NCT03020017

NU-0129 in Treating Patients With Recurrent Glioblastoma or Gliosarcoma Undergoing Surgery

A Phase 0 First-In-Human Study Using NU-0129: A Spherical Nucleic Acid (SNA) Gold Nanoparticle Targeting BCL2L12 in Recurrent Glioblastoma Multiforme or Gliosarcoma Patients

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Northwestern University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this research study is to evaluate the safety of the study drug, NU-0129, based on Spherical Nucleic Acid (SNA) platform when infused in patients with recurrent glioblastoma multiforme or gliosarcoma. The SNA consists of nucleic acids arranged on the surface of a small spherical gold nanoparticle. This is a first-in-human trial to determine the safety of NU-0129. NU-0129 can cross the blood brain barrier (a filtering mechanism that carry blood to the brain). Once within the tumor, the nucleic acid component is able to target a gene called Bcl2L12 that is present in glioblastoma multiforme, and is associated with tumor growth. This gene prevents tumor cells from apoptosis, which is the process of programmed cell death, thus promoting tumor growth. Researchers think that targeting the Bcl2L12 gene with NU-0129 will help stop cancer cells from growing.

Detailed description

PRIMARY OBJECTIVES: I. To assess the safety of intravenous NU-0129 in patients with recurrent glioblastoma multiforme (GBM) or gliosarcoma (GS). SECONDARY OBJECTIVES: I. To analyze drug concentration in serum at specific time points after drug administration. II. To demonstrate intratumoral penetration of NU-0129. III. To assess the feasibility of giving NU-0129 as a standard treatment for recurrent GBM or GS. TERTIARY OBJECTIVES: I. To analyze tumor tissue for Bcl2L12 expression levels after NU-0129 administration. II. Preliminary response (progression free survival \[PFS\] and overall survival \[OS\] at 6 months; overall response rate \[ORR\]). OUTLINE: Patients receive NU-0129 intravenously (IV) over 20-50 minutes and undergo standard of care tumor resection within 8-48 hours. After completion of study treatment, patients are followed up at 7, 14, 21, and 28 days and then every 84 days for up to 2 years.

Conditions

• Gliosarcoma
• Recurrent Glioblastoma

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
OTHER	Pharmacological Study	Correlative studies
DRUG	Targeted Molecular Therapy	Given NU-0129 IV

Timeline

• Start date: 2017-05-25
• Primary completion: 2018-09-06
• Completion: 2020-08-19
• First posted: 2017-01-13
• Last updated: 2022-08-26
• Results posted: 2022-08-26

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study