AstrocytomaPhase 1NCT05236036
Mycophenolate Mofetil in Combination With Standard of Care for the Treatment of Glioblastoma
A Phase 1 study testing Mycophenolate Mofetil, Quality-of-Life Assessment and Radiation Therapy in people with Astrocytoma and Glioblastoma, run by Northwestern University at 3 sites in United States.
Trial summary
This phase I/Ib trial tests the safety, side effects, and best dose of mycophenolate mofetil in combination with temozolomide and/or radiation therapy (standard of care) in treating patients with glioblastoma. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Astrocytoma, Glioblastoma, Glioblastoma, IDH-Wildtype
- What is being tested?
- Mycophenolate Mofetil, Quality-of-Life Assessment and Radiation Therapy
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 86 participants
- Where
- 3 sitesUnited States
- Study dates
- 8 Aug 2022to 3 Jan 2029
- 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
- GROUPS 1-3: Histologically confirmed glioblastoma (GBM), IDH wild-type (by immunohistochemistry [IHC] R132H negative [neg] or sequencing). Astrocytoma with molecular features of GBM are eligible.
- GROUPS 1-3: Newly diagnosed glioblastoma and:
- Group 1: Received surgical resection or biopsy followed by chemoradiation;
- Group 2: Received surgical resection or biopsy only and have documented unmethylated glioblastoma (may have been done at an outside facility);
- Group 3: Received surgical resection or biopsy only
- GROUP S: Newly suspected glioblastoma or recurrent glioblastoma, and scheduled to undergo a standard of care surgical resection or biopsy.
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- Group R: Recurrent glioblastoma.
- Stable or decreasing dose of corticosteroids equivalent to =< 8 mg dexamethasone daily, for >= 7 days prior to registration.
- Note: There are no restrictions on steroid use on study
- Patients must be age >= 18 years.
- Patients must exhibit a Karnofsky performance status >= 70.
- Leukocytes (white blood cells [WBC]) >= 3,000/mcL (within 14 days prior to study registration)
- Absolute neutrophil count (ANC) >= 1,500/mcL (within 14 days prior to study registration)
- Hemoglobin (Hgb) >= 8 g/dL (within 14 days prior to study registration) (transfusion may be used for eligibility if >= 7 days)
- Platelets (PLT) >= 100,000/mcL (within 14 days prior to study registration) (transfusion or growth factor may be used for eligibility if >= 7 days).
- Total bilirubin =< 2x institutional upper limit of normal (ULN) (within 14 days prior to study registration)
- Aspartate aminotransferase (AST)(serum glutamic-oxaloacetic transaminase [SGOT])/Alanine aminotransferase (ALT)(serum glutamate pyruvate transaminase [SGPT]) =< 3 x institutional ULN (within 14 days prior to study registration)
- Creatinine =< 1.5 x Institutional ULN (within 14 days prior to study registration)
- International normalized ratio (INR) =< 1.5 x ULN (within 14 days prior to study registration)
- Prothrombin time (PT)/Partial thromboplastin Time (PTT) =< 1.5 x ULN (within 14 days prior to study registration)
- Females of child-bearing potential (FOCBP) must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) from time of informed consent, for the duration of study participation, and for 3 months following completion of therapy. Should a female patient become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception from time of informed consent, for the duration of study participation, and 4 months after completion of administration.
- NOTE: A FOCBP is any woman (regardless of sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
- Has not undergone a hysterectomy or bilateral oophorectomy
- Has had menses at any time in the preceding 12 consecutive months (and therefore has not been naturally postmenopausal for > 12 months)
- FOCBP must have a negative pregnancy test within 14 days prior to registration on study.
- Patient or their legally authorized representative must provide written, signed, and dated informed consent prior to study registration. Patient or their legally authorized representative (LAR) must have the ability to understand and the willingness to sign a written informed consent document. The patient or their LAR must be willing and able to comply with the protocol for the duration of the study.
- NOTE: no study-specific screening procedures may be performed until written consent has been obtained.
Who cannot join
- Patients who are receiving any other investigational agents.
- Exception: COVID-19 vaccine and treatment is allowed
- Patient who have a prior or concurrent malignancy that may interfere with study treatment or safety.
