What side effects are associated with this type of intervention?

Common treatments for glioblastoma, such as temozolomide and radiation therapy, have several known side effects. Temozolomide can cause nausea, vomiting, fatigue, constipation, and myelosuppression, which includes decreased levels of white blood cells, red blood cells, and platelets, increasing the risk of infection, anemia, and bleeding. Radiation therapy can lead to fatigue, hair loss at the treatment site, skin changes, and potential cognitive decline. When combined with mycophenolate mofetil, an immunosuppressant, there may be additional risks of infections and gastrointestinal disturbances.

What prior FDA approvals exist?

Temozolomide is a key FDA-approved drug for the treatment of glioblastoma, often used in combination with radiation therapy. It works by sensitizing tumor cells to chemotherapy, similar to the approach being studied with Mycophenolate Mofetil. Other treatments include bevacizumab, an anti-VEGF monoclonal antibody, which has shown activity in recurrent glioblastoma primarily due to its vascular normalization and antiedema effects. These treatments aim to improve the efficacy of chemotherapy and manage tumor progression.

What other types of research exist?

Promising novel alternatives for Glioblastoma treatment in 2024 include: 1) Imetelstat, an investigational antisense oligonucleotide targeting telomerase, which has shown partial and complete responses in myelofibrosis and may have potential in glioblastoma. 2) Novel immunotherapy approaches such as T-cell transfer immunotherapy and checkpoint inhibitors like nivolumab, which have shown efficacy in other cancers and are being explored for glioblastoma. 3) Oncolytic virus therapies, which use genetically modified viruses to target and kill cancer cells while stimulating an immune response. These alternatives are under investigation and may offer new hope for glioblastoma patients.

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Immunosuppressant

Mycophenolate Mofetil + Standard Care for Glioblastoma

Phase 1

Recruiting

Led By Priya U Kumthekar, MD

Research Sponsored by Northwestern University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with histologically confirmed glioblastoma (GBM), IDH wild-type or astrocytoma with molecular features of GBM

Patients with Karnofsky performance status >= 70

Must not have

Patients with specific medical conditions or illnesses including uncontrolled epilepsy, concurrent malignancy requiring treatment, certain infections, immunodeficiency, psychiatric illness, pregnancy or nursing, swallowing difficulties, and known history of HIV, HBV, or HCV

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 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

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trial tests if combining mycophenolate mofetil with temozolomide and radiation can better treat glioblastoma, an aggressive brain cancer. Mycophenolate mofetil may help chemotherapy work better by making cancer cells easier to kill. Temozolomide is a standard chemotherapeutic drug for glioblastoma, often combined with radiotherapy to improve patient survival.

Who is the study for?

Adults diagnosed with glioblastoma, who've had surgery or biopsy and are stable on low-dose corticosteroids. They must be able to perform daily activities (Karnofsky score >=70), have adequate organ function, and agree to use contraception. Excluded are those with other cancers, certain infections, uncontrolled illnesses, pregnant/nursing women, inability to take oral meds, or known HIV/HBV/HCV.

What is being tested?

The trial is testing the safety and optimal dose of Mycophenolate Mofetil when combined with standard treatments for glioblastoma: Temozolomide and Radiation Therapy. The goal is to see if this drug makes cancer cells more sensitive to treatment.

What are the potential side effects?

Mycophenolate Mofetil may cause immune system suppression leading to a higher risk of infections. Other side effects can include upset stomach, blood cell changes increasing infection risk or bleeding problems, liver issues, and potential interactions with live vaccines.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My brain tumor is confirmed as glioblastoma or has similar features.

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I am mostly able to care for myself.

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I have a new glioblastoma diagnosis and have had surgery or biopsy, with or without chemoradiation.

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I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have uncontrolled epilepsy, another cancer needing treatment, severe infections, immune issues, major psychiatric illness, and I'm not pregnant or nursing. I also don't have trouble swallowing or a history of HIV, HBV, or HCV.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 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

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 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 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

MTD/RP2D for MMF (Group 2)

Maximum tolerated dose/recommended phase 2 dose (MTD/RP2D) for mycophenolate mofetil (MMF) (Group 1)

Secondary study objectives

Frequency of adverse events

Overall response rate (ORR)

Overall survival (OS)

+2 more

Other study objectives

Mycophenolic acid levels

XMP concentration

Side effects data

From 2014 Phase 3 trial • 87 Patients • NCT00075478

23%

Blood/Bone marrow

11%

Cardiovascular

Pulmonary

Gastrointestinal

Hepatic

Graft versus host disease with infection and organ failure

Dermatology/Skin

respiratory failure

Hemorrhage

subdural hematoma

thrombosis

Renal/Genitourinary

Metabolic/Laboratory

100%

80%

60%

40%

20%

Study treatment Arm

Arm II (TBI, Transplant, GVHD Prophylaxis)

Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis)

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Group S (pre-surgical MMF, TMZ)Experimental Treatment3 Interventions

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.

Group II: Group 3 (TMZ, MMF, radiation therapy)Experimental Treatment4 Interventions

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.

Group III: Group 2 (TMZ, MMF, radiation therapy)Experimental Treatment4 Interventions

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 IV: Group 1 (TMZ, MMF)Experimental Treatment3 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mycophenolate mofetil

FDA approved

Temozolomide

FDA approved

Radiation Therapy

2017

Completed Phase 3

~7250

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Glioblastoma treatments often involve a combination of chemotherapy, radiation therapy, and sometimes surgery. Temozolomide, a common chemotherapeutic agent, works by alkylating/methylating DNA, which damages the DNA of cancer cells and inhibits their replication. Radiation therapy uses high-energy particles to destroy cancer cells by damaging their DNA. Mycophenolate Mofetil, an immunosuppressant, is being studied for its potential to make tumor cells more sensitive to chemotherapy, thereby enhancing the effectiveness of drugs like temozolomide. This is crucial for Glioblastoma patients as it could improve treatment outcomes by making cancer cells more vulnerable to existing therapies, potentially leading to better control of the disease.