Azeliragon for Glioblastoma

Not currently recruiting at 8 trial locations

Overseen By Stephen G Marcus, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Cantex Pharmaceuticals

Must not be taking: CYP 2C8 inhibitors

Disqualifiers: Prior invasive malignancy, Recurrent disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, azeliragon, to determine its safety and effectiveness when combined with radiation therapy as an initial treatment for glioblastoma, a fast-growing brain cancer. Glioblastoma can invade healthy brain tissue, making it difficult to treat, so new treatments are essential. Participants take azeliragon daily before, during, and after radiation therapy. Individuals with a recent glioblastoma diagnosis that hasn't spread beyond the brain and who haven't received certain prior treatments might be suitable for this trial. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to significant advancements in glioblastoma treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does exclude patients who are taking CYP 2C8 inhibitors. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that azeliragon is likely to be safe for humans?

Research has shown that azeliragon has been tested in over 2,000 patients with Alzheimer's disease, yielding promising safety results. In these trials, most patients tolerated the treatment well, suggesting it might be safe for other uses too. Although limited data exists on its use for glioblastoma, azeliragon has received "orphan drug" status for this condition, indicating potential and sufficient safety for further study. However, despite its safety in past trials, each new study is crucial to confirm these results in different contexts, such as when combined with radiation therapy for glioblastoma. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for glioblastoma, such as chemotherapy and radiation, aim to destroy rapidly dividing cancer cells. But Azeliragon works differently by targeting a receptor called RAGE (Receptor for Advanced Glycation End-products), which is involved in inflammation and tumor growth. This unique mechanism offers a fresh approach, potentially slowing down the progression of the disease by interfering with the pathways that support tumor development. Researchers are excited about Azeliragon because it could enhance the effectiveness of existing therapies and improve outcomes for patients with this aggressive brain cancer.

What evidence suggests that azeliragon might be an effective treatment for glioblastoma?

Research has shown that azeliragon, which participants in this trial will receive, might help treat glioblastoma by blocking a protein called RAGE. This protein is believed to aid cancer growth and resistance to other treatments. In animal studies, azeliragon slowed tumor growth. This trial tests whether the drug works well with radiation therapy to combat glioblastoma. Although it's still early, these findings suggest azeliragon could offer a new approach to tackling this aggressive brain cancer. ^{1 2 3 4 5}

Who Is on the Research Team?

Stephen G Marcus, MD

Principal Investigator

Cantex Pharmaceuticals

Are You a Good Fit for This Trial?

Adults with a specific brain cancer called glioblastoma that hasn't spread, who haven't had certain other cancers or treatments, and whose major organs are functioning well. They must have had surgery for tumor removal but not just a needle biopsy. Participants need to be able to follow the study plan and agree to use birth control if they can have children.

Inclusion Criteria

I have fully recovered from any surgery or its complications before joining the study.

My diagnosis is IDH-wildtype glioblastoma, WHO grade 4.

Patient has been informed about the nature of the study, and has agreed to participate in the study, and signed the Informed Consent Form (ICF) prior to participation in any study-related activities (legally authorized representative permitted)

Exclusion Criteria

Patients with any condition (e.g., psychological, geographical, etc.) that does not permit compliance with the protocol

I do not have a serious infection or health condition that would stop me from following the study treatment.

I have a stomach or intestine condition that affects my eating or digestion.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Radiation and Treatment

Participants receive daily oral azeliragon in combination with radiation therapy

6-8 weeks

Daily visits for radiation therapy

Continued Treatment

Continued administration of azeliragon after completion of radiation therapy

Up to 18 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

• Azeliragon

Trial Overview The trial is testing Azeliragon combined with radiation therapy as an initial treatment for glioblastoma without MGMT promoter methylation. It's in phase 2, which means it's checking safety and how well it might work after showing promise in earlier research.

How Is the Trial Designed?

Group I: Daily oral azeliragon Experimental Treatment 1 Intervention

Azeliragon to be administered once daily for several days before, during, and after radiation therapy.

Who Is Running the Clinical Trial?

Cantex Pharmaceuticals

Lead Sponsor

Trials

14

Recruited

390+

Medpace, Inc.

Industry Sponsor

Trials

98

Recruited

30,400+

Dr. August J. Troendle

Medpace, Inc.

Chief Executive Officer since 1992

MD from the University of Maryland, School of Medicine; MBA from Boston University

Dr. Reinilde Heyrman

Medpace, Inc.

Chief Medical Officer since 2017

MD

Citations

1. onclive.com onclive.com/view/fda-grants-orphan-drug-designation-to-azeliragon-for-gbm

FDA Grants Orphan Drug Designation to Azeliragon for ... By preventing interaction of RAGE with these ligands, azeliragon may inhibit glioblastoma and overcome its resistance to effective treatment.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11718873/

The RAGE Inhibitor TTP488 (Azeliragon) Demonstrates ... Azeliragon (1 mg/kg, i.p.) or vehicle control was started on day 14 post-tumor implantation. Mice were euthanized at day 24 post-tumor implantation, and tumors ...

3. targetedonc.com targetedonc.com/view/fda-okays-phase-2-study-of-azeliragon-for-patients-with-glioblastoma

FDA Okays Phase 2 Study of Azeliragon for Patients With ... Based on the FDA's review of a new drug application, a phase 2 study will evaluate the safety and efficacy of azeliragon for patients with glioblastoma.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05789589

Study Details | NCT05789589 | Effect of Azeliragon ... To determine the safety and efficacy of using the drug azeliragon combined with stereotactic radiosurgery. Specifically, to determine if this combination ...

5. targetedonc.com targetedonc.com/view/fda-grants-orphan-drug-designation-to-azeliragon-for-patients-with-glioblastoma

FDA Grants Orphan Drug Designation to Azeliragon for ... Through preventing interaction of RAGE with these ligands, azeliragon may inhibit GBM and overcome its resistance to effective treatment.