Recurrent GlioblastomaPhase 1NCT04477200

Mycophenolate Mofetil Combined With Radiation Therapy in Glioblastoma

A Phase 1 study testing Mycophenolate Mofetil, Radiation Therapy and Temozolomide in people with Recurrent Glioblastoma and Recurrent Gliosarcoma, run by University of Michigan Rogel Cancer Center 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

Search all trials

Trial summary

This is a phase 0/1 dose-escalation trial to determine the maximum tolerated dose of Mycophenolate Mofetil (MMF) when administered with radiation, in patients with glioblastoma or gliosarcoma. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Recurrent Glioblastoma, Recurrent Gliosarcoma, Recurrent Astrocytoma, Grade IV
What is being tested?: Mycophenolate Mofetil, Radiation Therapy and Temozolomide
Type of treatment: Pill or oral medicine, Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 68 participants
Where: 1 siteUnited States
Study dates: 5 Aug 2020to 5 Nov 2027
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Glioblastoma or gliosarcoma (recurrent or newly diagnosed).
Karnofsky Performance Status 60 or greater.
Phase 0: Candidate for clinically indicated re-resection or biopsy of glioblastoma or gliosarcoma per treating physician(s).
Phase 1, Recurrent: Candidate for clinically indicated re-irradiation of glioblastoma or gliosarcoma per treating physician(s) (No more than one prior course of radiation for GBM).
Phase 1, Newly Diagnosed: Candidate for upfront standard of care chemoradiation for glioblastoma or gliosarcoma per treating physician(s), to start no earlier than 14 days post- operatively from last definitive surgery for glioblastoma or gliosarcoma (if more than one surgery done. Ex. biopsy prior to resection).
ANC >=1,500 cells/mm^3 within 14 days prior to enrollment.
Patient (men and childbearing age women) agrees to the use of highly effective contraception during study participation and for at least 6 weeks for female patients and 90 days for male patients after final MMF administration.
Ability to understand and the willingness to sign a written informed consent.

Who cannot join

Lack of histopathological diagnosis of the tumor.
Gliomatosis cerebri pattern (tumor involving 3 or more lobes) of disease.
Leptomeningeal disease.
Use of bevacizumab within 8 weeks of study enrollment.
Known history of HIV.
Active hepatitis B or C infection.

Show all 23Show fewer

Active systemic or central nervous system (CNS) infection.
Grade 4 lymphopenia (if ALC <0.5, patient must be on Pneumocystis jirovecii prophylaxis).
Estimated CrCl < 25 ml/min.
History of organ transplantation.
Patients with known hypoxanthine-guanine phosphoribosyl-transferase deficiency.
Serious intercurrent disease.
History of allergic reaction or hypersensitivity to mycophenolate mofetil or mycophenolic acid or any component of the drug product; or medical contraindication for MMF per treating physician(s).
Known immunosuppressive condition from autoimmune disease, immune deficiency syndrome, or chronic immunosuppressive therapy.
Inability to undergo MRI brain with and without contrast.
Pregnant or lactating women.
Patients with known phenylketonuria.
Phase 0: Patients undergoing biopsy who are deemed unlikely to have sufficient tissue to spare for research purposes (e.g., those whose tumors are in an eloquent brain location where all tissue taken must be used for diagnostic purposes).
Phase I: Increase in steroid requirement within 7 days of study enrollment (stable or decreasing dose allowed).
Phase I, Recurrent: Radiation within 6 months prior to study enrollment.
Phase I, Recurrent: Surgery within 4 weeks of re-irradiation.
Phase I, Newly Diagnosed: History of hypersensitivity reactions to temozolomide or any other ingredients in temozolomide and dacarbazine.
Phase I, Newly Diagnosed: Prior chemotherapy or radiation therapy for glioblastoma or gliosarcoma.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, 48109

Other trials in United States

About this study

Official title: Phase 0/I Dose Escalation Study of Mycophenolate Mofetil Combined With Radiation Therapy in Glioblastoma

This is a phase 0/1 dose-escalation trial to determine the maximum tolerated dose of Mycophenolate Mofetil (MMF) when administered with radiation, in patients with glioblastoma or gliosarcoma.

