GlioblastomaPhase 2NCT05986851
Azeliragon in MGMT Unmethylated Glioblastoma
A Phase 2 study testing Azeliragon in people with Glioblastoma, run by Cantex Pharmaceuticals at 8 sites in United States.
Trial summary
This is a phase 2 study to evaluate the safety and preliminary evidence of effectiveness of azeliragon, in combination with radiation therapy, as an initial treatment of a form of glioblastoma. Glioblastoma is a type of brain cancer that grows quickly and can invade and destroy healthy tissue. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glioblastoma
- What is being tested?
- Azeliragon
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 8 sitesUnited States
- Study dates
- 12 Sep 2023to 30 Dec 2026
- Phase
- Phase 2
- 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
- 1. Histopathologically proven diagnosis of IDH-wildtype glioblastoma (GBM, WHO grade 4) according to the 2021 WHO classification (including subtypes such as gliosarcoma).
- 2. Diagnosis must be established by open biopsy or tumor resection. Patients who have only had a stereotactic biopsy are not eligible.
- 3. Supratentorial location. 4. MGMT promoter methylation is negative based on local CLIA-certified commercial laboratory tests.
- 5. Must have recovered from the effects of surgery, postoperative infection, and other complications at the time the patient signs the informed consent and is determined to be eligible to participate in the study, as deemed eligible to participate per PI and sub-investigator.
- 6. ≥ 18 years old. 7. Karnofsky performance status ≥ 60. 8. A diagnostic contrast-enhanced MRI or CT scan (if MRI is not available) of the brain must be performed preoperatively and postoperatively. The postoperative scan must be done within 21 days of the signing of informed consent prior to the initiation of radiotherapy. Preoperative and postoperative scans must be the same type. If CT scans were performed perioperatively, a CT should be performed before the signing of the informed consent.
- 9. Study therapy must begin ≤ 7 weeks after the most recent brain tumor surgery.
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- 10. Adequate organ and bone marrow function as defined below:
- Absolute neutrophil count (ANC) ≥ 1,000 cells/mm3;
- Untransfused platelet count ≥ 75,000 cells/mm3;
- Hemoglobin > 9.0 g/dL (Note: the use of transfusion or other intervention to achieve Hgb >9.0 g/dL is acceptable);
- Total bilirubin ≤ 1.5 ULN
- AST (SGOT) and ALT (SGPT) ≤ 3x ULN 11. • Creatinine ≤ 1.5 ULN or creatinine clearance ≥ 60 mL/min using the CKD- EPI Creatinine Equation
- If there is history of human immunodeficiency virus (HIV) infection, patients must be on effective antiretroviral therapy, and HIV viral load must be undetectable within 6 months of study enrollment.
- If there is history of chronic hepatitis B virus (HBV) infection, patients must have either been treated or are on suppressive therapy (as indicated), and HBV viral load must be undetectable.
- If there is history of hepatitis C virus (HCV) infection, patients must have been treated, and HCV viral load must be undetectable.
- 12. Females of childbearing potential (defined as a female who is non-menopausal or surgically sterilized) must be willing to use an acceptable method of birth control (i.e., hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide, or abstinence) for the duration of the study. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately 13. 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).
Who cannot join
- Prior invasive malignancy (except for non-melanomatous skin cancer) unless disease free or not requiring active therapy for ≥ 3 years. (For example, carcinoma in situ of the breast, oral cavity, and cervix are all permissible).
- Prior cranial RT or RT to the head and neck where potential field overlap may exist.
- Prior use of carmustine (Gliadel) wafers or any other intratumoral or intracavitary treatment.
- Recurrent or multicentric disease. Multicentric disease is defined as multiple discrete areas of tumor without connecting T2 signal abnormality.
- Infratentorial disease or metastatic disease beyond the brain.
- Known IDH mutation. IDH status could be determined by either immunohistochemistry or sequencing as evaluated per routine clinical care.
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- Patients with a serious active infection (such as a wound infection requiring parenteral antibiotics) at the time of study entry or other serious underlying medical conditions that would impair the ability of the patient to receive protocol treatment
- Patients with any condition (e.g., psychological, geographical, etc.) that does not permit compliance with the protocol.