- NOTE: Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible. Per principal investigator (PI) discretion
- Patients who have a history of allergic reactions attributed to compounds of similar chemical composition to temozolomide or mycophenolate mofetil.
- Patients with spinal cord and diffuse leptomeningeal disease GBM
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- Patients requiring live vaccinations within 2 weeks of initiation of MMF and/or TMZ therapy. Consider completion of vaccination with live vaccines prior to starting immunosuppressive therapy, as indicated.
- Patients on viral-vector based therapy due to increased risk for disseminated herpetic infection.
- Patients who have an uncontrolled intercurrent illness including, but not limited to any of the following, are not eligible:
- Have uncontrolled epilepsy
- Have an uncontrolled intercurrent illness
- Concurrent malignancy (outside of glioblastoma) that requires tumor directed treatment
- Known deficiency of hypoxanthine-guanin-phosphoribosyltransferase (HGPRT) deficiency, e.g. Lesch-Nyhan- oder Kelley-Seegmiller-Syndrome.
- Known concurrent shingles, herpes, CMV (cytomegalovirus) infection
- Known concurrent opportunistic fungal infection
- Known concurrent or history of unexplained opportunistic infection
- Known immunodeficiency that could lead to opportunistic infections
- Psychiatric illness/social situations that would limit compliance with study requirements. Any other illness or condition that the treating investigator feels would interfere with study compliance or would compromise the patient's safety or study endpoints.
- Female patients who are pregnant or nursing. Pregnant women are excluded from this study because temozolomide is an alkylating agent with potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with temozolomide, breastfeeding should be discontinued if the mother is treated with temozolomide.
- Patients who are unable to swallow oral medication or have problems/ diseases that affect absorption of oral medication.
- Patients with a known history of human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV).
- Note: Temozolomide and mycophenolate mofetil are immunosuppressive agents. Patients with a known history of HIV, HBV, and HCV, and unexplained opportunistic infections are not eligible due to safety reasons.
- Patients who require treatment with a therapy that is categorized as a "prohibited therapy" as listed in Section 4.10 of the protocol and who are unable to safety discontinue the prohibited therapy prior to the first dose of MMF or TMZ.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Northwestern University
Chicago, Illinois, 60611
Northwestern Lake Forest Hospital
Lake Forest, Illinois, 60045
Northwestern Medicine Warrenville
Warrenville, Illinois, 60555
About this study
Official title: A Phase 1/1b Adaptive Dose Escalation Study of Mycophenolate Mofetil (MMF) in Combination With Standard of Care for Patients With Glioblastoma
This phase I/Ib trial tests the safety, side effects, and best dose of mycophenolate mofetil in combination with temozolomide and/or radiation therapy (standard of care) in treating patients with glioblastoma. Mycophenolate mofetil is an immunosuppressant drug that is typically used to prevent organ rejection in transplant recipients. However, mycophenolate mofetil may also help chemotherapy with temozolomide work better by making tumor cells more sensitive to the drug. The purpose of this trial is to determine if mycophenolate mofetil combined with temozolomide can stop glioblastoma.
Read the detailed description
PRIMARY OBJECTIVE:
- To evaluate the recommended phase 2 dose (RP2D) for mycophenolate mofetil (MMF) in combination with radiation therapy as well as in combination with temozolomide (TMZ).
II. Phase 1b (Groups 3 and R): To estimate median and corresponding 95% CI for progression-free survival (PFS) and overall survival (OS).
III. Phase 0 (Group S; Sub-Group S1 and Sub-Group S2): To evaluate drug uptake and target effect in the brain.
SECONDARY OBJECTIVES:
- To evaluate the safety profile of mycophenolate mofetil (MMF) in combination with temozolomide (TMZ).
II. To estimate progression free survival (PFS) per Response Assessment in Neuro-Oncology Criteria (RANO).
III. To estimate overall survival (OS). IV. To estimate the overall response rate (ORR) per RANO criteria. V. To evaluate quality of life per Functional Assessment of Cancer Therapy Scale-Brain (FACT-Br) for patients treated with mycophenolate mofetil and/or temozolomide.
