monoclonal antibody Ch14.18 together with sargramostim, aldesleukin, and isotretinoin after autologous stem cell transplant in treating patients with neuroblastoma. Monoclonal antibodies, such as Ch14.18, may find tumor cells and help kill them. Colony-stimulating factors, such as sargramostim, may increase the number of immune cells found in bone marrow or peripheral blood. Aldesleukin may stimulate the white blood cells to kill tumor cells. Isotretinoin may help neuroblastoma cells become more like normal cells, and to grow and spread more slowly. Giving monoclonal antibody Ch14.18 with sargramostim, aldesleukin, and isotretinoin after autologous stem cell transplant may be an effective treatment for neuroblastoma.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
105

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Dec 2009

Longer than P75 for phase_3

Geographic Reach
1 country

32 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 21, 2009

Completed
10 days until next milestone

First Submitted

Initial submission to the registry

December 31, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

January 1, 2010

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2013

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

February 19, 2015

Completed
6.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2021

Completed
Last Updated

August 17, 2021

Status Verified

June 1, 2021

Enrollment Period

4 years

First QC Date

December 31, 2009

Results QC Date

February 3, 2015

Last Update Submit

July 30, 2021

Conditions

Outcome Measures

Primary Outcomes (1)

  • Percentage of Patients Who Experienced a Significant (CTC Grade 3-5) Nonhematologic Toxicity of Interest (Pain, Hypotension, Allergic Reactions, Capillary Leak Syndrome, or Fever).

    Designed to collect comprehensive safety/toxicity data, as well as additional efficacy data for the immunotherapy. To address the primary objective, descriptive analyses summarizing the number and type of AEs will be performed. The percentage of patients reporting each unacceptable (Grade 3 or higher) CTC toxicity code, tabulated by course, are reported.

    Up to 6 courses of therapy

Secondary Outcomes (2)

  • Event-free Survival (EFS)

    From enrollment until the first occurrence of relapse, progressive disease, secondary malignancy, or death, or until last contact if no event occurred, up to 3 years

  • Overall Survival (OS)

    From enrollment until death, or until last contact with the patient, up to 3 years

Study Arms (1)

Treatment (Ch14.18, GM-CSF, IL-2, isotretinoin)

EXPERIMENTAL

Patients receive sargramostim SC or IV over 2 hours on days 0-13 of courses 1, 3, and 5; monoclonal antibody Ch14.18 IV over 10 hours on days 3-6 of courses 1, 3, and 5 and on days 7-10 of courses 2 and 4; and isotretinoin PO BID on days 11-24 of course 1, on days 14-27 of courses 2, 4, and 6, and on days 10-23 of courses 3 and 5. Patients also receive aldesleukin IV continuously on days 0-3 and on days 7-10 of courses 2 and 4. Treatment repeats every 24-32 days for 6 courses in the absence of disease progression or unacceptable toxicity.

Biological: AldesleukinOther: Diagnostic Laboratory Biomarker AnalysisBiological: DinutuximabDrug: IsotretinoinBiological: Sargramostim

Interventions

AldesleukinBIOLOGICAL

Given IV

Also known as: 125-L-Serine-2-133-interleukin 2, Proleukin, r-serHuIL-2, Recombinant Human IL-2, Recombinant Human Interleukin-2
Treatment (Ch14.18, GM-CSF, IL-2, isotretinoin)

Correlative studies

Treatment (Ch14.18, GM-CSF, IL-2, isotretinoin)
DinutuximabBIOLOGICAL

Given IV

Also known as: Ch 14.18UTC, Ch14.18, MOAB Ch14.18, monoclonal antibody Ch14.18, Unituxin
Treatment (Ch14.18, GM-CSF, IL-2, isotretinoin)

Given PO

Also known as: 13-cis retinoic acid, 13-cis-Retinoate, 13-cis-Retinoic Acid, 13-cis-Vitamin A Acid, 13-cRA, Absorica, Accure, Accutane, Amnesteem, cis-Retinoic Acid, Cistane, Claravis, Isotretinoinum, Isotrex, Isotrexin, Myorisan, Neovitamin A, Neovitamin A Acid, Oratane, Retinoicacid-13-cis, Ro 4-3780, Ro-4-3780, Roaccutan, Roaccutane, Roacutan, Sotret, ZENATANE
Treatment (Ch14.18, GM-CSF, IL-2, isotretinoin)
SargramostimBIOLOGICAL

Given IV or SC

Also known as: 23-L-Leucinecolony-Stimulating Factor 2, DRG-0012, Leukine, Prokine, rhu GM-CFS, Sagramostim, Sargramostatin
Treatment (Ch14.18, GM-CSF, IL-2, isotretinoin)