Read the detailed description

The goal of the Phase 0 component is to determine if MMF achieves active concentrations in brain tumors. Eight participants in Phase 0 will receive MMF for one week before undergoing an already planned biopsy or re-resection (surgical removal) of glioblastoma or gliosarcoma (GBM/GS). A small portion of the tumor, removed as part of clinical care, will be used for testing in this study. Sixty additional participants will be enrolled in the Phase 1 component of the trial (30 with recurrent GBM/GS and 30 with newly diagnosed GBM/GS). The goal of the Phase 1 component is to find the dose of MMF that works best without causing severe side effects (the maximum tolerated dose) when combined with radiation in recurrent GBM/GS and with radiation and chemotherapy in newly diagnosed GBM/GS. Participants in Phase 0 who meet the eligibility criteria for the Phase 1 component may participate in both phases.

Conditions: Recurrent Glioblastoma, Recurrent Gliosarcoma, Recurrent Astrocytoma, Grade IV, Newly Diagnosed Glioblastoma, Newly Diagnosed Gliosarcoma, Newly Diagnosed Astrocytoma, Grade IV
Being tested: Mycophenolate Mofetil, Radiation Therapy, Re-resection (as part of standard of care), Temozolomide
Keywords: recurrent Glioblastoma, recurrent Gliosarcoma, newly diagnosed Glioblastoma, newly diagnosed Gliosarcooma, Recurrent Astrocytoma, Grade IV, Newly Diagnosed Astrocytoma, Grade IV
Medical subject headings: Glioblastoma, Gliosarcoma

What participants receive

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

Mycophenolate MofetilDrug
Also called MMF
500-2000mg orally twice daily, one week prior to re-resection (2 participants at each of 4 dose levels: 500mg, 1000mg, 1500mg and 2000mg)
Radiation TherapyRadiation
Also called RT
40.5 Gy in 15 fractions
Re-resection (as part of standard of care)Procedure or surgery
Re-resection or biopsy of tumor as part of standard of care
TemozolomideDrug
Also called TMZ
Temozolomide capsules are an approved oral chemotherapeutic drug for the treatment of adult patients with newly diagnosed GBM/GS concomitantly with radiotherapy and then as adjuvant treatment. The dosing and timing of temozolomide therapy will be determined as per standard-of-care for the individual patient by the treating oncologist.
Mycophenolate MofetilDrug
Also called MMF
250-2000mg orally twice daily, one week prior to and concurrent with RT.
Mycophenolate MofetilDrug
Also called MMF
250-2000mg orally twice daily, one week prior to and concurrent with RT and cyclic chemotherapy with temozolomide.
Radiation TherapyRadiation
Also called RT
60 Gy in 30 fractions

Study groups in NCT04477200
Study group	Role	What they receive
Phase 0 - Recurrent glioblastoma (GBM) / gliosarcoma (GS)	Experimental	Mycophenolate mofetil Receives: Drug: Mycophenolate Mofetil; Procedure: Re-resection (as part of standard of care)
Phase 1 - Recurrent GBM / GS	Experimental	Mycophenolate mofetil; radiation therapy Receives: Radiation: Radiation Therapy; Drug: Mycophenolate Mofetil
Phase 1 - Newly Diagnosed GBM / GS	Experimental	Mycophenolate mofetil; radiation therapy; temozolomide Receives: Drug: Temozolomide; Drug: Mycophenolate Mofetil; Radiation: Radiation Therapy