EXPLORATORY OBJECTIVES:
- To investigate the relationship between the molecular signature of individual glioblastoma multiforme (GBM) with clinical outcome, by measuring levels of serum mycophenolic acid in patient's plasma post MMF administration.
Ia. Perform molecular characterization of all GBM tissues by ribonucleic acid sequencing (RNAseq) analysis.
Ib. Perform bulk metabolomics for GBM tissue. Ic. Measure plasma and serum concentration of mycophenolic acid, the MMF's primary active metabolite, during and after combination therapy.
Id. Measure IMPDH activity assay in patients' peripheral blood mononuclear cells (PBMCs) as well as in GBM tissue.
Ie. Analyze relevant gene mutations from standard of care next generation sequencing (NGS) tumor gene mutation data, if available. II. Group R only: To assess early efficacy of mycophenolate mofetil (MMF) in combination with temozolomide (TMZ) in recurrent GBM.
OUTLINE: This is a dose-escalation study of MMF (Part 1), followed by a dose-expansion study (Part 2).
PART 1: Patients are assigned to 1 of 3 groups.
GROUP 1: Patients who have already undergone surgery or biopsy followed by chemoradiation receive TMZ orally (PO) once daily (QD) on days 1-5 of each cycle and MMF PO twice daily (BID). Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.
GROUP 2: Patients with unmethylated glioblastoma who have already undergone surgery or biopsy receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Starting at the same time as TMZ and MMF administration, patients also receive radiation therapy daily, 5 days per week, for 6 weeks.
GROUP S: Patients planning to undergo surgery receive MMF PO BID and TMZ PO QD for 5 days prior to surgery in the absence of disease progression or unacceptable toxicity.
PART 2: Patients are assigned to Group 3 or enrolled to Group R.
GROUP 3: Patients who have already undergone surgery or biopsy receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Starting at the same time as TMZ and MMF administration, patients also receive radiation therapy daily, 5 days per week, for 6 weeks.
After completion of study treatment, patients are followed up at 30 days and then every 3 months for up to 18 months.
Group R (Recurrent): MMF + TMZ at MTD. Enrollment to begin concurrently with Group 3.Group R will be given the MTD for temozolomide + MMF via the same schedule and dosing as the adjuvant group (Group 1) and will open once the RP2D from Group 1 has been determined. Patients receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.
- Conditions
- Astrocytoma, Glioblastoma, Glioblastoma, IDH-Wildtype, MGMT-Unmethylated Glioblastoma, Recurrent Glioblastoma
- Being tested
- Mycophenolate Mofetil, Quality-of-Life Assessment, Radiation Therapy, Temozolomide
- Medical subject headings
- Astrocytoma, Glioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called CellCept, MMF
Given PO
Quality-of-Life AssessmentOther
Also called Quality of Life Assessment
Ancillary studies
Radiation TherapyRadiation
Also called Cancer Radiotherapy, ENERGY_TYPE, Irradiate, Irradiated
Receive radiation therapy
TemozolomideDrug
Also called CCRG-81045, Imidazo[5,1-d]-1,2,3,5-tetrazine-8-carboxamide, 3, 4-dihydro-3-methyl-4-oxo-, M & B 39831, M and B 39831
Given Orally (PO)
| Study group | Role | What they receive |
|---|---|---|
| Group 1 (TMZ, MMF) | Experimental | Patients who have already undergone surgery or biopsy followed by chemoradiation receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Mycophenolate Mofetil; Other: Quality-of-Life Assessment; Drug: Temozolomide |
| Group 2 (TMZ, MMF, radiation therapy) | Experimental | Patients with unmethylated glioblastoma who have already undergone surgery or biopsy receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Starting at the same time as TMZ and MMF administration, patients also receive radiation therapy daily, 5 days per week, for 6 weeks. Receives: Drug: Mycophenolate Mofetil; Other: Quality-of-Life Assessment; Radiation: Radiation Therapy; Drug: Temozolomide |
| Group 3 (TMZ, MMF, radiation therapy) | Experimental | Patients who have already undergone surgery or biopsy receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Starting at the same time as TMZ and MMF administration, patients also receive radiation therapy daily, 5 days per week, for 6 weeks. Receives: Drug: Mycophenolate Mofetil; Other: Quality-of-Life Assessment; Radiation: Radiation Therapy; Drug: Temozolomide |
| Group S (pre-surgical MMF, TMZ) | Experimental | Patients planning to undergo surgery receive MMF PO BID and TMZ PO QD for 5 days prior to surgery in the absence of disease progression or unacceptable toxicity. Receives: Drug: Mycophenolate Mofetil; Other: Quality-of-Life Assessment; Drug: Temozolomide |
