Study of Niacin in Glioblastoma

NCT04677049 | Suspended Phase 1 DMC

• 1 other identifier: NiacinCNS2020
• Study Type: interventional
• Target: 59
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single institution Phase I-II study to evaluate the tolerability and Maximum Tolerated Dose (MTD) (Phase I) and efficacy (Phase II) of adding Niacin CRT™ to standard first line treatment (concurrent Radiation Therapy (RT) and Temozolomide (TMZ) following by monthly TMZ - AKA Stupp protocol) in patients with newly diagnosed glioblastoma isocitrate dehydrogenase (IDH) wild type.

Conditions

Glioblastoma IDH (Isocitrate Dehydrogenase) Wildtype

Keywords

Glioblastoma

Outcome Measures

Primary Outcomes (2)

• Determining the Maximum Tolerated Dose

  To evaluate and determine maximum tolerated dose (MTD) of Niacin CRT added to concurrent radiotherapy (RT) and temozolomide (TMZ) in patients with newly diagnosed glioblastoma (GB).

  Up to 24 weeks after registration onto the study

• Evaluating if Niacin CRT Improves Glioblastoma Survival Rates

  To evaluate if adding Niacin CRT to current standard first line treatment of GB improves progression free survival (PFS) at 6 months.

  6 months after determining the maximum tolerated dose which can last up to 24 weeks after registration onto the study

Secondary Outcomes (5)

• Effect of Niacin CRT in Peripheral Monocytes

  From date of registration until the date of first documented disease progression or date of death from any cause, whichever came first, assessed up to 5 years.

• Response Rate Associated with Niacin

  From date of registration until the date of first documented disease progression or date of death from any cause, whichever came first, assessed up to 5 years.

• Overall Survival Rate Associated with Niacin

  From date of registration until the date of first documented disease progression or date of death from any cause, whichever came first, assessed up to 5 years.

• Quality of Life While on Study using EORTC QLQ-C30 Questionnaires

  From date of registration until the date of first documented disease progression or date of death from any cause, whichever came first, assessed up to 5 years.

• Quality of Life While on Study using EORTC BN-20 Questionnaires

  From date of registration until the date of first documented disease progression or date of death from any cause, whichever came first, assessed up to 5 years.

Study Arms (1)

Niacin

EXPERIMENTAL

Niacin controlled release technology (CRT): Niacin CRT™ is to be started 7 days before concurrent Radiation Therapy (RT)- Temozolomide (TMZ) treatment. Chemo/Radiation Therapy: For all patients, regardless of the phase of the study, concurrent RT and TMZ for 6 weeks followed by 6-12 cycles of monthly TMZ will be given. Concurrent Temozolomide: TMZ will be administered from the first to the last day of RT at 75 mg/m2 orally (PO) for a maximum of 49 days. Monthly Temozolomide: Cycles of chemotherapy Day 1 to Day 5 every 28 days will start 28 days (+/- 2 days) after the end of RT-TMZ. First cycle of TMZ is administered at 150 mg/m2 Day 1-Day 5 by mouth (PO) and increased to 200 mg/m2 Day 1-Day 5 PO from cycle 2 onwards if well tolerated. While 6 cycles are standard of care, the Neuro-Oncologist may continue up to 12 cycles if clinically appropriate.

Drug: Niacin CRT

Interventions

Niacin CRT DRUG

A controlled release technology (CRT) tablet of Niacin

Also known as: Nicotinic acid, Vitamin B3

Niacin

Eligibility Criteria

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

You may qualify if:

• Adults, 18 years old to 75 years old inclusive.
• New diagnoses of glioblastoma IDH wild type.
• ECOG 0-2 (Appendix I).
• Candidates for concurrent standard first line treatment according to their Neuro-Oncologist and Radiotherapy Oncologist after maximal safe debulking neurosurgery. Patients that only had biopsy are included as long as pathology confirms the diagnoses and it is considered the maximal safe procedure for that patient.
• Adequate hematological, renal and hepatic function (see details in Section 4.1 of the protocol).
• Absence of known human immunodeficiency virus (HIV) infection, chronic hepatitis B or hepatitis C infection.
• Absence of any other serious medical condition according to the medical judgment of the Qualified Investigator prior to registration.
• Absence of any medical condition, which could interfere with oral medication intake.
• Signed informed consent.
• Patients must be accessible for treatment and follow-up. Patients registered on this trial must be treated and followed at the participating centre.
• Women/men of childbearing potential must have agreed to use a highly effective contraceptive method.

You may not qualify if:

• Glioblastoma, IDH-mutant.
• Patients with a history of other malignancies, except: adequately treated non-melanoma skin cancer, curatively treated in-situ cancer of the cervix, or other solid tumours curatively treated with no evidence of disease for ≥ 5 years. Additionally, any low grade or low risk malignancy not requiring treatment will not exclude a patient from participation in the trial.
• Known hypersensitivity to niacin.
• Inability to provide informed consent.
• Active liver disease or unexplained persistent elevations of serum transaminases.
• Active peptic ulcer or active gastrointestinal bleeding.
• Unstable angina or myocardial infarction within 6 months.
• Symptomatic gout.
• Patients on 3-hydroxy-3-methylglutaryl-coenzyme (HMG-COA reductase) inhibitors that cannot discontinue them at least 2 weeks before starting Niacin CRT™.
• Any prior systemic treatment for glioblastoma (standard, evidence based or experimental) or radiotherapy/radiosurgery.
• Individuals with MRI non-compatible metal in the body, or unable to undergo MRI procedures including allergy to gadolinium.
• Patients unfit for any treatment component, including contraindications for radiotherapy or Connective Tissue Disease.
• Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
• Has known psychiatric or substance abuse disorders that would interfere with compliance with the requirements of the trial.
• Pregnant, breast-feeding, unable and/or unwilling to use contraception methods.

Sponsors & Collaborators

• AHS Cancer Control Alberta: lead
• Tom Baker Cancer Centre: collaborator

Study Sites (1)

Tom Baker Cancer Centre/Arthur J E Child Comprehensive Cancer Centre

Calgary, Alberta, T2N 5G2, Canada

Location

Related Publications (1)

• Poon CC, Hagen KM, Sarkar S, Mirzaei R, Silva C, Ueno A, de Robles P, Roldan-Urgoiti G, Yong VW. Niacin Modulates Immune Responses in a Phase I Dose-Escalation Clinical Trial of Newly Diagnosed Glioblastoma. Neurol Neuroimmunol Neuroinflamm. 2026 Mar;13(2):e200530. doi: 10.1212/NXI.0000000000200530. Epub 2026 Feb 3.

  PMID: 41632924 DERIVED

MeSH Terms

Conditions

Glioblastoma

Interventions

Niacin; Niacinamide

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

Intervention Hierarchy (Ancestors)

Nicotinic Acids; Acids, Heterocyclic; Heterocyclic Compounds; Pyridines; Heterocyclic Compounds, 1-Ring

Study Officials

• Gloria Roldan Urgoiti, MD

  Tom Baker Cancer Centre/Arthur J.E. Child Comprehensive Cancer Centre

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: December 3, 2020
• First Posted: December 21, 2020
• Study Start: March 18, 2021
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: January 2, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)