What is being measured

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

Concentration of Mycophenolic Acid (MPA) in Tumor Tissue in Phase 0 Participants
The concentration of MPA (the active metabolite of mycophenolate mofetil [MMF]) in tumor tissue, measured by mass spectrometry on a continuous scale after one week of MMF administration.
This measure includes all phase 0 participants.
Measured: At 1 week
Number of Recurrent Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level
DLT will be defined based on the rate of drug related grade 3-5 adverse events experienced from the start of treatment with MMF and for up to 4 weeks after completion of MMF + radiation therapy (RT). Assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
This measure includes only phase 1 participants with recurrent GBM/GS.
Measured: Up to 28 days following completion of MMF + RT (up to ~9 weeks)
Number of Newly Diagnosed Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level -- DLT1 Period
DLT will be defined based on the rate of drug related grade 3-5 adverse events experienced from the start of treatment with MMF and for up to 4 weeks after completion of MMF + RT + TMZ. Assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
This measure includes only newly diagnosed phase 1 participants.
Measured: Up to 28 days following completion of MMF + RT + TMZ (up to ~11 weeks)
Number of Newly Diagnosed Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level -- DLT2 Period
DLT will be defined based on the rate of drug related grade 3-5 adverse events experienced during the first 2 cycles (8 weeks) of MMF with adjuvant TMZ. (The first cycle of MMF with adjuvant TMZ begins 28 days post-RT.) These will be assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
This measure includes only newly diagnosed phase 1 participants.
Measured: During the first 2 cycles (8 weeks) of MMF with adjuvant TMZ (up to ~19 weeks)

Secondary outcomes (7)

Concentrations of Guanosine Triphosphate (GTP) in Tumor Tissue in Phase 0 Participants
The concentrations of GTP in tumor tissue, measured by mass spectrometry on a continuous scale.
This measure includes all phase 0 participants.
Measured: After one week of MMF administration
Adverse Events Associated With Treatment in All Phase 1 Participants
Toxicities at each dose level will be tabulated, categorized by grade and attribution.
This measure includes all phase 1 participants.
Measured: Up to 28 days following completion of MMF + RT (up to ~9 weeks)
Adverse Events Associated With Treatment in Newly Diagnosed Phase 1 Participants
Toxicities at each dose level will be tabulated, categorized by grade and attribution.
This measure includes only newly diagnosed phase 1 participants.
Measured: Up to 28 days following completion of MMF with adjuvant temozolomide (up to ~15 months)
Overall Response Rate in Phase 1 Participants With Recurrent GBM/GS
Determined by modified Response Assessment for Neuro-Oncology (mRANO) criteria. The number and proportion of patients with progressive disease, stable disease, partial and complete response will be calculated for each dose level and overall.
This measure includes only phase 1 participants with recurrent GBM/GS.
Measured: Until study stops or death; up to approximately 3 years.
Median Progression Free Survival (PFS) in Phase 1 Participants With Recurrent GBM/GS
PFS defined as time from date of registration to the date of documented progressive disease, other disease related therapy or death. Determined by mRANO criteria.
This measure includes only phase 1 participants with recurrent GBM/GS.
Measured: Until study stops or death; up to approximately 3 years.
Median Freedom From Local Progression (FFLP) in Phase 1 Participants With Recurrent GBM/GS
FFLP defined as time from date of registration to the date of documented local progressive disease. Determined by mRANO criteria.
This measure includes only phase 1 participants with recurrent GBM/GS.
Measured: Until study stops or death; up to approximately 3 years.
Median Overall Survival (OS) in Phase 1 Participants With Recurrent GBM/GS
OS defined as time from date of registration to date of death or last follow up. Determined by Kaplan Meier method.
This measure includes only phase 1 participants with recurrent GBM/GS.
Measured: Until study stops or death; up to approximately 3 years.

Timeline

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

5 Aug 20205 Nov 2027

First posted20 Jul 2020
Study start5 Aug 2020
Primary completion6 Jan 2025
Study completion5 Nov 2027 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Recurrent GlioblastomaStudies of the same condition, open and under way.
More trials run by University of Michigan Rogel Cancer CenterThe 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
Model description: Phase 0 will include 8 participants; eligible participants from phase 0 may continue on to phase 1.
Masking: None (open label)
Enrolment: 68 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04477200
Sponsor's protocol ID: UMCC 2019.192
Other IDs: HUM00175785, R37CA258346
Status last verified: Apr 2026
First submitted: 14 Jul 2020
Last updated: 14 May 2026
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Zhao G, Newbury P, Ishi Y, Chekalin E, Zeng B, Glicksberg BS, Wen A, Paithankar S, Sasaki T, Suri A, Nazarian J, Pacold ME, Brat DJ, Nicolaides T, Chen B, Hashizume R. Reversal of cancer gene expression identifies repurposed drugs for diffuse intrinsic pontine glioma. Acta Neuropathol Commun. 2022 Oct 23;10(1):150. doi: 10.1186/s40478-022-01463-z.
PubMed 36274161