| Group R (TMZ, MMF) | Experimental | Group R (Recurrent): MMF + TMZ at MTD. Patients receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Mycophenolate Mofetil; Other: Quality-of-Life Assessment; Drug: Temozolomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose/recommended phase 2 dose (MTD/RP2D) for mycophenolate mofetil (MMF) (Group 1)
Will be based on treatment-emergent and drug related toxicity of grade >= 3 during the first cycle of treatment. MTD indicates maximum tolerated dose for dose limiting toxicity (DLT). DLT is defined as side effects of a drug or other treatment that are serious enough to prevent an increase in dose or level of that treatment. Recommended phase 2 dose (RP2D) is the highest dose of a drug or treatment that does not cause unacceptable side effects. For the purposes of this study, RP2D is interchangeable with MTD. DLTs and Adverse Events (AEs) will be assessed by Common Terminology Criteria for Adverse Events (CTCAE) version (v). 5.0.
Measured: Up to completion of first cycle of treatment + 7 days (1 cycle = 28 days)
MTD/RP2D for MMF (Group 2)
Will be based on treatment-emergent and drug related toxicity of grade >= 3 during focal radiation treatment. MTD indicates maximum tolerated dose for DLT. DLT is defined as side effects of a drug or other treatment that are serious enough to prevent an increase in dose or level of that treatment. RP2D is the highest dose of a drug or treatment that does not cause unacceptable side effects. For the purposes of this study, RP2D is interchangeable with MTD. DLTs and AEs will be assessed by CTCAE v.5.0.
Measured: Up to 6 weeks + 7 days
Secondary outcomes (5)
Frequency of adverse events
The occurrence of toxicity (including [AEs] and serious adverse events [SAEs]), and the number of patients who discontinue treatment due to toxicity, as assessed by CTCAE v. 5.0. This endpoint will collect and report the frequency of adverse events by type, severity (grade), timing, and attribution to MMF, according the National Cancer Institute (NCI)-CTCAE v. 5.0.
Measured: Up to 30 days after completion of study treatment
Progression Free Survival (PFS)
Progression will be determined by clinical progression or by radiographic imaging as assessed by Response Assessment in Neuro-Oncology Criteria (RANO). If disease progression or death from any cause is not observed prior to initiating subsequent anti-cancer therapy or completing study participation, the PFS will be censored as the last available disease assessment. Kaplan-Meier curves will be plot. Log-rank test will be used to compare it with historical standard care control in literature.
Measured: From baseline until the patient experiences disease progression, initiates subsequent anti-cancer therapy, completes study participation, or experiences death from any cause (whichever is sooner), assessed up to 18 months
Overall survival (OS)
If death from any cause is not observed prior to initiating subsequent anti-cancer therapy or completing study participation, the OS will be censored as the last available follow-up. will be described by Kaplan-Meier curve. The median and corresponding 95% confidence interval will also be estimated.
Measured: From time of diagnosis until the patient initiates subsequent anti-cancer therapy, completes study participation, or experiences death from any cause (whichever is sooner), assessed up to 18 months
Overall response rate (ORR)
Overall response rate will be assessed by RANO criteria. To determine the ORR, this endpoint will calculate the proportion of treated patients who experience an overall response (complete response [CR] or partial response [PR] per RANO criteria). The date of first response for either CR or PR will be used for the calculation of ORR. Will be estimated using the expansion cohort, along with its 95% confidence interval.
Measured: From baseline, the patient experiences disease progression, the patient initiates subsequent anti-cancer therapy, or the patient completes study participation (whichever occurs first), assessed up to 18 months
Quality of life (QOL)
The quality of life indicated by FACT-Br will be evaluated by estimating its mean, standard deviation and 95% confidence interval using the expansion cohort. Patients who received at least 1 dose of temozolomide (TMZ) and/or MMF, and completed at least baseline and cycle 2 day 1 (C2D1) FACT-Br QOL (for Groups 1-3) will be evaluable for this secondary endpoint.