- Patients receiving CYP 2C8 inhibitors noted in Section 6.3
- 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
- Pregnant or breast feeding. Women of childbearing potential must a negative pregnancy test within 14 days of study entry. 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
- Patients with concurrent participation in another interventional clinical trial or use of another investigational agent within 30 days prior to study entry. 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
- -
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
Miami Cancer Institute - Baptist Health
Miami, Florida, 33176
Lenox Hill Hospital
New York, New York, 10021
The University of Oklahoma Health Science Center
Oklahoma City, Oklahoma, 73104
Corewell Health
Royal Oak, Michigan, 48073
University of Utah Health Huntsman Cancer Center
Salt Lake City, Utah, 84112
University of Washington
Seattle, Washington, 98109
Washington University in St. Louis
St Louis, Missouri, 63110
About this study
Official title: A Phase II Study to Assess Safety and Preliminary Evidence of a Therapeutic Effect of Azeliragon in Patients With MGMT Unmethylated Glioblastoma
This is a phase 2 study to evaluate the safety and preliminary evidence of effectiveness of azeliragon, in combination with radiation therapy, as an initial treatment of a form of glioblastoma. Glioblastoma is a type of brain cancer that grows quickly and can invade and destroy healthy tissue. There's no cure for glioblastoma, which is also known as glioblastoma multiforme. Treatments, including surgery, radiation, and chemotherapy might slow cancer growth and reduce symptoms. New treatments of glioblastoma are needed.
Read the detailed description
The study to be conducted is a phase 2 study in newly diagnosed "unmethylated" glioblastoma.
As compared to "methylated" glioblastoma "unmethylated" glioblastoma carries a worse prognosis, as it is resistant to temozolomide, the most commonly prescribed chemotherapeutic treatment. Based upon pre-clinical evidence suggesting that azeliragon may enhance the effectiveness of radiation, as well as have a delaying effect on disease progression, this study will combine daily oral azeliragon with radiation treatment, followed by continued administration of azeliragon after completion of radiation therapy.
Azeliragon is administered once daily as an oral capsule. In previous studies in patients with Alzheimer's disease and in normal volunteers, azeliragon was well tolerated for up to 18 months.
- Conditions
- Glioblastoma
- Being tested
- Azeliragon
- Keywords
- Unmethylated
- Medical subject headings
- Glioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzeliragonDrug
Oral capsule
| Study group | Role | What they receive |
|---|---|---|
| Daily oral azeliragon | Experimental | Azeliragon to be administered once daily for several days before, during, and after radiation therapy. Receives: Drug: Azeliragon |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression-free survival
Progression-free survival (PFS) of newly diagnosed unmethylated GBM treated with the combination of RT and azeliragon.
Measured: Up to 2 years
Secondary outcomes (3)
Overall survival
Overall survival (OS) of newly diagnosed unmethylated GBM after RT and azeliragon
Measured: Up to 2 years
Response rate
To evaluate overall response rate and duration of response as per RANO criteria
Measured: Up to 2 years
Steroid requirement
Change in requirement of dexamethasone after RT
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2023
- Study start12 Sep 2023
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GlioblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cantex Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Medpace, Inc.
- Responsible party
- Sponsor
- Investigators
- Stephen G Marcus, MD, Study chair, Cantex Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05986851
- Sponsor's protocol ID
- CAN-401
- Status last verified
- Mar 2026
- First submitted
- 3 Aug 2023
- Last updated
- 31 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study to Assess Safety and Preliminary Evidence of a Therapeutic Effect of Azeliragon in Patients With MGMT Unmethylated Glioblastoma
- ClinicalTrials.gov ID
- NCT05986851
- Acronym / sponsor ID
- CAN-401
- Conditions
- Glioblastoma
- Keywords
- Unmethylated
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Cantex PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Medpace, Inc.