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1/2
Testing the Addition of an Anti-cancer Drug, Selinexor, to the Usual Chemotherapy Treatment (Temozolomide) for Brain Tumors That Have Returned After Previous Treatment
Recurrent Glioblastoma, IDH-Wildtype, Recurrent MGMT-Methylated Glioblastoma
RecruitingPhase 1/2
PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
Glioma, Medulloblastoma
RecruitingObservational study
Collection of Blood and Urine Samples in Patients Receiving Radiation Therapy for Glioblastoma Multiforme
Glioblastoma Multiforme
Active, not recruitingPhase 3
Sonocloud-9 in Association With Carboplatin Versus Standard-of-Care Chemotherapies (CCNU or TMZ) in Recurrent GBM
Glioblastoma, Recurrent Glioblastoma
RecruitingPhase 1
A Phase 1 Safety and Dose Finding Study of GLIX1 in Adults With Recurrent or Progressive High-grade Glioma
Glioblastoma Multiforme of Brain, Glioma

All Recurrent Glioblastoma trials

Study at a glance

From the registry record, last updated by the sponsor on 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase 0/I Dose Escalation Study of Mycophenolate Mofetil Combined With Radiation Therapy in Glioblastoma
ClinicalTrials.gov ID: NCT04477200
Acronym / sponsor ID: UMCC 2019.192
Conditions: Recurrent Glioblastoma, Recurrent Gliosarcoma, Recurrent Astrocytoma, Grade IV, Newly Diagnosed Glioblastoma, Newly Diagnosed Gliosarcoma, Newly Diagnosed Astrocytoma, Grade IV
Keywords: recurrent Glioblastoma, recurrent Gliosarcoma, newly diagnosed Glioblastoma, newly diagnosed Gliosarcooma, Recurrent Astrocytoma, Grade IV, Newly Diagnosed Astrocytoma, Grade IV
Phase: Phase 1
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 68 participants (actual)
Lead sponsor: University of Michigan Rogel Cancer CenterUniversity, hospital or other
Collaborators: National Cancer Institute (NCI)
Study start: 5 Aug 2020 (actual)
Primary completion: 6 Jan 2025 (actual)
Study completion: 5 Nov 2027 (estimated)
First posted: 20 Jul 2020 (actual)
Results: Posted 14 May 2026

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 0/1 dose-escalation trial to determine the maximum tolerated dose of Mycophenolate Mofetil (MMF) when administered with radiation, in patients with glioblastoma or gliosarcoma.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 3
Model description: Phase 0 will include 8 participants; eligible participants from phase 0 may continue on to phase 1.

Arm	Type	Receives
Phase 0 - Recurrent glioblastoma (GBM) / gliosarcoma (GS)	Experimental	Drug: Mycophenolate Mofetil, Procedure: Re-resection (as part of standard of care)Mycophenolate mofetil
Phase 1 - Recurrent GBM / GS	Experimental	Radiation: Radiation Therapy, Drug: Mycophenolate MofetilMycophenolate mofetil; radiation therapy
Phase 1 - Newly Diagnosed GBM / GS	Experimental	Drug: Temozolomide, Drug: Mycophenolate Mofetil, Radiation: Radiation TherapyMycophenolate mofetil; radiation therapy; temozolomide

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

Glioblastoma or gliosarcoma (recurrent or newly diagnosed).
Karnofsky Performance Status 60 or greater.
Phase 0: Candidate for clinically indicated re-resection or biopsy of glioblastoma or gliosarcoma per treating physician(s).
Phase 1, Recurrent: Candidate for clinically indicated re-irradiation of glioblastoma or gliosarcoma per treating physician(s) (No more than one prior course of radiation for GBM).
Phase 1, Newly Diagnosed: Candidate for upfront standard of care chemoradiation for glioblastoma or gliosarcoma per treating physician(s), to start no earlier than 14 days post- operatively from last definitive surgery for glioblastoma or gliosarcoma (if more than one surgery done. Ex. biopsy prior to resection).

Key exclusion criteria (23 listed)

Lack of histopathological diagnosis of the tumor.
Gliomatosis cerebri pattern (tumor involving 3 or more lobes) of disease.
Leptomeningeal disease.
Use of bevacizumab within 8 weeks of study enrollment.
Known history of HIV.