Measured: Up to 6 cycles (1 cycle = 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2022
- Study start8 Aug 2022
- Primary completion3 Jan 2029 (estimated)
- Study completion3 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AstrocytomaStudies of the same condition, open and under way.
- More trials run by Northwestern UniversityThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 86 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Priya U Kumthekar, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05236036
- Sponsor's protocol ID
- NU 21C01
- Other IDs
- NCI-2021-12424, STU00215766, NU 21C01, P30CA060553
- Status last verified
- Aug 2026
- First submitted
- 2 Feb 2022
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/1b Adaptive Dose Escalation Study of Mycophenolate Mofetil (MMF) in Combination With Standard of Care for Patients With Glioblastoma
- ClinicalTrials.gov ID
- NCT05236036
- Acronym / sponsor ID
- NU 21C01
- Conditions
- Astrocytoma, Glioblastoma, Glioblastoma, IDH-Wildtype, MGMT-Unmethylated Glioblastoma, Recurrent Glioblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 86 participants (actual)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 8 Aug 2022 (actual)
- Primary completion
- 3 Jan 2029 (estimated)
- Study completion
- 3 Jan 2029 (estimated)
- First posted
- 11 Feb 2022 (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 phase I/Ib trial tests the safety, side effects, and best dose of mycophenolate mofetil in combination with temozolomide and/or radiation therapy (standard of care) in treating patients with glioblastoma. Mycophenolate mofetil is an immunosuppressant drug that is typically used to prevent organ rejection in transplant recipients. However, mycophenolate mofetil may also help chemotherapy with temozolomide work better by making tumor cells more sensitive to the drug. The purpose of this trial is to determine if mycophenolate mofetil combined with temozolomide can stop glioblastoma.
Detailed description
PRIMARY OBJECTIVE:
- To evaluate the recommended phase 2 dose (RP2D) for mycophenolate mofetil (MMF) in combination with radiation therapy as well as in combination with temozolomide (TMZ).
II. Phase 1b (Groups 3 and R): To estimate median and corresponding 95% CI for progression-free survival (PFS) and overall survival (OS).
III. Phase 0 (Group S; Sub-Group S1 and Sub-Group S2): To evaluate drug uptake and target effect in the brain.
SECONDARY OBJECTIVES:
- To evaluate the safety profile of mycophenolate mofetil (MMF) in combination with temozolomide (TMZ).
II. To estimate progression free survival (PFS) per Response Assessment in Neuro-Oncology Criteria (RANO).
III. To estimate overall survival (OS). IV. To estimate the overall response rate (ORR) per RANO criteria. V. To evaluate quality of life per Functional Assessment of Cancer Therapy Scale-Brain (FACT-Br) for patients treated with mycophenolate mofetil and/or temozolomide.
EXPLORATORY OBJECTIVES:
- To investigate the relationship between the molecular signature of individual glioblastoma multiforme (GBM) with clinical outcome, by measuring levels of serum mycophenolic acid in patient's plasma post MMF administration.
Ia. Perform molecular characterization of all GBM tissues by ribonucleic acid sequencing (RNAseq) analysis.
Ib. Perform bulk metabolomics for GBM tissue. Ic. Measure plasma and serum concentration of mycophenolic acid, the MMF's primary active metabolite, during and after combination therapy.
Id. Measure IMPDH activity assay in patients' peripheral blood mononuclear cells (PBMCs) as well as in GBM tissue.
Ie. Analyze relevant gene mutations from standard of care next generation sequencing (NGS) tumor gene mutation data, if available. II. Group R only: To assess early efficacy of mycophenolate mofetil (MMF) in combination with temozolomide (TMZ) in recurrent GBM.
OUTLINE: This is a dose-escalation study of MMF (Part 1), followed by a dose-expansion study (Part 2).
PART 1: Patients are assigned to 1 of 3 groups.
