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

NCT03020017 | Completed Early Phase 1 Results DMC FDA Drug

• 4 other identifiers: NU 16C01STU00203790P30CA060553NCI-2016-02007
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Gliosarcoma; Recurrent Glioblastoma

Outcome Measures

Primary Outcomes (1)

• Number of Patients With Adverse Events

  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

  Up to 21 days after study drug administration

Secondary Outcomes (4)

• NU-0129 Concentration in Blood After Drug Administration Using Maximum Concentration

  At 1, 3, 5, 10, 30, and 60 minutes, and 4, 8, and 24 hours post infusion

• Biodistribution of NU-0129 in Tumor Tissue

  At time of surgery

• Feasibility of Giving NU-0129 as a Standard Treatment

  At time of infusion (8-48 hours prior to resection) and during surgery

• NU-0129 Concentration in Blood After Drug Administration Using Half-life

  At 1, 3, 5, 10, 30, and 60 minutes, and 4, 8, and 24 hours post infusion

Study Arms (1)

Treatment (NU-0129)

EXPERIMENTAL

Patients receive NU-0129 IV over 20-50 minutes and undergo standard of care tumor resection within 8-48 hours.

Other: Laboratory Biomarker Analysis; Other: Pharmacological Study; Drug: Targeted Molecular Therapy

Interventions

Laboratory Biomarker Analysis OTHER

Correlative studies

Treatment (NU-0129)

Pharmacological Study OTHER

Correlative studies

Treatment (NU-0129)

Targeted Molecular Therapy DRUG

Given NU-0129 IV

Also known as: molecularly targeted therapy

Treatment (NU-0129)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 =\< 2 x upper limit of normal (ULN)
• Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =\< 2 x ULN
• Creatinine =\< 1.5 x ULN
• Urine protein =\< 3 x ULN

You may not qualify if:

• 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

Sponsors & Collaborators

• Northwestern University: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Northwestern University

Chicago, Illinois, 60611, United States

Location

MeSH Terms

Conditions

Gliosarcoma; Glioblastoma

Interventions

Molecular Targeted Therapy

Condition Hierarchy (Ancestors)

Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Astrocytoma

Intervention Hierarchy (Ancestors)

Drug Therapy; Therapeutics

Results Point of Contact

• Title: Priya Kumthekar
• Organization: Northwestern University

p-kumthekar@northwestern.edu

312 908 5073

Study Officials

• Priya Kumthekar, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Priya Kumthekar, MD

Study Record Dates

• First Submitted: January 11, 2017
• First Posted: January 13, 2017
• Study Start: May 25, 2017
• Primary Completion: September 6, 2018
• Study Completion: August 19, 2020
• Last Updated: August 26, 2022
• Results First Posted: August 26, 2022

Record last verified: 2022-08

Locations

US (1)