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: MMF500-2000mg orally twice daily, one week prior to re-resection (2 participants at each of 4 dose levels: 500mg, 1000mg, 1500mg and 2000mg)	Drug	Phase 0 - Recurrent glioblastoma (GBM) / gliosarcoma (GS)
Radiation TherapyAlso: RT40.5 Gy in 15 fractions	Radiation	Phase 1 - Recurrent GBM / GS
Re-resection (as part of standard of care)Re-resection or biopsy of tumor as part of standard of care	Procedure or surgery	Phase 0 - Recurrent glioblastoma (GBM) / gliosarcoma (GS)
TemozolomideAlso: TMZTemozolomide capsules are an approved oral chemotherapeutic drug for the treatment of adult patients with newly diagnosed GBM/GS concomitantly with radiotherapy and then as adjuvant treatment. The dosing and timing of temozolomide therapy will be determined as per standard-of-care for the individual patient by the treating oncologist.	Drug	Phase 1 - Newly Diagnosed GBM / GS
Mycophenolate MofetilAlso: MMF250-2000mg orally twice daily, one week prior to and concurrent with RT.	Drug	Phase 1 - Recurrent GBM / GS
Mycophenolate MofetilAlso: MMF250-2000mg orally twice daily, one week prior to and concurrent with RT and cyclic chemotherapy with temozolomide.	Drug	Phase 1 - Newly Diagnosed GBM / GS
Radiation TherapyAlso: RT60 Gy in 30 fractions	Radiation	Phase 1 - Newly Diagnosed GBM / GS

Primary outcome

Concentration of Mycophenolic Acid (MPA) in Tumor Tissue in Phase 0 Participants
Time frame: At 1 week
The concentration of MPA (the active metabolite of mycophenolate mofetil [MMF]) in tumor tissue, measured by mass spectrometry on a continuous scale after one week of MMF administration. This measure includes all phase 0 participants.
Number of Recurrent Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level
Time frame: Up to 28 days following completion of MMF + RT (up to ~9 weeks)
DLT will be defined based on the rate of drug related grade 3-5 adverse events experienced from the start of treatment with MMF and for up to 4 weeks after completion of MMF + radiation therapy (RT). Assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0. This measure includes only phase 1 participants with recurrent GBM/GS.
Number of Newly Diagnosed Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level -- DLT1 Period
Time frame: Up to 28 days following completion of MMF + RT + TMZ (up to ~11 weeks)
DLT will be defined based on the rate of drug related grade 3-5 adverse events experienced from the start of treatment with MMF and for up to 4 weeks after completion of MMF + RT + TMZ. Assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0. This measure includes only newly diagnosed phase 1 participants.
Number of Newly Diagnosed Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level -- DLT2 Period
Time frame: During the first 2 cycles (8 weeks) of MMF with adjuvant TMZ (up to ~19 weeks)
DLT will be defined based on the rate of drug related grade 3-5 adverse events experienced during the first 2 cycles (8 weeks) of MMF with adjuvant TMZ. (The first cycle of MMF with adjuvant TMZ begins 28 days post-RT.) These will be assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0. This measure includes only newly diagnosed phase 1 participants.

7 secondary outcomes

Concentrations of Guanosine Triphosphate (GTP) in Tumor Tissue in Phase 0 Participants
Time frame: After one week of MMF administration
Adverse Events Associated With Treatment in All Phase 1 Participants
Time frame: Up to 28 days following completion of MMF + RT (up to ~9 weeks)
Adverse Events Associated With Treatment in Newly Diagnosed Phase 1 Participants
Time frame: Up to 28 days following completion of MMF with adjuvant temozolomide (up to ~15 months)
Overall Response Rate in Phase 1 Participants With Recurrent GBM/GS
Time frame: Until study stops or death; up to approximately 3 years.
Median Progression Free Survival (PFS) in Phase 1 Participants With Recurrent GBM/GS
Time frame: Until study stops or death; up to approximately 3 years.
Median Freedom From Local Progression (FFLP) in Phase 1 Participants With Recurrent GBM/GS
Time frame: Until study stops or death; up to approximately 3 years.
Median Overall Survival (OS) in Phase 1 Participants With Recurrent GBM/GS
Time frame: Until study stops or death; up to approximately 3 years.