GROUP 1: Patients who have already undergone surgery or biopsy followed by chemoradiation receive TMZ orally (PO) once daily (QD) on days 1-5 of each cycle and MMF PO twice daily (BID). Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.
GROUP 2: Patients with unmethylated glioblastoma who have already undergone surgery or biopsy receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Starting at the same time as TMZ and MMF administration, patients also receive radiation therapy daily, 5 days per week, for 6 weeks.
GROUP S: Patients planning to undergo surgery receive MMF PO BID and TMZ PO QD for 5 days prior to surgery in the absence of disease progression or unacceptable toxicity.
PART 2: Patients are assigned to Group 3 or enrolled to Group R.
GROUP 3: Patients who have already undergone surgery or biopsy receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Starting at the same time as TMZ and MMF administration, patients also receive radiation therapy daily, 5 days per week, for 6 weeks.
After completion of study treatment, patients are followed up at 30 days and then every 3 months for up to 18 months.
Group R (Recurrent): MMF + TMZ at MTD. Enrollment to begin concurrently with Group 3.Group R will be given the MTD for temozolomide + MMF via the same schedule and dosing as the adjuvant group (Group 1) and will open once the RP2D from Group 1 has been determined. Patients receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Group 1 (TMZ, MMF) | Experimental | Drug: Mycophenolate Mofetil, Other: Quality-of-Life Assessment, Drug: TemozolomidePatients who have already undergone surgery or biopsy followed by chemoradiation receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. |
| Group 2 (TMZ, MMF, radiation therapy) | Experimental | Drug: Mycophenolate Mofetil, Other: Quality-of-Life Assessment, Radiation: Radiation Therapy, Drug: TemozolomidePatients with unmethylated glioblastoma who have already undergone surgery or biopsy receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Starting at the same time as TMZ and MMF administration, patients also receive radiation therapy daily, 5 days per week, for 6 weeks. |
| Group 3 (TMZ, MMF, radiation therapy) | Experimental | Drug: Mycophenolate Mofetil, Other: Quality-of-Life Assessment, Radiation: Radiation Therapy, Drug: TemozolomidePatients who have already undergone surgery or biopsy receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Starting at the same time as TMZ and MMF administration, patients also receive radiation therapy daily, 5 days per week, for 6 weeks. |
| Group S (pre-surgical MMF, TMZ) | Experimental | Drug: Mycophenolate Mofetil, Other: Quality-of-Life Assessment, Drug: TemozolomidePatients planning to undergo surgery receive MMF PO BID and TMZ PO QD for 5 days prior to surgery in the absence of disease progression or unacceptable toxicity. |
| Group R (TMZ, MMF) | Experimental | Drug: Mycophenolate Mofetil, Other: Quality-of-Life Assessment, Drug: TemozolomideGroup R (Recurrent): MMF + TMZ at MTD. Patients receive TMZ PO QD on days 1-5 of each cycle and MMF PO BID. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (27 listed)
- GROUPS 1-3: Histologically confirmed glioblastoma (GBM), IDH wild-type (by immunohistochemistry [IHC] R132H negative [neg] or sequencing). Astrocytoma with molecular features of GBM are eligible.
- GROUPS 1-3: Newly diagnosed glioblastoma and:
- GROUP S: Newly suspected glioblastoma or recurrent glioblastoma, and scheduled to undergo a standard of care surgical resection or biopsy.
- Group R: Recurrent glioblastoma.
- Stable or decreasing dose of corticosteroids equivalent to =< 8 mg dexamethasone daily, for >= 7 days prior to registration.
Key exclusion criteria (23 listed)
- Patients who are receiving any other investigational agents.
- Patient who have a prior or concurrent malignancy that may interfere with study treatment or safety.
- Patients who have a history of allergic reactions attributed to compounds of similar chemical composition to temozolomide or mycophenolate mofetil.
- Patients with spinal cord and diffuse leptomeningeal disease GBM
- Patients requiring live vaccinations within 2 weeks of initiation of MMF and/or TMZ therapy. Consider completion of vaccination with live vaccines prior to starting immunosuppressive therapy, as indicated.
