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

NCT ID: NCT05789589

Last Updated: 2025-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

46 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-11-17

Study Completion Date

2027-08-31

Brief Summary

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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).

Detailed Description

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Conditions

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Cancer Metastasis Metastatic Cancer Brain Metastases Brain Metastases, Adult

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Azeliragon and Stereotactic Radiosurgery (SRS)

In the Phase 1 portion of the study, three treatment regimens will be systematically evaluated:

1. Azeliragon + SRS + loading corticosteroid dose (LD) + corticosteroid taper (CT)
2. Azeliragon + SRS + loading corticosteroid dose (LD)
3. Azeliragon + SRS

The starting cohort will receive Regimen #2, and depending on the tolerability, participants will be allocated to subsequent cohorts as follows: if Regimen #2 is not well tolerated, participants will be allocated to Regimen #1; if #2 is well tolerated, participants will be allocated to Regimen #3.

Once a Regimen has been identified as safe and tolerable, it will be used for the Phase 2 portion of the study.

Group Type EXPERIMENTAL

Stereotactic radiosurgery

Intervention Type RADIATION

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

Azeliragon

Intervention Type DRUG

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)

Corticosteroid

Intervention Type DRUG

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).

Interventions

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Stereotactic radiosurgery

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

Intervention Type RADIATION

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)

Intervention Type 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).

Intervention Type DRUG

Other Intervention Names

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TTP488 PF-04494700 dexamethasone methylprednisolone

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Miami Cancer Institute

OTHER

Sponsor Role collaborator

Cantex Pharmaceuticals Inc.

UNKNOWN

Sponsor Role collaborator

Baptist Health South Florida

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Minesh Mehta, M.D.

Role: PRINCIPAL_INVESTIGATOR

Miami Cancer Institute at Baptist Health, Inc.

Yazmin Odia, M.D.

Role: PRINCIPAL_INVESTIGATOR

Miami Cancer Institute at Baptist Health, Inc.

Locations

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Miami Cancer Institute at Baptist Health, Inc.

Miami, Florida, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Minesh Mehta, M.D.

Role: CONTACT

Phone: (786) 596-2000

Email: [email protected]

Kristy Reyes

Role: CONTACT

Phone: (786) 596-2000

Email: [email protected]

Facility Contacts

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Minesh Mehta, M.D.

Role: primary

Kristy M Reyes

Role: backup

References

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Chen X, Zhang L, Zhang IY, Liang J, Wang H, Ouyang M, Wu S, da Fonseca ACC, Weng L, Yamamoto Y, Yamamoto H, Natarajan R, Badie B. RAGE expression in tumor-associated macrophages promotes angiogenesis in glioma. Cancer Res. 2014 Dec 15;74(24):7285-7297. doi: 10.1158/0008-5472.CAN-14-1240. Epub 2014 Oct 17.

Reference Type BACKGROUND
PMID: 25326491 (View on PubMed)

van Grinsven EE, Nagtegaal SHJ, Verhoeff JJC, van Zandvoort MJE. The Impact of Stereotactic or Whole Brain Radiotherapy on Neurocognitive Functioning in Adult Patients with Brain Metastases: A Systematic Review and Meta-Analysis. Oncol Res Treat. 2021;44(11):622-636. doi: 10.1159/000518848. Epub 2021 Sep 3.

Reference Type BACKGROUND
PMID: 34482312 (View on PubMed)

Yang L, Liu Y, Wang Y, Li J, Liu N. Azeliragon ameliorates Alzheimer's disease via the Janus tyrosine kinase and signal transducer and activator of transcription signaling pathway. Clinics (Sao Paulo). 2021 Mar 8;76:e2348. doi: 10.6061/clinics/2021/e2348. eCollection 2021.

Reference Type BACKGROUND
PMID: 33681944 (View on PubMed)

Davis HM, Essex AL, Valdez S, Deosthale PJ, Aref MW, Allen MR, Bonetto A, Plotkin LI. Short-term pharmacologic RAGE inhibition differentially affects bone and skeletal muscle in middle-aged mice. Bone. 2019 Jul;124:89-102. doi: 10.1016/j.bone.2019.04.012. Epub 2019 Apr 24.

Reference Type BACKGROUND
PMID: 31028960 (View on PubMed)

Riuzzi F, Sorci G, Sagheddu R, Chiappalupi S, Salvadori L, Donato R. RAGE in the pathophysiology of skeletal muscle. J Cachexia Sarcopenia Muscle. 2018 Dec;9(7):1213-1234. doi: 10.1002/jcsm.12350. Epub 2018 Oct 18.

Reference Type BACKGROUND
PMID: 30334619 (View on PubMed)

Chiappalupi S, Sorci G, Vukasinovic A, Salvadori L, Sagheddu R, Coletti D, Renga G, Romani L, Donato R, Riuzzi F. Targeting RAGE prevents muscle wasting and prolongs survival in cancer cachexia. J Cachexia Sarcopenia Muscle. 2020 Aug;11(4):929-946. doi: 10.1002/jcsm.12561. Epub 2020 Mar 11.

Reference Type BACKGROUND
PMID: 32159297 (View on PubMed)

Related Links

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http://cancer.baptisthealth.net/

Miami Cancer Institute

Other Identifiers

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2022-KOT-002

Identifier Type: -

Identifier Source: org_study_id