Results and safety

Results posted to the registry on 14 May 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 2	2	2
Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 3	2	2
Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 4	2	2
Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 5	2	2
Phase 1 - Recurrent GBM / GS - Level 3	3	3
Phase 1 - Recurrent GBM / GS - Level 4	4	4
Phase 1 - Recurrent GBM / GS - Level 5	19	17
Phase 1 - Newly Diagnosed GBM / GS - Level 3	4	3
Phase 1 - Newly Diagnosed GBM / GS- Level 4	11	6
Phase 1 - Newly Diagnosed GBM / GS- Level 5	16	11

Primary outcome: Concentration of Mycophenolic Acid (MPA) in Tumor Tissue in Phase 0 Participants

Time frame: At 1 week. Median (Full Range). Unit: nmol/g

Measure	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 2 n = 2	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 3 n = 2	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 4 n = 2	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 5 n = 2	Phase 1 - Recurrent GBM / GS - Level 3 n = 0	Phase 1 - Recurrent GBM / GS - Level 4 n = 0	Phase 1 - Recurrent GBM / GS - Level 5 n = 0	Phase 1 - Newly Diagnosed GBM / GS - Level 3 n = 0	Phase 1 - Newly Diagnosed GBM / GS- Level 4 n = 0	Phase 1 - Newly Diagnosed GBM / GS- Level 5 n = 0
Enhancing Tumor	1.87 (1.55 to 2.19)	2.65 (2.15 to 3.15)	2.65 (1.74 to 3.56)	1.8 (1.5 to 2.11)	–	–	–	–	–	–
Non- Enhancing Tumor	1.11 (0.76 to 1.45)	0.81 (0.81 to 0.81)	1.53 (1.01 to 2.05)	1.25 (0.72 to 1.79)	–	–	–	–	–	–
Normal Brain	–	2.55 (2.55 to 2.55)	2.59 (1.66 to 3.51)	0.64 (0.4 to 0.88)	–	–	–	–	–	–

Population analysed: only phase 0 subjects were analyzed

Primary outcome: Number of Recurrent Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level

Time frame: Up to 28 days following completion of MMF + RT (up to ~9 weeks). Number. Unit: number of patients reporting a DLT

Measure	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 2 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 3 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 4 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 5 n = 0	Phase 1 - Recurrent GBM / GS - Level 3 n = 3	Phase 1 - Recurrent GBM / GS - Level 4 n = 4	Phase 1 - Recurrent GBM / GS - Level 5 n = 19	Phase 1 - Newly Diagnosed GBM / GS - Level 3 n = 0	Phase 1 - Newly Diagnosed GBM / GS- Level 4 n = 0	Phase 1 - Newly Diagnosed GBM / GS- Level 5 n = 0
Value	–	–	–	–	0	0	0	–	–	–

Population analysed: Only phase 1 recurrent subjects reported

Primary outcome: Number of Newly Diagnosed Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level -- DLT1 Period

Time frame: Up to 28 days following completion of MMF + RT + TMZ (up to ~11 weeks). Number. Unit: number of patients reporting a DLT

Measure	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 2 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 3 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 4 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 5 n = 0	Phase 1 - Recurrent GBM / GS - Level 3 n = 0	Phase 1 - Recurrent GBM / GS - Level 4 n = 0	Phase 1 - Recurrent GBM / GS - Level 5 n = 0	Phase 1 - Newly Diagnosed GBM / GS - Level 3 n = 4	Phase 1 - Newly Diagnosed GBM / GS- Level 4 n = 10	Phase 1 - Newly Diagnosed GBM / GS- Level 5 n = 16
Value	–	–	–	–	–	–	–	0	0	5

Population analysed: Only Phase 1 newly diagnosed subjects analyzed

Primary outcome: Number of Newly Diagnosed Phase 1 Participants Who Experience Dose-limiting Toxicities (DLTs) at Each Dose Level -- DLT2 Period

Time frame: During the first 2 cycles (8 weeks) of MMF with adjuvant TMZ (up to ~19 weeks). Number. Unit: number of patients reporting a DLT