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 |
|---|---|---|
| Mycophenolate MofetilAlso: CellCept, MMFGiven PO | Drug | Group 1 (TMZ, MMF), Group 2 (TMZ, MMF, radiation therapy), Group 3 (TMZ, MMF, radiation therapy), Group R (TMZ, MMF), Group S (pre-surgical MMF, TMZ) |
| Quality-of-Life AssessmentAlso: Quality of Life AssessmentAncillary studies | Other | Group 1 (TMZ, MMF), Group 2 (TMZ, MMF, radiation therapy), Group 3 (TMZ, MMF, radiation therapy), Group R (TMZ, MMF), Group S (pre-surgical MMF, TMZ) |
| Radiation TherapyAlso: Cancer Radiotherapy, ENERGY_TYPE, Irradiate, IrradiatedReceive radiation therapy | Radiation | Group 2 (TMZ, MMF, radiation therapy), Group 3 (TMZ, MMF, radiation therapy) |
| TemozolomideAlso: CCRG-81045, Imidazo[5,1-d]-1,2,3,5-tetrazine-8-carboxamide, 3, 4-dihydro-3-methyl-4-oxo-, M & B 39831, M and B 39831Given Orally (PO) | Drug | Group 1 (TMZ, MMF), Group 2 (TMZ, MMF, radiation therapy), Group 3 (TMZ, MMF, radiation therapy), Group R (TMZ, MMF), Group S (pre-surgical MMF, TMZ) |
Primary outcome
Maximum tolerated dose/recommended phase 2 dose (MTD/RP2D) for mycophenolate mofetil (MMF) (Group 1)
Time frame: Up to completion of first cycle of treatment + 7 days (1 cycle = 28 days)
Will be based on treatment-emergent and drug related toxicity of grade >= 3 during the first cycle of treatment. MTD indicates maximum tolerated dose for dose limiting toxicity (DLT). DLT is defined as side effects of a drug or other treatment that are serious enough to prevent an increase in dose or level of that treatment. Recommended phase 2 dose (RP2D) is the highest dose of a drug or treatment that does not cause unacceptable side effects. For the purposes of this study, RP2D is interchangeable with MTD. DLTs and Adverse Events (AEs) will be assessed by Common Terminology Criteria for Adverse Events (CTCAE) version (v). 5.0.
MTD/RP2D for MMF (Group 2)
Time frame: Up to 6 weeks + 7 days
Will be based on treatment-emergent and drug related toxicity of grade >= 3 during focal radiation treatment. MTD indicates maximum tolerated dose for DLT. DLT is defined as side effects of a drug or other treatment that are serious enough to prevent an increase in dose or level of that treatment. RP2D is the highest dose of a drug or treatment that does not cause unacceptable side effects. For the purposes of this study, RP2D is interchangeable with MTD. DLTs and AEs will be assessed by CTCAE v.5.0.
5 secondary outcomes
Frequency of adverse events
Time frame: Up to 30 days after completion of study treatment
Progression Free Survival (PFS)
Time frame: From baseline until the patient experiences disease progression, initiates subsequent anti-cancer therapy, completes study participation, or experiences death from any cause (whichever is sooner), assessed up to 18 months
Overall survival (OS)
Time frame: From time of diagnosis until the patient initiates subsequent anti-cancer therapy, completes study participation, or experiences death from any cause (whichever is sooner), assessed up to 18 months
Overall response rate (ORR)
Time frame: From baseline, the patient experiences disease progression, the patient initiates subsequent anti-cancer therapy, or the patient completes study participation (whichever occurs first), assessed up to 18 months
Quality of life (QOL)
Time frame: Up to 6 cycles (1 cycle = 28 days)
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 26 Aug 2026.
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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.
Priya U Kumthekar, MD principal investigator
Northwestern University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Mycophenolate Mofetil, Quality-of-Life Assessment and Radiation Therapy in people with Astrocytoma and Glioblastoma, run by Northwestern University at 3 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 27 inclusion and 23 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 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 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?
Maximum tolerated dose/recommended phase 2 dose (MTD/RP2D) for mycophenolate mofetil (MMF) (Group 1) (time frame: Up to completion of first cycle of treatment + 7 days (1 cycle = 28 days)). The study lists 2 primary outcomes in all. 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 26 Aug 2026.