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Solid TumorPhase 1NCT06724926

Concurrent Azeliragon With Craniospinal Irradiation

A Phase 1 study testing Azeliragon and CSI in people with Solid Tumor and High-grade Glioma, run by NYU Langone Health at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

Single institution study to assess the safety of concurrent Azeliragon with craniospinal irradiation (CSI) in patients with leptomeningeal metastasis from solid tumor malignancies and high-grade gliomas. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Azeliragon and CSI
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
1 siteUnited States
Study dates
18 Feb 2025to Jan 2031
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patient with solid tumor malignancy or high grade glioma with leptomeningeal metastasis established radiographically and/or through cerebrospinal fluid (CSF) cytology
  • Patients who are candidates for radiation therapy for the treatment of leptomeningeal metastasis
  • Patients with Karnofsky Performance Status of 60 or greater.
  • Male or non-pregnant and non-lactating female and ≥ 18 years of age.
  • Patients with absolute neutrophil count (ANC) ≥ 1.0 × 109/L, platelet count ≥ 75,000/mm3 (75 × 109/L), and hemoglobin (Hgb) ≥ 8 g/dL. Transfusion or growth factor support is allowed.
  • Patients with aspartate aminotransferase (AST) [serum glutamic-oxaloacetic transaminase(SGOT)], alanine aminotransferase (ALT) [serum glutamic-pyruvic transaminase (SGPT)] ≤ 2.5 × upper limit of normal range (ULN), unless liver metastases are present, then ≤ 5 x ULN is acceptable, total bilirubin ≤ 1.5 × ULN, and estimated creatinine clearance of > 30 mL/min (per Cockroft-Gault formula).
  • 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.

Who cannot join

  • Patient has a life expectancy, per investigator assessment, of less than 2 months.
  • Patients unable to complete the English quality of life questionnaires
  • Patient with extensive systemic disease and who declined standard systemic treatment options
  • Patient who is unable to undergo magnetic resonance imaging (MRI) brain and spine with gadolinium contrast
  • Previous radiotherapy to the intended treatment site that precludes developing a treatment plan that respects normal tissue tolerance
  • Active, uncontrolled bacterial, or fungal infection(s) requiring systemic therapy.
Show all 11Show fewer
  • Patient has a concomitant serious medical or psychiatric illness that, in the opinion of the investigator, could compromise the patient's safety or the study data integrity.
  • Patient is unwilling or unable to comply with study procedures, including, but not limited to self-administration of oral medication.
  • Patients with a gastrointestinal condition that could interfere with swallowing or absorption.
  • 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 study drug.
  • Pregnant or lactating women

Trial locations and contacts

1 site in 1 country.

United States1 site

  • NYU Langone Health

    New York, New York, 10016

Other trials in United States

About this study

Official title: A Phase IB Study to Assess Safety of Concurrent Azeliragon With Craniospinal Irradiation in Patients With Leptomeningeal Metastasis From Solid Tumor Malignancies or High-Grade Gliomas

Single institution study to assess the safety of concurrent Azeliragon with craniospinal irradiation (CSI) in patients with leptomeningeal metastasis from solid tumor malignancies and high-grade gliomas.

Being tested
Azeliragon, CSI
Medical subject headings
Glioma, Meningeal Carcinomatosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AzeliragonDrug

    Azeliragon is orally administered. Patients start Azeliragon 7 days before CSI and take their last dose 7 days post-CSI.

    The first 6 patients will start at Dose Level 1 [DL1] (Loading dose for 7 days: 30 mg twice daily; Concurrent/adjuvant dose: 20 mg once daily).

    If 1 or fewer of 6 patients develop dose-limiting toxicity (DLT) then the recommended dose will be DL1. If at least 2 of 6 develops a DLT, then an additional 6 patients will be enrolled at Dose Level -1 [DL-1] (Loading dose for 7 days: 15mg twice daily; Concurrent/adjuvant dose: 10mg once daily).

    If 1 or fewer out of 6 patients at DL-1 develop DLT, then the recommended dose will be DL-1. If at least 2 of 6 at DL-1 develops a DLT, then an additional 6 patients will be enrolled at Dose Level -2 [DL-2] (Loading dose for 7 days: 15mg twice daily; Concurrent/adjuvant dose: 5mg once daily).

    At the recommended dose, an additional 14 patients will be enrolled for the Dose Expansion portion.

  • CSIProcedure or surgery

    Patients will receive CSI.

Study groups in NCT06724926
Study groupRoleWhat they receive
Dose Determination followed by Dose ExpansionExperimentalPatients with leptomeningeal metastasis from a solid tumor or primary CNS high-grade glioma will be enrolled and start Azeliragon 7 days prior to CSI, during CSI, and for 7 days days after completion of CSI (Dose Determination). Patients will be monitored for dose limiting toxicities during protocol therapy. Once the recommended dose is identified, additional patients will be treated with Azeliragon at recommended dose in combination with craniospinal irradiation (Dose Expansion).
Receives: Drug: Azeliragon; Procedure: CSI

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Patients with Dose-Limiting Toxicities (DLTs) at Dose Level 1

    DLTs assess using Common Terminology Criteria for Adverse Events (CTCAE) v 5.0.

    Measured: Up to Week 4

  2. Number of Patients with DLTs at Dose Level -1

    DLTs assess using CTCAE v 5.0.

    Measured: Up to Week 4

  3. Number of Patients with DLTs at Dose Level -2

    DLTs assess using CTCAE v 5.0.

    Measured: Up to Week 4

Secondary outcomes (2)

  1. Central nervous system (CNS) progression-free survival (PFS)

    CNS PFS is defined as time from randomization until CNS progression or death, whichever occurs first.

    Measured: Up to Month 13 (12 Months Post-Protocol Therapy)

  2. Overall Survival (OS)

    OS is defined as time from randomization until death.

    Measured: Up to Month 13 (12 Months Post-Protocol Therapy)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Dec 2024
  2. Study start18 Feb 2025
  3. Primary completionJan 2027 (estimated)
  4. Study completionJan 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06724926
Sponsor's protocol ID
24-00982
Status last verified
May 2026
First submitted
6 Dec 2024
Last updated
18 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The de-identified participant data from the final research dataset will be shared upon reasonable request beginning 9 to 36 months after publication or as required by a condition of awards or supporting agreements, provided the requesting investigator executes a data use agreement with NYU Langone Health. This instance of data sharing will also require separate IRB review as well as review from NYU Langone's Data Sharing Strategy Board (DSSB). Requests should be directed to: Jonathan.Yang@nyulangone.org. The protocol and statistical analysis plan will be posted on Clinicaltrials.gov only as required by federal regulation or supporting awards and agreements.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Azeliragon and CSI in people with Solid Tumor and High-grade Glioma, run by NYU Langone Health at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 18 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Patients with Dose-Limiting Toxicities (DLTs) at Dose Level 1 (time frame: Up to Week 4). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06724926.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2026.

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