18 and older, any sex, with Gliosarcoma or Recurrent Glioblastoma. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Patients With Adverse EventsPrimary· Up to 21 days after study drug administration
To evaluate the safety of intravenous NU-0129 in patients with recurrent GBM or GS, the number of adverse events will be assessed and will be graded according to the NCI's Common Terminology Criteria in Adverse Events (CTCAE) version 4.03 where the grading is as follows:
Grade 1: Mild Grade 2: Moderate Grade 3: Severe Grade 4: Life-threatening Grade 5: Fatal
Experienced any AE
Group
Value
95% CI
NU-0129 Treatment
8
Experienced an AE related to NU-01289
Group
Value
95% CI
NU-0129 Treatment
4
Experienced any SAE
Group
Value
95% CI
NU-0129 Treatment
1
Experienced an SAE related to NU-0129
Group
Value
95% CI
NU-0129 Treatment
0
NU-0129 Concentration in Blood After Drug Administration Using Maximum ConcentrationSecondary· At 1, 3, 5, 10, 30, and 60 minutes, and 4, 8, and 24 hours post infusion
Blood samples will be collected post-infusion to analyze drug concentration at specific time points after drug administration. Median plasma concentrations of NU-0129 were derived from time profiles for both Seven different small interfering RNA (siRNA) and gold (Au) concentrations, with Au plasma concentration determined by inductively coupled plasma mass spectrometry (ICP-MS) and siRNA concentration assessed by liquid chromatography-high performance liquid chromatography (LC-HPLC) using an atto dye-labeled PNA probe.
siRNA maximum observed plasma concentration
Group
Value
95% CI
NU-0129 Treatment
62.1
22.8 – 123
Au maximum observed plasma concentration
Group
Value
95% CI
NU-0129 Treatment
4290
3120 – 7140
Biodistribution of NU-0129 in Tumor TissueSecondary· At time of surgery
Tissue will be collected during the scheduled surgery and assayed with Inductively Coupled Plasma Mass Spectrometry (ICP-MS) to analyze the concentration of particles in various parts of tumor tissue. To analyze spatial distribution of Au within tumor tissue, synchrotron XFM elemental maps of GBM tissue slices were acquired at micron and submicron resolution and matched to adjacent hematoxylin and eosin (H\&E)- and Ki67-stained tumor sections. Approximate percentage of gold (Au) found in cancer cells is reported below.
Group
Value
95% CI
NU-0129 Treatment
20.5
± 8.15
Feasibility of Giving NU-0129 as a Standard TreatmentSecondary· At time of infusion (8-48 hours prior to resection) and during surgery
Feasibility will be calculated as the rate of successful production, delivery, and administration of the investigational product and subsequent resection.
Number of patients who had drug infused successfully
Group
Value
95% CI
NU-0129 Treatment
8
Number of patient who underwent subsequent resection
Group
Value
95% CI
NU-0129 Treatment
8
NU-0129 Concentration in Blood After Drug Administration Using Half-lifeSecondary· At 1, 3, 5, 10, 30, and 60 minutes, and 4, 8, and 24 hours post infusion
Blood samples will be collected post-infusion to analyze drug concentration at specific time points after drug administration. Median plasma concentrations of NU-0129 were derived from time profiles for both Seven different small interfering RNA (siRNA) and gold (Au) concentrations, with Au plasma concentration determined by inductively coupled plasma mass spectrometry (ICP-MS) and siRNA concentration assessed by liquid chromatography-high performance liquid chromatography (LC-HPLC) using an atto dye-labeled PNA probe.
siRNA half-life
Group
Value
95% CI
NU-0129 Treatment
0.06
0.02 – 0.16
Au half-life
Group
Value
95% CI
NU-0129 Treatment
18
12 – 23
Adverse events — posted to ClinicalTrials.gov
Time frame: Patients were monitored for adverse events from the time of NU-0129 infusion through 21 days post infusion.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Northwestern University
Last refreshed: 26 August 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03020017.