Medical Affairs

Study Title

A Phase II, Open Label, Two-Arm Study of Therapeutic Iobenguane (¹³¹I) as Single Agent or in Combination with Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects (OPTIMUM TRIAL)

Purpose of the Study

To determine the efficacy and safety of ¹³¹I-MIBG treatment in combination with vorinostat in subjects with iobenguane avid, recurrent or progressive high-risk neuroblastoma.

Protocol Summary

The study is conducted in the United States and will include up to 70 high-risk neuroblastoma patients. Eligible patients will receive up to two cycles of vorinostat, once daily for 14 days, and ¹³¹I-MIBG treatment (as a single infusion per cycle).

Participation in the study may involve up to 14 visits over a 2 1/2-year period, depending on the number of treatment cycles received and the response to treatment.

Basic Eligibility Criteria

• Patients 1 year of age or older
• Diagnosis of iobenguane avid high-risk neuroblastoma
• Refractory, recurrent or progressive disease
• May have had prior ¹³¹I-MIBG therapy

Sites Participating in the Study

• Dana-Farber/Boston Children’s Cancer and Blood Disorders Center (Boston, MA)
• Northwell Health (Long Island, NY)
• Children's Hospital of Philadelphia (Philadelphia, PA)
• UPMC Children’s Hospital of Pittsburgh (Pittsburgh, PA)
• Atrium Health (Charlotte, NC)
• Nemours Children’s Health (Jacksonville, FL)
• Cincinnati Children's Hospital Medical Center (Cincinnati, OH)
• University of Wisconsin, American Family Children’s Hospital (Madison, WI)
• University of Chicago (Chicago, IL)
• University of Iowa (Iowa City, IA)
• Washington Univ. Medical Center St. Louis (St Louis, MO)
• University of Minnesota (Minneapolis, MN)
• Cook Children's Health Care System (Fort Worth, TX)
• Children’s Health (Dallas, TX)
• Texas Children's Hospital (Houston, TX)
• Children’s Hospital Colorado (Aurora, CO)
• University of California (San Francisco, CA)
• Seattle Children's Hospital (Seattle, WA)