Acetadote for Glioblastoma

Age: 18+
Sex: Any
Trial Phase: Phase < 1
Sponsor: University of Texas Southwestern Medical Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to assess how Acetadote affects the metabolism of brain tumors in individuals with glioblastoma, an aggressive brain cancer. Researchers believe Acetadote may reduce cell damage and slow tumor growth. Participants will receive Acetadote before specific scans and surgery to measure changes in tumor metabolism. The trial seeks individuals newly diagnosed with glioblastoma who have not yet undergone any cancer treatments. As an Early Phase 1 trial, this research focuses on understanding how Acetadote works in people, offering participants the opportunity to be among the first to receive this potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you cannot have had chemotherapy, radiotherapy, or other cancer treatments within 4 weeks before starting the study.

Is there any evidence suggesting that Acetadote is likely to be safe for humans?

Research shows that Acetadote, also known as N-acetylcysteine (NAC), is generally safe and well-tolerated, with serious side effects being uncommon. The likelihood of side effects mainly depends on the method of administration and the dose. In most cases, side effects are rare, especially when the drug is not taken in high amounts.

In past studies, Acetadote has been used in various situations and has proven safe for many patients. For instance, a study in Australia examined the safety of Acetadote when given by injection and found that allergic reactions could occur, but they were not common. Importantly, the FDA has already approved Acetadote for other uses, which increases confidence in its safety.

Overall, while some mild side effects might occur, many people have safely used Acetadote for other health issues.12345

Why do researchers think this study treatment might be promising?

Most treatments for glioblastoma involve surgery, radiation, and chemotherapy, often using drugs like temozolomide. However, Acetadote is unique because it's being explored for its potential to protect healthy brain tissue during treatment. Unlike the standard options, Acetadote uses N-acetylcysteine (NAC), an antioxidant, which is administered intravenously to potentially reduce oxidative stress and protect cells. Researchers are excited because this approach could enhance the effectiveness of existing therapies by safeguarding normal brain cells, potentially improving outcomes for patients with this aggressive cancer.

What evidence suggests that Acetadote might be an effective treatment for glioblastoma?

Research shows that Acetadote, also known as N-acetylcysteine (NAC), can affect the growth of glioblastoma, a type of brain tumor. Studies have found that NAC slows the growth of these cancer cells by making survival more difficult. In lab experiments, NAC killed glioblastoma cells by disrupting energy production in the mitochondria, the cell's energy producers. Additionally, NAC reduced tumor growth and prevented recurrence in mice. Overall, these findings suggest that Acetadote, which participants in this trial will receive, could be a promising treatment to slow glioblastoma progression.56789

Are You a Good Fit for This Trial?

This trial is for adults over 18 with a new diagnosis of glioblastoma, who haven't had surgery or other treatments yet. They should be relatively healthy (KPS > 70) and have normal levels of bilirubin, liver enzymes, kidney function, white blood cells, platelets, and hemoglobin.

Inclusion Criteria

I am 18 years old or older.
I am mostly able to care for myself and carry out daily activities.
I have not had any treatment for my cancer.
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Acetadote is administered twice: first before MRS on Day 1 and second before surgical resection between Days 2-21

3 weeks
2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Acetadote

Trial Overview

The trial is testing Acetadote's effect on brain tumor metabolism in glioblastoma patients. It involves MRI scans to monitor changes and analysis of tumor tissue after surgical removal to see how the drug alters cell damage levels.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: Acetadote 150 mg/kg IVExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Texas Southwestern Medical Center

Lead Sponsor

Trials
1,102
Recruited
1,077,000+

Cumberland Pharmaceuticals

Industry Sponsor

Trials
63
Recruited
9,400+

A.J. Kazimi

Cumberland Pharmaceuticals

Chief Executive Officer since 1999

B.S. from the University of Notre Dame, MBA from Vanderbilt University Owen Graduate School of Management

Ines Macias-Perez

Cumberland Pharmaceuticals

Chief Medical Officer

Ph.D. in Cancer Biology from Vanderbilt University

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/30606241/

N-acetylcysteine decreases malignant characteristics of ...

Results: Our data showed that NAC could decrease the protein level of Notch2. Meanwhile, NAC had a decreasing effect on the mRNA and protein ...

Imaging Acetadote Metabolism in Glioblastoma

We are evaluating how Acetadote affects glioblastoma metabolism by using MRI-based methods and by determining the changes in metabolism in brain tumor tissue ...

N-acetylcysteine decreases malignant characteristics of ...

In our study, NAC was found to inhibit cell viability of GBM cells at 10 mM and 20 mM effectively (Fig. 1a). Treatment of U87 and U251 cells ...

N-acetylcysteine remodels the tumor microenvironment of ...

NAC inhibited tumor growth and suppressed recurrence in mouse brain tumor models. Conclusion. NAC is a promising adjunctive drug to remodel the ...

Cysteine induces mitochondrial reductive stress in ...

We found that cysteine compounds, including N-acetylcysteine (NAC), kill GBM cells by reducing mitochondrial oxygen consumption and membrane potential.

Imaging Acetadote Metabolism in Glioblastoma

We are evaluating how Acetadote affects glioblastoma metabolism by using MRI-based methods and by determining the changes in metabolism in brain ...

N-Acetylcysteine (NAC): Impacts on Human Health - PMC

It has a well-established safety profile, and its toxicity is uncommon and dependent on the route of administration and high dosages. Its remarkable antioxidant ...

TMET-47. N-acetylcysteine induced lactate reprogramming is ...

Our data suggests that lactate reprogramming is a targetable susceptibility of NAC treatment. The unique sensitivity of GBM towards NAC and its ...

Acetadote (acetylcysteine) Injection Package Insert

Safety Study. A randomized, open-label, multi-center clinical study was conducted in Australia to compare the rates of anaphylactoid reactions ...