- Study start
- 12 Sep 2023 (actual)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Dec 2026 (estimated)
- First posted
- 14 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 2 study to evaluate the safety and preliminary evidence of effectiveness of azeliragon, in combination with radiation therapy, as an initial treatment of a form of glioblastoma. Glioblastoma is a type of brain cancer that grows quickly and can invade and destroy healthy tissue. There's no cure for glioblastoma, which is also known as glioblastoma multiforme. Treatments, including surgery, radiation, and chemotherapy might slow cancer growth and reduce symptoms. New treatments of glioblastoma are needed.
Detailed description
The study to be conducted is a phase 2 study in newly diagnosed "unmethylated" glioblastoma.
As compared to "methylated" glioblastoma "unmethylated" glioblastoma carries a worse prognosis, as it is resistant to temozolomide, the most commonly prescribed chemotherapeutic treatment. Based upon pre-clinical evidence suggesting that azeliragon may enhance the effectiveness of radiation, as well as have a delaying effect on disease progression, this study will combine daily oral azeliragon with radiation treatment, followed by continued administration of azeliragon after completion of radiation therapy.
Azeliragon is administered once daily as an oral capsule. In previous studies in patients with Alzheimer's disease and in normal volunteers, azeliragon was well tolerated for up to 18 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Daily oral azeliragon | Experimental | Drug: AzeliragonAzeliragon to be administered once daily for several days before, during, and after radiation therapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 1. Histopathologically proven diagnosis of IDH-wildtype glioblastoma (GBM, WHO grade 4) according to the 2021 WHO classification (including subtypes such as gliosarcoma).
- 2. Diagnosis must be established by open biopsy or tumor resection. Patients who have only had a stereotactic biopsy are not eligible.
- 3. Supratentorial location. 4. MGMT promoter methylation is negative based on local CLIA-certified commercial laboratory tests.
- 5. Must have recovered from the effects of surgery, postoperative infection, and other complications at the time the patient signs the informed consent and is determined to be eligible to participate in the study, as deemed eligible to participate per PI and sub-investigator.
- 6. ≥ 18 years old. 7. Karnofsky performance status ≥ 60. 8. A diagnostic contrast-enhanced MRI or CT scan (if MRI is not available) of the brain must be performed preoperatively and postoperatively. The postoperative scan must be done within 21 days of the signing of informed consent prior to the initiation of radiotherapy. Preoperative and postoperative scans must be the same type. If CT scans were performed perioperatively, a CT should be performed before the signing of the informed consent.
Key exclusion criteria (14 listed)
- Prior invasive malignancy (except for non-melanomatous skin cancer) unless disease free or not requiring active therapy for ≥ 3 years. (For example, carcinoma in situ of the breast, oral cavity, and cervix are all permissible).
- Prior cranial RT or RT to the head and neck where potential field overlap may exist.
- Prior use of carmustine (Gliadel) wafers or any other intratumoral or intracavitary treatment.
- Recurrent or multicentric disease. Multicentric disease is defined as multiple discrete areas of tumor without connecting T2 signal abnormality.
- Infratentorial disease or metastatic disease beyond the brain.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AzeliragonOral capsule | Drug | Daily oral azeliragon |
Primary outcome
Progression-free survival
Time frame: Up to 2 years
Progression-free survival (PFS) of newly diagnosed unmethylated GBM treated with the combination of RT and azeliragon.
3 secondary outcomes
Overall survival
Time frame: Up to 2 years
Response rate
Time frame: Up to 2 years
Steroid requirement
Time frame: Up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2026.
- Individual participant dataSharing planned: No
Related trials for Glioblastoma
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Testing the Addition of an Anti-cancer Drug, Selinexor, to the Usual Chemotherapy Treatment (Temozolomide) for Brain Tumors That Have Returned After Previous Treatment
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Oral Gallium Maltolate for the Treatment of Relapsed and Refractory Glioblastoma
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stephen G Marcus, MD study chair
Cantex Pharmaceuticals
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Azeliragon in people with Glioblastoma, run by Cantex Pharmaceuticals at 8 sites 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 16 inclusion and 14 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 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Progression-free survival (time frame: Up to 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Mar 2026.