Vorinostat + Isotretinoin + Temozolomide for Glioblastoma

Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: M.D. Anderson Cancer Center
Must be taking: Isotretinoin, Temozolomide
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 tests whether a combination of three drugs—vorinostat (a type of histone deacetylase inhibitor), isotretinoin, and temozolomide—can help control glioblastoma or gliosarcoma, aggressive brain tumors. Researchers are also assessing the safety of this drug combination for patients. Individuals with glioblastoma or gliosarcoma that has recurred or progressed after radiation therapy, and who have already tried other treatments, might be suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new combination therapy.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you cannot take valproic acid (an anticonvulsant) unless switched to another drug before starting the trial. Also, if you are on enzyme-inducing anti-epileptic drugs, your doctor might need to monitor your drug levels.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that the combination of vorinostat, isotretinoin, and temozolomide has been studied for safety in patients with high-grade gliomas. Vorinostat, tested in several studies, is generally considered safe but can cause side effects like tiredness and nausea. Isotretinoin, often used for other conditions, may lead to side effects such as dry skin or increased sensitivity to sunlight. Temozolomide, approved by the FDA for treating glioblastoma, is usually well-tolerated, though it may cause issues like low blood cell counts.

One study showed that the combination of vorinostat, isotretinoin, and temozolomide reduced tumor cell survival. However, other trials indicate that adding isotretinoin to temozolomide might not always be beneficial and could be harmful in some cases. These findings suggest that while the treatments have potential, risks exist, and side effects vary from person to person.12345

Why are researchers excited about this trial's treatments?

Researchers are excited about these treatments because they combine known cancer-fighting drugs in innovative ways to tackle glioblastoma, a particularly aggressive brain cancer. Unlike the standard treatments like radiation and surgery, which can be limited in their effectiveness, these combinations utilize isotretinoin, temozolomide, and vorinostat to potentially enhance anti-tumor activity. Vorinostat, in particular, works by inhibiting enzymes that cancer cells need to grow, offering a novel mechanism of action compared to traditional therapies. By targeting the cancer cells more directly and possibly improving the effectiveness of existing drugs, these combinations could lead to more effective treatment options for patients with glioblastoma.

What evidence suggests that this trial's treatments could be effective for glioblastoma?

Research shows that vorinostat, a type of drug, may help treat glioblastoma by slowing and stopping tumor growth. Studies have found that vorinostat can make glioblastoma cells less likely to survive and can even cause them to die. Temozolomide, a well-known chemotherapy drug, effectively treats glioblastoma by damaging the DNA of cancer cells, preventing their growth. Isotretinoin, commonly used for severe acne, has also shown some potential against glioblastoma when used alone.

In this trial, participants will join different treatment arms to evaluate the effectiveness of these drug combinations. One arm will receive temozolomide plus isotretinoin, another will receive vorinostat plus isotretinoin, and a third will receive vorinostat plus isotretinoin plus temozolomide. Combining these treatments might provide a stronger approach by attacking cancer cells in different ways. Early findings suggest that using these treatments together could better control glioblastoma growth.12678

Who Is on the Research Team?

MP

Marta Penas-Prado, MD

Principal Investigator

M.D. Anderson Cancer Center

VP

Vinay Puduvalli, MD

Principal Investigator

Brain Tumor Trials Collaborative (BTTC), and Ohio State University

Are You a Good Fit for This Trial?

Adults over 18 with recurrent WHO grade IV glioma (glioblastoma or gliosarcoma) who've failed radiation therapy can join. They must be able to sign consent, have a Karnofsky performance status >=60, stable health post-prior treatments, and agree to contraception. Exclusions include other cancers within 3 years (except certain types), prior bevacizumab treatment, valproic acid use without switching drugs first, inability to tolerate study procedures or swallow tablets.

Inclusion Criteria

My recent MRI was done within the last 17 days and I've been on a stable or decreasing dose of steroids for at least 5 days.
I can care for myself but may need occasional help.
I have recovered from side effects of my previous cancer treatments.
See 10 more

Exclusion Criteria

Patient must be able to tolerate study procedures and comply with protocol
I do not have an active infection, serious illness, or have been treated with HDAC inhibitors.
I am not currently taking valproic acid, or I have switched to another medication.
See 5 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive vorinostat, isotretinoin, and temozolomide in 28-day cycles

Up to 12 months
Weekly visits for blood tests and monitoring, with additional visits every 2 cycles for MRI and exams

Follow-up

Participants are monitored for safety and effectiveness after treatment

Every 2 months
Phone calls every 2 months, clinic visits if stopped due to side effects

What Are the Treatments Tested in This Trial?

