Effect of Azeliragon Combined With Stereotactic Radiation Therapy in Patients With Brain Metastases

A Phase I/II Study to Assess Safety and Preliminary Evidence of a Therapeutic Effect of Azeliragon Combined With Stereotactic Radiation Therapy in Patients With Brain Metastases

Effect of Azeliragon Combined With Stereotactic Radiation Therapy in Patients With Brain Metastases (NCT05789589) is a Phase 1 / Phase 2 interventional studying Cancer and Metastasis, sponsored by Baptist Health South Florida. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Cancer, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 46 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Patient must have diagnosed by tissue sample (biopsy-confirmed) diagnosis of cancer within the past 5 years. If original histologic proof of malignancy is \> 5 years, then biological \[such as presence of tumor markers, circulating tumor (ctDNA), etc.\], or pathological (i.e., more recent) confirmation is required (e.g., from a systemic metastasis or brain metastasis) 2. Age ≥ 18 3. Karnofsky performance status ≥ 50 or Eastern Cooperative Oncology Group (ECOG) ≤ 3 4. Brain metastasis with a maximum tumor diameter of the largest lesion ≤ 2 cm 5. Patients must have discontinued corticosteroids at least 5 days prior to SRS. (Note: This does not apply to corticosteroids administered as part of this protocol.) 6. Patients must not be pregnant (positive pregnancy test) or breast feeding. Effective contraception (men and women) must be used in patients of child-bearing potential during radiotherapy and for 6 months after. 7. Patients who have received prior SRS are eligible, provided that there are new non-irradiated brain lesions and that the patient is ≥ 2 months post prior cranial radiation therapy 8. Patient has adequate biological parameters as demonstrated by the following blood counts at Screening (obtained ≤ 14 days prior to enrollment): - Absolute neutrophil count (ANC) ≥ 1.0 × 10\^9/L - Platelet count ≥ 75,000/mm\^3 (75 × 10\^9/L) - Hemoglobin (Hgb) ≥ 9 g/dL without transfusion or growth factor support 9. Absolute neutrophil count (ANC) ≥ 1.0 × 10\^9/L; Platelet count ≥ 75,000/mm\^3 (75 × 10\^9/L); Hemoglobin (Hgb) ≥ 9 g/dL without transfusion or growth factor support 10. Patient has the following blood chemistry levels at Screening (obtained ≤ 14 days prior to enrollment): - Aspartate aminotransferase (AST; SGOT), alanine transaminase (ALT; SGPT) ≤ 2.5 × upper limit of normal range (ULN). Total bilirubin ≤ 1.5 × ULN. - Estimated creatinine clearance of \> 60 mL/min (per Cockcroft-Gault formula) Who Should NOT Join This Trial: ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: 1. Patient must have histologically or cytologically confirmed diagnosis of cancer within the past 5 years. If original histologic proof of malignancy is \> 5 years, then biological \[such as presence of tumor markers, circulating tumor (ctDNA), etc.\], or pathological (i.e., more recent) confirmation is required (e.g., from a systemic metastasis or brain metastasis) 2. Age ≥ 18 3. Karnofsky performance status ≥ 50 or Eastern Cooperative Oncology Group (ECOG) ≤ 3 4. Brain metastasis with a maximum tumor diameter of the largest lesion ≤ 2 cm 5. Patients must have discontinued corticosteroids at least 5 days prior to SRS. (Note: This does not apply to corticosteroids administered as part of this protocol.) 6. Patients must not be pregnant (positive pregnancy test) or breast feeding. Effective contraception (men and women) must be used in patients of child-bearing potential during radiotherapy and for 6 months after. 7. Patients who have received prior SRS are eligible, provided that there are new non-irradiated brain lesions and that the patient is ≥ 2 months post prior cranial radiation therapy 8. Patient has adequate biological parameters as demonstrated by the following blood counts at Screening (obtained ≤ 14 days prior to enrollment): * Absolute neutrophil count (ANC) ≥ 1.0 × 10\^9/L * Platelet count ≥ 75,000/mm\^3 (75 × 10\^9/L) * Hemoglobin (Hgb) ≥ 9 g/dL without transfusion or growth factor support 9. Absolute neutrophil count (ANC) ≥ 1.0 × 10\^9/L; Platelet count ≥ 75,000/mm\^3 (75 × 10\^9/L); Hemoglobin (Hgb) ≥ 9 g/dL without transfusion or growth factor support 10. Patient has the following blood chemistry levels at Screening (obtained ≤ 14 days prior to enrollment): * Aspartate aminotransferase (AST; SGOT), alanine transaminase (ALT; SGPT) ≤ 2.5 × upper limit of normal range (ULN). Total bilirubin ≤ 1.5 × ULN. * Estimated creatinine clearance of \> 60 mL/min (per Cockcroft-Gault formula) Exclusion Criteria: 1. Patients with leptomeningeal disease 2. Patients unable to undergo magnetic resonance imaging (MRI) 3. Patients receiving Cytochrome P450 (CYP) 2C8 inhibitors as indicated in the protocol 4. Patients with a gastrointestinal condition that could interfere with swallowing or absorption. 5. Females of childbearing potential who are sexually active or males with female partners of childbearing potential, where either the female or the male is unwilling to use a highly effective method of contraception during the trial and for 6 months after the last administration of azeliragon. 6. Patients with concurrent participation in another interventional clinical trial or use of another investigational agent within 7 days of starting azeliragon. Patients who are participating in non-interventional clinical trials (e.g., QOL, imaging, observational, follow-up studies, etc.) are eligible, regardless of the timing of participation. 7. Any patient that in the opinion of the principal investigator is not an appropriate candidate for this trial

Treatments Being Tested

DRUG

Azeliragon

Dosing will begin on Day 0 with the loading dose and continue daily through Day 7. Starting on Day 8, dosing will resume with the continuous dose until disease progression or 8 weeks. If there is evidence of antitumor effect at 8 weeks, dosing may continue for up 2 two years. All doses are taken orally. There are three levels of dosing, including a starting dose and two lower levels of dosing. Participants will start with the starting dose, and in the event of the dose limiting toxicities, the dose will be reduced as described below. Starting Dose Level: 30 mg twice daily (Loading Dose) or 20 mg once daily (Continuous Dose) Dose Level -1: 15 mg twice daily (Loading Dose) or 10 mg once daily (Continuous Dose) Dose Level -2: 15 mg once daily (Loading Dose) or 5 mg once daily (Continuous Dose)

RADIATION

Stereotactic radiosurgery

Patients will undergo standard of care SRS as per the treating facility's policies.

DRUG

Corticosteroid

Two corticosteroid regimens are used depending on the study cohort. Cohorts 1 and 2 will receive the loading dose (LD). Only Cohort 1 will receive the corticosteroid taper (CT). LD: Oral (8 mg) or IV bolus dose (10 mg) of dexamethasone or 40 to 80 mg of methylprednisolone on the day of SRS CT: Oral 2-4 mg of dexamethasone twice daily for 5 days and then 2-4 mg daily for 5 days at the discretion of the treating physician (concurrent use of a proton pump inhibitor or H2 receptor antagonists are encouraged during the CT).

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Miami Cancer Institute at Baptist Health, Inc.

Miami, Florida, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT05789589), the sponsor (Baptist Health South Florida), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT05789589 clinical trial studying?

To determine the safety and efficacy of using the drug azeliragon combined with stereotactic radiosurgery. Specifically, to determine if this combination will lead to improved response in the brain (tumor shrinking in size) and overall tumor control (how long tumor remains controlled). The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT05789589?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT05789589?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT05789589. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT05789589. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.