Testing the Addition of an Anti-Cancer Drug, Mycophenolate Mofetil, to the Usual Treatment (Radiation Therapy and Temozolomide) for Advanced Brain Cancer

NCT07605364 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: A072402
• Study Type: interventional
• Target: 422
• Locations: 0 countries
• Sites: N/A

Brief Summary

This phase II/III trial tests how well adding mycophenolate mofetil, to the usual treatment with intensity-modulated radiation therapy and temozolomide works for the treatment of glioblastoma that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced). Mycophenolate mofetil blocks enzyme activity which can disrupt tumor activity and may make the tumor more sensitive to radiation and/or temozolomide. Intensity modulated radiation therapy is a type of 3-dimensional radiation therapy that uses computer-generated images to show the size and shape of the tumor. Thin beams of radiation of different intensities are aimed at the tumor from many angles. This type of radiation therapy reduces the damage to healthy tissue near the tumor. Temozolomide is a chemotherapy drug and in a class of medications called alkylating agents. It damages the cell's deoxyribonucleic acid (DNA) and may kill cancer cells. Giving mycophenolate mofetil with intensity-modulated radiation therapy and temozolomide may be more effective than intensity-modulated radiation therapy and temozolomide alone for the treatment of advanced glioblastoma.

Conditions

Advanced Glioblastoma

Outcome Measures

Primary Outcomes (2)

• Progression free survival (PFS) (phase II)

  PFS will be calculated from randomization until disease progression according to RANO 2.0 criteria or death due to any cause, censoring patients at their last disease evaluation. The Kaplan-Meier method will be used to estimate PFS for each arm and a stratified log-rank test will be used to compare distributions

  From randomization to the disease progression according to Response Assessment in Neuro-Oncology (RANO) 2.0 criteria or death due to any cause, up to 5 years

• Overall survival (OS) (phase III)

  OS will be calculated from randomization until death due to any cause, censoring patients at their last disease evaluation. The Kaplan-Meier method will be used to estimate OS for each arm and a stratified log-rank test will be used to compare distributions.

  From randomization until death due to any cause, up to 5 years

Secondary Outcomes (4)

• Tumor response

  Up to 5 years

• Incidence of adverse events

  Up to 5 years

• Overall survival (OS)

  From randomization until death due to any cause, up to 5 years

• Progression free survival (PFS)

  From randomization to the disease progression according to RANO 2.0 criteria or death due to any cause, up to 5 years

Study Arms (2)

Arm I (radiation, temozolomide)

EXPERIMENTAL

Patients under 70 years undergo IMRT, Monday-Friday for 30 treatments and patients greater than or equal to 70 years undergo hypofractionated radiation therapy, Monday-Friday for 15 treatments in the absence of disease progression or unacceptable toxicity. Starting 24 hours prior to radiation, patients also receive temozolomide PO QD until the day of the last radiation treatment, in the absence of disease progression or unacceptable toxicity. Patients then undergo a 4-week rest period. Patients then receive temozolomide PO QD on days 1-5 of each cycle. Cycles repeat every 28 days for 6 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo MRI and blood sample collection throughout the study.

Radiation: Intensity-Modulated Radiation Therapy; Radiation: Hypofractionated Radiation Therapy; Drug: Temozolomide (TMZ); Procedure: Magnetic Resonance Imaging; Procedure: Biospecimen Collection

Arm II (radiation, temozolomide, mycophenolate mofetil)

EXPERIMENTAL

Patients under 70 years undergo IMRT, Monday-Friday for 30 treatments and patients greater than or equal to 70 years undergo hypofractionated radiation therapy, Monday-Friday for 15 treatments in the absence of disease progression or unacceptable toxicity. Starting 24 hours prior to radiation, patients also receive temozolomide PO QD until the day of the last radiation treatment, and, starting on the first day of radiation, patients also receive mycophenolate mofetil PO twice daily (BID) in the absence of disease progression or unacceptable toxicity. Patients then undergo a 4-week rest period. Patients then receive temozolomide PO QD on days 1-5 and mycophenolate mofetil PO BID on days 1-28 of each cycle. Cycles repeat every 28 days for 6 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo MRI and blood sample collection throughout the study.

Radiation: Intensity-Modulated Radiation Therapy; Drug: Temozolomide (TMZ); Procedure: Magnetic Resonance Imaging; Procedure: Biospecimen Collection; Drug: Mycophenolate Mofetil

Interventions

Hypofractionated Radiation Therapy RADIATION

Undergo hypofractionated radiation therapy

Arm I (radiation, temozolomide)

Temozolomide (TMZ) DRUG

Given PO

Arm I (radiation, temozolomide); Arm II (radiation, temozolomide, mycophenolate mofetil)

Magnetic Resonance Imaging PROCEDURE

Undergo MRI

Arm I (radiation, temozolomide); Arm II (radiation, temozolomide, mycophenolate mofetil)

Mycophenolate Mofetil DRUG

Given PO

Arm II (radiation, temozolomide, mycophenolate mofetil)

Intensity-Modulated Radiation Therapy RADIATION

Undergo IMRT

Also known as: IMRT

Arm I (radiation, temozolomide); Arm II (radiation, temozolomide, mycophenolate mofetil)

Biospecimen Collection PROCEDURE

undergo Biospecimen Collection

Arm I (radiation, temozolomide); Arm II (radiation, temozolomide, mycophenolate mofetil)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Newly diagnosed primary glioblastoma by World Health Organization (WHO) 2021 criteria
• No spinal cord glioblastoma
• No leptomeningeal disease
• No extracranial metastatic disease
• Patient is a candidate for first-line standard of care chemoradiation per treating physician(s)
• No prior treatment for glioblastoma other than resection (i.e. prior chemotherapy, radiation therapy, or other therapies such as laser ablation are not allowed)
• Prior biopsy and/or resection of glioblastoma must be completed at least 14 days prior to registration with adequate wound healing
• Age ≥ 18 years
• Karnofsky performance status (KPS) ≥ 60
• Absolute neutrophil count (ANC) ≥ 1,500/mm\^3
• Platelet count ≥ 100,000 cells/mm\^3
• Hemoglobin (Hg) ≥ 9.0 g/dL
• Calculated (Calc.) creatinine clearance (CrCl) ≥ 25 mL/min
• \* Calculated using the Cockcroft-Gault equation
• Bilirubin ≤ 1.5 x upper limit of normal (ULN)

Sponsors & Collaborators

• Alliance for Clinical Trials in Oncology: lead
• National Cancer Institute (NCI): collaborator

MeSH Terms

Interventions

Radiotherapy, Intensity-Modulated; Temozolomide; Magnetic Resonance Spectroscopy; Mycophenolic Acid

Intervention Hierarchy (Ancestors)

Radiotherapy, Conformal; Radiotherapy, Computer-Assisted; Radiotherapy; Therapeutics; Dacarbazine; Triazenes; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Spectrum Analysis; Chemistry Techniques, Analytical; Investigative Techniques; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids

Study Officials

• Priya Kumthekar, MD

  Alliance for Clinical Trials in Oncology

  STUDY CHAIR

• Yoshie Umemura, MD, MS

  Alliance for Clinical Trials in Oncology

  STUDY CHAIR

Central Study Contacts

Amanda Clark

CONTACT

(773) 702-9171; neurooncologyprotocols@alliancenctn.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2026
• First Posted: May 26, 2026
• Study Start: June 10, 2026
• Primary Completion (Estimated): September 30, 2029
• Study Completion (Estimated): January 5, 2031
• Last Updated: May 26, 2026

Record last verified: 2026-05