Evaluation of Ad-RTS-hIL-12 + Veledimex in Subjects With Recurrent or Progressive Glioblastoma, a Substudy to ATI001-102

NCT03679754 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: ATI001-102 EXP Substudy 2.0
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 4

Brief Summary

This research study involves an investigational product: Ad-RTS-hIL-12 given with veledimex for production of human IL-12. IL-12 is a protein that can improve the body's natural response to disease by enhancing the ability of the immune system to kill tumor cells and may interfere with blood flow to the tumor. The main purpose of this study is to evaluate the safety and tolerability of a single intratumoral injection of Ad-RTS-hIL-12 given with oral veledimex.

Conditions

Glioblastoma

Outcome Measures

Primary Outcomes (2)

• Safety of Intratumoral Ad-RTS-hIL-12 and Oral Veledimex in Subjects With Recurrent or Progressive Glioblastoma Based on Evaluation of Adverse Events Summarized by Incidence, Intensity and Type of Adverse Event.

  Evaluation of adverse events as assessed by CTCAE v4.03. Adverse events will be summarized based on the incidence, intensity and type of adverse event.

  From the first dose of study treatment until 30 days after the last dose of veledimex, lasting approximately 5 months.

• Tolerability of Intratumoral Ad-RTS-hIL-12 and Oral Veledimex in Subjects With Recurrent or Progressive Glioblastoma Will be Assessed Based on Expected Dose Compliance

  The Day 0 (as applicable), and Day 1 doses of veledimex were administered by study personnel at the study center. Thereafter, subjects could self-administer the remaining once daily dose of veledimex. Subjects were instructed to document veledimex dosing compliance in a subject diary, including the time each dose was taken, the time the subject ate the last meal prior to administration of veledimex, the number of capsules taken, whether the subject missed any veledimex doses, and reason for any missed doses. Investigational product container(s) with any remaining capsules were returned to the study staff on Day 15, and staff assessed dose compliance.

  Days 1 through 14 of each 21-day treatment cycle, over a total treatment period of up to 6 cycles (approximately 4 months)

Secondary Outcomes (14)

• Determine the Overall Survival (OS) of Ad-RTS-hIL-12 + Veledimex

  From the first dose of study drug for up to 2 years.

• Veledimex Pharmacokinetic Profile: Maximum Plasma Concentration (Cmax)

  15 days

• Veledimex Pharmacokinetic Profile: Time to Maximum Plasma Concentration (Tmax)

  15 days

• Veledimex Pharmacokinetic Profile: Half-life (t1/2)

  15 days

• Veledimex Pharmacokinetic Profile: Area-under-the-concentration Versus Time Curve (AUC)

  15 days

Study Arms (1)

Ad-RTS-hIL-12 + veledimex

EXPERIMENTAL

Intratumoral Ad-RTS-hIL-12 and oral veledimex

Biological: Ad-RTS-hIL-12; Drug: veledimex

Interventions

Ad-RTS-hIL-12 BIOLOGICAL

* 2.0 x 10\^11 viral particles (vp) per injection * intratumoral injection of Ad-RTS-hIL-12

Ad-RTS-hIL-12 + veledimex

veledimex DRUG

* 20mg/day * 15 oral daily doses of veledimex

Ad-RTS-hIL-12 + veledimex

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subject ≥18 and ≤75 years of age
• Provision of written informed consent for tumor resection, tumor biopsy, samples collection, and treatment with investigational products prior to undergoing any study specific procedures
• Histologically confirmed glioblastoma
• Evidence of supratentorial tumor recurrence/progression by magnetic resonance imaging (MRI) according to Response Assessment in Neuro-Oncology (RANO) criteria after standard initial therapy
• Previous standard-of-care antitumor treatment including surgery and/or biopsy and chemoradiation. At the time of registration, subjects must have recovered from the toxic effects of previous treatments as determined by the treating physician. The washout periods from prior therapies are intended as follows: (windows other than what is listed below should be allowed only after consultation with the Medical Monitor)
• Nitrosureas: 6 weeks
• Other cytotoxic agents: 4 weeks
• Antiangiogenic agents: 4 weeks (NOTE: short use (\< 4 doses) of bevacizumab for controlling edema is allowed)
• Targeted agents, including small molecule tyrosine kinase inhibitors: 2 weeks
• Vaccine-based therapy: 3 months
• Able to undergo standard MRI scans with contrast agent before enrollment and after treatment
• Karnofsky Performance Status ≥70
• Adequate bone marrow reserves and liver and kidney function, as assessed by the following laboratory requirements:
• Hemoglobin ≥9 g/L
• Lymphocytes \>500/mm3

You may not qualify if:

• Previous treatment with bevacizumab for their disease (NOTE: short use (\< 4 doses) of bevacizumab for controlling edema is allowed)
• Subjects receiving systemic corticosteroids during the previous 4 weeks
• Radiotherapy treatment within 4 weeks of starting veledimex
• Subjects with clinically significant increased intracranial pressure (eg, impending herniation or requirement for immediate palliative treatment) or uncontrolled seizures
• Known immunosuppressive disease, or autoimmune conditions, and/or chronic viral infections (eg, human immunodeficiency virus \[HIV\], hepatitis)
• Use of systemic antibacterial, antifungal, or antiviral medications for the treatment of acute clinically significant infection within 2 weeks of first veledimex dose. Concomitant therapy for chronic infections is not allowed. Subjects must be afebrile prior to Ad-RTS-hIL-12 injection; only prophylactic antibiotic use is allowed perioperatively
• Use of enzyme-inducing antiepileptic drugs (EIAED) within 7 days prior to the first dose of study drug. Note: Levetiracetam (Keppra®) is not an EIAED and is allowed
• Other concurrent clinically active malignant disease, requiring treatment, with the exception of non-melanoma cancers of the skin or carcinoma in situ of the cervix or nonmetastatic prostate cancer
• Nursing or pregnant females
• Prior exposure to veledimex
• Use of medications that induce, inhibit, or are substrates of CYP4503A4 within 7 days prior to veledimex dosing without consultation with the Medical Monitor
• Presence of any contraindication for a neurosurgical procedure
• Unstable or clinically significant concurrent medical condition that would, in the opinion of the Investigator or Medical Monitor, jeopardize the safety of a subject and/or their compliance with the protocol. Examples may include, but are not limited to, colitis, pneumonitis, unstable angina, congestive heart failure, myocardial infarction within 2 months of screening, and ongoing maintenance therapy for life-threatening ventricular arrhythmia or uncontrolled asthma

Sponsors & Collaborators

• Alaunos Therapeutics: lead

Study Sites (4)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

Location

NYU - Langone Health

New York, New York, 10016, United States

Location

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Glioblastoma

Interventions

veledimex

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Jaymes Holland
• Organization: Alaunos Therapeutics

jholland@alaunos.com

6502732627

Study Officials

• Jaymes Holland

  Alaunos Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 29, 2018
• First Posted: September 20, 2018
• Study Start: September 5, 2018
• Primary Completion: April 2, 2019
• Study Completion: January 19, 2021
• Last Updated: August 28, 2025
• Results First Posted: August 28, 2025

Record last verified: 2025-08

Locations

US (4)