Early Phase 1
N=8
NU-0129 in Treating Patients With Recurrent Glioblastoma or Gliosarcoma Undergoing Surgery
Gliosarcoma · Recurrent Glioblastoma
Bottom Line
View on ClinicalTrials.gov: NCT03020017 ↗Enrolled (actual)
8
Serious AEs
12.5%
Results posted
Aug 2022
Primary outcome: Primary: Number of Patients With Adverse Events — 8; 4; 1; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Early Phase 1
- Interventions
- Laboratory Biomarker Analysis (Other); Pharmacological Study (Other); Targeted Molecular Therapy (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Northwestern University
- Primary completion
- Sep 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Adverse Events |
8; 4; 1; 0 | — |
| SECONDARY NU-0129 Concentration in Blood After Drug Administration Using Maximum Concentration |
62.1; 4290 | — |
| SECONDARY Biodistribution of NU-0129 in Tumor Tissue |
20.5 | — |
| SECONDARY Feasibility of Giving NU-0129 as a Standard Treatment |
8; 8 | — |
| SECONDARY NU-0129 Concentration in Blood After Drug Administration Using Half-life |
0.06; 18 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Patients must have histologically proven glioblastoma multiforme (GBM) or gliosarcoma (GS)
- Patients must have measurable disease by Response Assessment in Neuro-Oncology (RANO) 2010 criteria at the time of registration (pre-operative)
- Patients must have failed at least one regimen of chemo or radiation therapy; NOTE: There is no limit to the number or types of prior therapy
- The patient must be a candidate for surgical debulking (either subtotal or gross total resection); biopsy-only candidates will not be eligible
- All patients must be capable to voluntarily sign an informed consent indicating that they are aware of the investigational nature of this study prior to registration
- Patients must have a Karnofsky performance status of >= 70
- Patients must have adequate bone marrow, liver, coagulation and renal function within 7days prior to study registration, as defined below:
- White blood cell count (WBC) >= 3,000/uL
- Absolute neutrophil count (ANC) >= 1,500/mm^3
- Platelet count of >= 100, 000/mm^3 (Note: Transfusion or growth factor may be used for eligibility outside of 7 days)
- Hemoglobin >= 8 mg/dL (Note: Transfusion may be used for eligibility outside of 7 days)
- Bilirubin = 12 months)
- FOCBP must have a negative pregnancy test (either urine or serum) within 14 days prior to registration
Exclusion Criteria
- Patients must not have any significant infections or medical illnesses that in the investigator's opinion cannot be adequately controlled with appropriate therapy or would compromise the patient's ability to tolerate NU-0129
- Patients must not have a history of any other cancer unless they are in complete remission and off of all therapy for that disease for a minimum of 3 years
- Note: Non-melanoma skin cancer or carcinoma in-situ of the cervix are exceptions and may be permitted after discussion with study quality assurance manager (QAM)
- Patients must not have had radiation therapy within 12 weeks prior to registration
- Patients must not have had prior cancer therapy (including biologic, cytotoxic, and experimental therapies, nitrosoureas, and Gliadel wafers or other surgically implantable antitumor treatment) within 21 days of registration; if questions arise, please ask the principal investigator (PI)
- NOTE: Patients must not have Novocure within 24 hours
- Hormonal tumor therapies should not be administered within 14 days of registration; exceptions may be discussed with the PI
- Patients must not have symptomatic hypertension
- Patients with known human immunodeficiency virus (HIV) infection or chronic or acute hepatitis B or C are not eligible; Note: Patients do not need to have HIV, hepatitis B, or hepatitis C testing at screening
- Female patients who are pregnant or breast feeding are not eligible
- Patients are not eligible if they are unwilling or unable to comply with the protocol
Data sourced from ClinicalTrials.gov (NCT03020017). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.