Measure	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 2 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 3 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 4 n = 0	Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 5 n = 0	Phase 1 - Recurrent GBM / GS - Level 3 n = 0	Phase 1 - Recurrent GBM / GS - Level 4 n = 0	Phase 1 - Recurrent GBM / GS - Level 5 n = 0	Phase 1 - Newly Diagnosed GBM / GS - Level 3 n = 4	Phase 1 - Newly Diagnosed GBM / GS- Level 4 n = 6	Phase 1 - Newly Diagnosed GBM / GS- Level 5 n = 16
Value	–	–	–	–	–	–	–	0	1	0

Population analysed: Only phase 1 newly diagnosed subjects analyzed

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Events are collected from the time of initial study enrollment through 30 days after the last dose of MMF, approximately 6 months. Data is currently being collected. All Data collected through the primary completion date has been reported. Data broken down by dose is not yet available as data is still ongoing.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 2	1 of 2 (50%)	2 of 2 (100%)	1 of 2
Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 3	0 of 2 (0%)	0 of 2 (0%)	1 of 2
Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 4	0 of 2 (0%)	2 of 2 (100%)	0 of 2
Phase 0 - Recurrent Glioblastoma (GBM) / Gliosarcoma (GS) - Level 5	0 of 2 (0%)	2 of 2 (100%)	1 of 2
Phase 1 - Recurrent GBM / GS - Level 3	0 of 3 (0%)	3 of 3 (100%)	2 of 3
Phase 1 - Recurrent GBM / GS - Level 4	2 of 4 (50%)	4 of 4 (100%)	4 of 4
Phase 1 - Recurrent GBM / GS - Level 5	2 of 19 (10.5%)	16 of 19 (84.2%)	12 of 19
Phase 1 - Newly Diagnosed GBM / GS - Level 3	1 of 4 (25%)	4 of 4 (100%)	2 of 4
Phase 1 - Newly Diagnosed GBM / GS- Level 4	4 of 11 (36.4%)	10 of 11 (90.9%)	8 of 11
Phase 1 - Newly Diagnosed GBM / GS- Level 5	9 of 16 (56.3%)	16 of 16 (100%)	11 of 16

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 36274161Zhao G, Newbury P, Ishi Y, Chekalin E, Zeng B, Glicksberg BS, Wen A, Paithankar S, Sasaki T, Suri A, Nazarian J, Pacold ME, Brat DJ, Nicolaides T, Chen B, Hashizume R. Reversal of cancer gene expression identifies repurposed drugs for diffuse intrinsic pontine glioma. Acta Neuropathol Commun. 2022 Oct 23;10(1):150. doi: 10.1186/s40478-022-01463-z.
Individual participant dataSharing planned: No

Related trials for Recurrent Glioblastoma

RecruitingPhase 1/2
Testing the Addition of an Anti-cancer Drug, Selinexor, to the Usual Chemotherapy Treatment (Temozolomide) for Brain Tumors That Have Returned After Previous Treatment
Recurrent Glioblastoma, IDH-Wildtype, Recurrent MGMT-Methylated Glioblastoma
RecruitingPhase 1/2
PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
Glioma, Medulloblastoma
RecruitingObservational study
Collection of Blood and Urine Samples in Patients Receiving Radiation Therapy for Glioblastoma Multiforme
Glioblastoma Multiforme
Active, not recruitingPhase 3
Sonocloud-9 in Association With Carboplatin Versus Standard-of-Care Chemotherapies (CCNU or TMZ) in Recurrent GBM
Glioblastoma, Recurrent Glioblastoma
RecruitingPhase 1
A Phase 1 Safety and Dose Finding Study of GLIX1 in Adults With Recurrent or Progressive High-grade Glioma
Glioblastoma Multiforme of Brain, Glioma

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.

Nathan Clarke, MD principal investigator

University of Michigan Rogel Cancer Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 8 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 14 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?

Yes. Results are posted in the registry (first posted 14 May 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Concentration of Mycophenolic Acid (MPA) in Tumor Tissue in Phase 0 Participants (time frame: At 1 week). The study lists 4 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 NCT04477200, which also lists 1 linked publication.

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 14 May 2026.