Interventions

  • Isotretinoin
  • Temozolomide
  • Vorinostat
Trial Overview The trial tests if vorinostat combined with isotretinoin and temozolomide can control recurrent brain tumors in adults and assesses the safety of this drug combination. Participants will undergo surgical resection as needed and receive these medications under careful monitoring for effectiveness and adverse reactions.
How Is the Trial Designed?
5Treatment groups
Experimental Treatment
Active Control
Group I: Ph II: Arm 2Experimental Treatment1 Intervention
Group II: Ph I: Arm 3Experimental Treatment3 Interventions
Group III: Ph I: Arm 2Experimental Treatment2 Interventions
Group IV: Ph I: Arm 1Experimental Treatment2 Interventions
Group V: Ph II: Arm 1Active Control1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials
3,107
Recruited
1,813,000+

Merck Sharp & Dohme LLC

Industry Sponsor

Trials
4,096
Recruited
5,232,000+
Chirfi Guindo profile image

Chirfi Guindo

Merck Sharp & Dohme LLC

Chief Marketing Officer since 2022

Degree in Engineering from Ecole Centrale de Paris, MBA from New York University Stern School of Business

Robert M. Davis profile image

Robert M. Davis

Merck Sharp & Dohme LLC

Chief Executive Officer since 2021

JD from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University

Published Research Related to This Trial

The study confirmed that the generic temozolomide capsules (TOZ039) are bioequivalent to the brand Temodal® capsules, with pharmacokinetic parameters falling within the acceptable range for both 20-mg and 100-mg doses in 29 patients with brain tumors.
The treatment was found to be safe, with no treatment-related severe adverse events or mortality reported, although 82.8% of patients experienced some adverse effects, highlighting the importance of monitoring patient responses during treatment.
Bioequivalence study of 20-mg and 100-mg temozolomide capsules (TOZ309 and Temodal®) in glioma patients in China.Hu, C., Lin, Q., Liu, C., et al.[2021]
Naringenin (NGEN) significantly reduces the migration and invasion of drug-resistant glioma cells, suggesting it can enhance the effectiveness of temozolomide (TMZ) treatment.
The study provides evidence that NGEN can overcome TMZ resistance by modifying protein levels related to cell migration and invasion, making it a promising adjunct therapy for glioma patients.
Naringenin Sensitizes Resistant C6 Glioma Cells with a Repressive Impact on the Migrating Ability.J, J., Vanisree, AJ.[2022]
The maximum tolerated dose (MTD) of vorinostat when combined with temozolomide (TMZ) was determined to be 500 mg daily in the first part of the study, with dose-limiting toxicities including severe anorexia and thrombocytopenia.
Vorinostat was found to be well tolerated in patients with high-grade glioma, showing no significant pharmacokinetic interactions with TMZ, and it effectively caused hyperacetylation of histones, indicating its mechanism of action.
Phase I study of vorinostat in combination with temozolomide in patients with high-grade gliomas: North American Brain Tumor Consortium Study 04-03.Lee, EQ., Puduvalli, VK., Reid, JM., et al.[2021]

Citations

1.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/12805331/
Phase II evaluation of temozolomide and 13-cis-retinoic acid ...OS was 46% (95% CI, 36% to 57%) at 52 weeks and 21% (95% CI, 13% to 31%) at 104 weeks. Of 84 assessable patients, there were two (3%) complete responses and ...
Vorinostat, Isotretinoin and Temozolomide in Adults With ...The goal of this clinical research study is to learn if vorinostat when given with isotretinoin and temozolomide can help to control glioblastoma or gliosarcoma ...
3.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/25239666/
Randomized phase II adjuvant factorial study of dose-dense ...Conclusions: The results do not establish a benefit for these combinations but indicate that adding isotretinoin to ddTMZ may be detrimental.
Reused Drugs to Fight Glioblastoma (part one)Although accutane does not appear to improve results when added to the standard Temodar protocol, it appears to have activity as a single agent.
Emerging Therapies for GlioblastomaWhen both therapeutic agents were combined, the results surpassed the efficiency of each drug alone, showcasing a 35.6% ± 4.7% (TMZ 1 mM + CX-4945 30 μM) and ...
Vorinostat + Isotretinoin + Temozolomide for GlioblastomaThe combination of Vorinostat and Temozolomide has been studied for safety in patients with high-grade gliomas, and Temozolomide alone has been evaluated for ...
TEMODAR (temozolomide) Label - accessdata.fda.govTEMODAR® (temozolomide) is indicated for the treatment of adult patients with newly diagnosed glioblastoma multiforme concomitantly with radiotherapy and then ...
Study Details | NCT00112502 | Temozolomide Alone or in ...Thalidomide may stop the growth of glioblastoma multiforme by blocking blood flow to the tumor. Isotretinoin may help cells that are involved in the body's ...
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