Clinical Trials Actively Recruiting Patients With Neuroblastoma

The information contained in this Clinical Trials Resource Guide includes actively recruiting clinical studies for patients with newly diagnosed or relapsed or refractory neuroblastoma. The studies include pilot, observational, phase I, phase II, and phase III trials investigating single-agent and combination chemotherapies, immunotherapies, treatment toxicity, radiation therapy, T-cell transplantation, and proton therapy. All of the studies are listed on the National Institutes of Health website at ClinicalTrials.gov.

Study Type: Pilot/interventional

Study Title: 124I-Metaiodobenzylguanidine (MIBG) PET/CT Diagnostic Imaging and Dosimetry for Patients With Neuroblastoma: A Pilot Study

Study Sponsor and Collaborators: University of California, San Francisco

Purpose: To describe organ dosimetry and acute toxicities using no carrier added and carrier added 124I-MIBG PET/CT in patients with relapsed or refractory neuroblastoma who are currently enrolled on a treatment protocol with 131I-MIBG

Eligible Ages: 3 years or older

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures:

• Measurements of organ dosimetry using high specific activity (no carrier added) 124I-MIBG PET/CT (time frame: PET/CT scans on days 0-week 7)

• Measurements of organ dosimetry using low specific activity (carrier added) 124I-MIBG PET/CT (time frame: PET/CT scans on days 0-week 7)

• Change from baseline of blood pressure measurements at week 7 (time frame:  safety assessments on days 0- week 7)

• Number of participants with grade 3 or 4 imaging-related toxicities (time frame: from baseline up to 6 weeks post final imaging)

• Change from baseline of pulse measurements at 7 weeks (time frame:  safety assessments on days 0-week 7)

Principal Investigator: Katherine Matthay, MD, University of California, San Francisco; 415-476-3831, matthayk@peds.ucsf.edu

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT01583842

Study Type: Observational

Study Title: Neuroblastoma Biology Studies

Study Sponsor and Collaborators: Children’s Oncology Group, National Cancer Institute

Purpose: To study biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglioneuroblastoma

Eligible Ages: up to 30 years

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures:

• Factors currently used for risk-group assignment  (time frame: up to 3 years)

• Prevalence of 1p, 11q, 14q, and 17q allelic status (time frame: up to 3 years)

• MYCN copy number by quantitative PCR(time frame: up to 3 years)

• Expression pattern of neurotrophin-related genes in diagnostic neuroblastoma tumors (time frame: up to 3 years)

• Presence of rare tumor cells in biologic specimens by RT-PCR (time frame: up to 3 years)

• Database of the known biologic prognostic factors for patients on therapeutic studies (time frame: up to 3 years)

Principal Investigator: Michael Hogarty, MD, Children’s Oncology Group; 215-590-3931, hogartym@email.chop.edu

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT00904241

Study Type: Phase I/interventional/single-group assignment

Study Title: Pilot Study to Determine the Utility of [18F] 3'-Deoxy-3'-Fluorothymidine-Positron Emission Tomography in Monitoring Patient Response to Chemotherapy in Neuroblastoma

Study Sponsor and Collaborators: Barbara Ann Karmanos Cancer Institute, Children’s Hospital of Michigan

Purpose: To gain knowledge about how  best to diagnose and treat tumors, how tumors affect normal tissue, and how treatment of tumors with radiation therapy and chemotherapy affect tumors

Eligible Ages: up to 21 years

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures: To determine whether 18F-FLT-PET is a sensitive tool to image neuroblastoma (time frame: 1st PETtt diagnosis)

Principal Investigator: Anthony F. Shields, MD, PhD, Barbara Ann Karmanos Cancer Institute; 313-576-8735, shieldsa@karmanos.org

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT01308905

Study Title: Autologous Activated T Cells Transduced With a 3rd Generation GD-2 Chimeric Antigen Receptor and iCaspase9 Safety Switch Administered to Patients With Relapsed or Refractory Neuroblastoma (GRAIN)

Study Sponsor and Collaborators: Baylor College of Medicine; Center for Cell and Gene Therapy, Baylor College of Medicine; Texas Children’s Hospital; The Methodist Hospital System; Solving Kids’ Cancer; The EVAN Foundation; National Cancer Institute

Purpose: To determine the highest dose of iC9-GD2-CD28-OX40 (iC9-GD2) T cells that can safely be given to patients with relapsed/refractory ­neuroblastoma.