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • All patients must be diagnosed with neuroblastoma, and categorized as high-risk at the time of diagnosis
  • At pre-ASCT evaluation, patients must meet the International Neuroblastoma Response Criteria (INRC) for complete response (CR), very good partial response (VGPR), or partial response (PR) for primary site, soft tissue metastases and bone metastases; patients who meet those criteria must also meet the protocol specified criteria for bone marrow response as outlined below: =\< 10% tumor (of total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy; patients who have no tumor seen on the prior bone marrow, and then have =\< 10% tumor on any of the bilateral marrow aspirate/biopsy specimens done at pre-ASCT and/or pre-enrollment evaluation will also be eligible; (Note that, per INRC, this would have been defined as an "overall" response of progressive disease \[PD\])
  • Prior to enrollment on ANBL0931, a determination of residual disease must be performed (tumor imaging studies including metaiodobenzylguanidine \[MIBG\] scan, computed tomography \[CT\] or magnetic resonance imaging \[MRI\], bone marrow aspiration and biopsy); this disease assessment is required for eligibility, and should be done preferably within 2 weeks but must be done within a maximum of 4 weeks before enrollment
  • Patients with residual disease are eligible; biopsy is not required
  • Patients must not have progressive disease except for protocol specified bone marrow response
  • All patients must have completed therapy including intensive induction chemotherapy followed by ASCT and radiotherapy to be eligible; radiotherapy may be waived for patients who either had a small adrenal mass which was completely resected upfront, or who never had an identifiable primary tumor
  • No more than 9 months from the date of starting the first induction chemotherapy after diagnosis to the date of ASCT except for the rare occasions as noted below; for tandem ASCT patients, this will be the date of the FIRST stem cell infusion; Exception: for those who are initially diagnosed as non-high risk neuroblastoma, but later converted (and/ or relapsed) to high risk neuroblastoma, the 9 months restriction should start from the date of induction therapy for high risk neuroblastoma (not from the initial induction therapy for non-high risk disease), to the date of ASCT
  • Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age; required patients must have a Lansky or Karnofsky performance scale score of \>= 50%
  • Patients must have a life expectancy of \>= 2 months (8 weeks)
  • Total absolute phagocyte count (APC = neutrophils + monocytes) is at least 1000/uL
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/gender as follows:
  • month to \< 6 months: 0.4 mg/dL
  • months to \< 1 year: 0.5 mg/dL
  • to \< 2 years: 0.6 mg/dL
  • to \< 6 years: 0.8 mg/dL
  • +12 more criteria

You may not qualify if:

  • Females of childbearing potential must have a negative pregnancy test
  • Patients of childbearing potential must agree to use an effective birth control method
  • Female patients who are lactating must agree to stop breast-feeding
  • Patients must not have received prior anti-GD2 antibody therapy
  • Patients must not have received prior vaccine therapy administered as treatment of neuroblastoma not routine infectious disease vaccinations

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (32)

Loma Linda University Medical Center

Loma Linda, California, 92354, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

Lucile Packard Children's Hospital Stanford University

Palo Alto, California, 94304, United States

Location

Rady Children's Hospital - San Diego

San Diego, California, 92123, United States

Location

UCSF Medical Center-Mount Zion

San Francisco, California, 94115, United States

Location

UCSF Medical Center-Parnassus

San Francisco, California, 94143, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's Healthcare of Atlanta - Egleston

Atlanta, Georgia, 30322, United States

Location

Emory University Hospital/Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States

Location

Indiana University/Melvin and Bren Simon Cancer Center

Indianapolis, Indiana, 46202, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Children's Hospital New Orleans

New Orleans, Louisiana, 70118, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

C S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospitals and Clinics of Minnesota - Minneapolis

Minneapolis, Minnesota, 55404, United States

Location

University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States

Location

New York Medical College

Valhalla, New York, 10595, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Saint Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States

Location

UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, 75390, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

University of Wisconsin Hospital and Clinics

Madison, Wisconsin, 53792, United States

Location

Related Links

MeSH Terms

Interventions

aldesleukindinutuximabIsotretinoinsargramostimColony-Stimulating Factors

Intervention Hierarchy (Ancestors)

RetinoidsCarotenoidsPolyenesAlkenesHydrocarbons, AcyclicHydrocarbonsOrganic ChemicalsCyclohexenesCyclohexanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicTerpenesPigments, BiologicalBiological FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteins

Results Point of Contact

Title
Results Reporting Coordinator
Organization
Children's Oncology Group

Study Officials

  • Mehmet F Ozkaynak

    Children's Oncology Group

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 31, 2009

First Posted

January 1, 2010

Study Start

December 21, 2009

Primary Completion

December 31, 2013

Study Completion

June 30, 2021

Last Updated

August 17, 2021

Results First Posted

February 19, 2015

Record last verified: 2021-06

Locations