Eigible Ages: N/A

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures: Dose-limiting toxicities at 6 weeks post T-cell infusion (time frame: 6 weeks after infusion of the last dose of iC9-GD2 T cells)

Principal Investigator: Andras A. Heczey, MD, Baylor College of Medicine; contact Helton D. Cruz, 832-824-1798, hdcruz@texaschildrens.org

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT01822652

Study Type: Phase I/II/interventional/single-group assignment

Study Title: Phase I/II Trial of a Bivalent Vaccine With Escalating Doses of the Immunological Adjuvant OPT-821, in Combination With Oral ß-glucan for High-Risk Neuroblastoma

Study Sponsor and Collaborators: Memorial Sloan Kettering Cancer Center

Purpose: To test the safety and effects treatment with a vaccine against neuroblastoma has on the patient and the cancer

Eligible Ages: up to 21 years

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures:

• To determine the maximally tolerated dose of OPT-821 in a vaccine containing two antigens abundantly expressed on neuroblastoma (phase I; time frame: 2 years)

• To improve event-free survival of patients who are in second (or later) complete/very good partial remission (phase II; time frame: 2 years) 

• To assess anti-NB activity of the bivalent vaccine plus oral β-glucan in patients who are enrolled with evidence of minimal residual disease by molecular biological testing of bone marrow (phase II; time frame: 2 years)

Principal Investigator: Brian Kushner, MD, Memorial Sloan Kettering Cancer Center; 212-639-6793

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT00911560

Study Title: Treatment of Neuroblastoma and GD-2 Positive Tumors With Activated T Cells Armed With OKT3 X Humanized 3F8 Bispecific Antibodies (GD2Bi): A Phase I/II Study

Study Sponsor and Collaborators: Barbara Ann Karmanos Cancer Institute, National Cancer Institute

Purpose: To study the side effects and best dose of activated T cells armed with GD2 bispecific antibody and how well they work in treating patients with neuroblastoma, osteosarcoma, and other GD-2 positive solid tumors

Eligible Ages: 13 months to 29 years

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures: Maximum tolerated dose  of GD2Bi-aATC [(time frame: 35 days)

Principal Investigator: Maxim Yankelevich, MD, Barbara Ann Karmanos Cancer Institute; 313-745-5516, myankele@med.wayne.edu

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT02173093

Study Type: Phase II/interventional/single-group assignment

Study Title: Phase II Study of Proton Radiation Therapy for Neuroblastoma

Study Sponsor and Collaborators: Massachusetts General Hospital, National Cancer Institute 

Purpose: To evaluate the effectiveness of using proton radiation  in the treatment of neuroblastoma to reduce side effects associated with radiation treatment, and to assess late side effects experienced by participants in the treatment group

Eligible Ages: 3  25 years

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures: To describe late complications of radiation therapy delivered with proton radiotherapy in place of photon radiation (time frame: 5 years); and to evaluate acute and subacute toxicities of proton radiotherapy in place of photon radiation (time frame: 5 years)

Principal Investigator: Shannon MacDonald, MD, Massachusetts General Hospital; 617-726-2000, smacdonald@partners.org

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT02112617

Study Title: Neuroblastoma Protocol 2012: Therapy for Children With Advanced Stage High-Risk Neuroblastoma

Study Sponsor and Collaborators: St. Jude Children’s Research Hospital, Cookies for Kids’ Cancer, CURE Childhood Cancer Inc

Purpose: To study the efficacy of two initial courses of cyclophosphamide and topotecan combined with hu14.18K322A (4 doses/course followed by GM-CSF) in previously untreated children with high-risk neuroblastoma.

Eligible Ages: up to 18 years

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures: Number of participants with complete or partial response (time frame: after two initial courses of chemotherapy; approximately 6 weeks after enrollment)

Principal Investigator: Wayne L. Furman, MD, St. Jude Children’s Research Hospital; 866-278-5833, info@stjude.org

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT01857934

Study Type: Phase III/interventional/parallel assignment

Study Title: Phase III Randomized Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue

Study Sponsor and Collaborators: National Cancer Institute

Purpose: To study isotretinoin with dinutuximab, aldesleukin, and sargramostim, as opposed to isotretinoin alone, following stem cells transplant in treating patients with neuroblastoma

Eligible Ages: up to 30 years

Eligible Genders: Both

Accepts Healthy Volunteers: No

Primary Outcome Measures: Event-free survival (time frame: from study enrollment until the first occurrence of an event or until last contact with the patient if no event occurs, assessed up to 3 years)

Principal Investigator: Alice Yu, MD, Children’s Oncology Group; visit ClinicalTrials.gov to see all study locations and regional contacts.

For More Information: Visit ClinicalTrials.gov and refer to this study by its identifier: NCT00026312

Editor’s Note: The clinical trials presented here do not represent all the trials listed on ClinicalTrials.gov. For the complete list, go to ClinicalTrials.gov. ■