Ruxolitinib + Standard Treatment for Brain Cancer

Overseen By Manmeet Ahluwalia, M.D., MBA

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Baptist Health South Florida

Must not be taking: Warfarin

Disqualifiers: Pregnancy, Active infections, Cardiovascular disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new combination treatment for individuals recently diagnosed with glioblastoma, a type of brain cancer. Researchers aim to determine if adding the drug ruxolitinib to standard treatments, such as radiation and temozolomide (a chemotherapy drug), can be safe and more effective. Participants will receive either the standard treatment with or without ruxolitinib, assigned randomly. Ideal candidates have a confirmed glioblastoma diagnosis and require some assistance with daily activities. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are using warfarin or receiving other treatments for your brain tumor. It's best to discuss your current medications with the study team to see if they might affect your eligibility.

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

Research has shown that ruxolitinib, when combined with radiation and temozolomide, is generally well-tolerated by patients. One study found that taking 20 mg of ruxolitinib twice a day is safe alongside these treatments and demonstrated promising survival rates compared to past results. Ruxolitinib has been used for over ten years for other conditions, and its safety is well-established. Most people can take it with or without food, and it leaves the body quickly, reducing the risk of long-lasting side effects.

Overall, evidence suggests that ruxolitinib is generally safe when used with standard brain cancer treatments like radiation and temozolomide. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for brain cancer?

Ruxolitinib is unique because it works by targeting the JAK-STAT signaling pathway, which is often overactive in brain cancer cells. This is different from the standard treatments like temozolomide and radiation, which primarily focus on damaging the DNA of cancer cells. By specifically inhibiting this pathway, ruxolitinib may help to slow down or stop the growth of cancer cells more effectively. Researchers are excited because this targeted approach could potentially improve outcomes for patients with brain cancer, offering a new avenue of hope beyond traditional methods.

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

This trial will compare the effectiveness of adding ruxolitinib to the standard glioblastoma treatment, which includes radiation and temozolomide. Research has shown that patients receiving ruxolitinib with radiation and temozolomide lived longer without cancer progression and had longer overall survival compared to those receiving only the standard treatment. This combination has also proven safe and manageable for patients. These findings offer hope that ruxolitinib could enhance the effectiveness of current glioblastoma treatments. ^{6 7 8 9 10}

Who Is on the Research Team?

Manmeet Ahluwalia, M.D., MBA

Principal Investigator

Miami Cancer Institute at Baptist Health, Inc.

Are You a Good Fit for This Trial?

This trial is for individuals newly diagnosed with glioblastoma, a type of brain cancer. Participants should not have received prior treatments for this condition. Specific eligibility criteria are not provided, but typically include factors like age, overall health status, and the stage of the tumor.

Inclusion Criteria

Provision of signed informed consent form

My liver, kidneys, and bone marrow are functioning well.

I can take care of myself but sometimes need help.

Exclusion Criteria

I am not pregnant or breast-feeding.

Any other serious medical/psychiatric condition, in the judgement of the investigator, that likely to interfere or limit compliance with study requirements/treatment

I have taken ruxolitinib before or am allergic to the study drug.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Radiation and Temozolomide

Participants receive radiation therapy and temozolomide, with or without ruxolitinib, during this phase

6 weeks

Maintenance

Participants continue with temozolomide, with or without ruxolitinib, after radiation therapy is complete

6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 years

What Are the Treatments Tested in This Trial?

Interventions

• Radiation Therapy
• Ruxolitinib
• Temozolomide

Trial Overview The study is testing the safety and effectiveness of ruxolitinib combined with standard care (radiation therapy and temozolomide) versus standard care alone in treating glioblastoma. Participants will be randomly assigned to one of these two groups in equal numbers.

How Is the Trial Designed?

Group I: Ruxolitinib plus Radiation and Temozolomide Experimental Treatment 3 Interventions

There will be two phases of drug administration: (1) Ruxolitinib and temozolomide will be administered "During Radiation Therapy" and (2) Maintenance phase after radiation therapy is complete.

Group II: Radiation and Temozolomide Active Control 2 Interventions

There will be two phases of drug administration: (1) Temozolomide will be administered "During Radiation Therapy" and (2) Maintenance phase after radiation therapy is complete.

Who Is Running the Clinical Trial?

Baptist Health South Florida

Lead Sponsor

Trials

54

Recruited

8,100+

Incyte Corporation

Industry Sponsor

Trials

408

Recruited

66,800+

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06991101

Study Details | NCT06991101 | Ruxolitinib With Radiation ... Randomized Phase 2 Trial of Ruxolitinib in Combination With Radiation and Temozolomide Compared to Radiation and Temozolomide for Newly Diagnosed Glioblastoma.

2. ascopubs.org ascopubs.org/doi/10.1200/JCO.2021.39.15_suppl.2060

Phase I study of ruxolitinib with radiation and ... Patients that received ruxolitinib + radiation x 60 Gy + daily temozolomide at 75 mg/m2 for 6 weeks over 6 weeks (Arm 2) had significantly better PFS and OS.

3. targetedonc.com targetedonc.com/view/ruxolitinib-plus-radiation-and-temozolomide-is-safe-and-feasible-for-high-grade-glioma

Ruxolitinib Plus Radiation and Temozolomide is Safe and ... The use of ruxolitinib (Jakafi) in combination with temozolomide and radiation therapy appears to be a safe and feasible option.

4. trials.braintumor.org trials.braintumor.org/trials/NCT06991101

Ruxolitinib With Radiation and Temozolomide Compared to ... The purpose of this research is to test the safety and effectiveness of the investigational drug ruxolitinib when it is combined with standard of care treatment ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7650934/

CTNI-41. PHASE I STUDY OF RUXOLITINIB WITH ... Patients with methylated MGMT glioblastoma or grade III glioma were eligible for Arm 2. Every patient received ruxolitinib + radiation x 60 Gy + daily ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06991101

Study Details | NCT06991101 | Ruxolitinib With Radiation ... The purpose of this research is to test the safety and effectiveness of the investigational drug ruxolitinib when it is combined with standard of care ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4920055/

Efficacy and Safety of Ruxolitinib in the Treatment of Patients ... Overall, the data indicate that ruxolitinib may be taken with or without food. Ruxolitinib has a short terminal half-life of approximately 3 h for doses up ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S1567576925006198

Ruxolitinib suppresses tumor growth in PTEN-deficient ... According to data from The Cancer Genome Atlas (TCGA), over 80 % of GBM cases exhibit PTEN homozygous or hemizygous deletion, making it a ...

9. ascopubs.org ascopubs.org/doi/10.1200/JCO.2022.40.16_suppl.2061

Phase 1 trial of ruxolitinib, temozolomide, and radiation in ... Dose of 20 mg twice daily of ruxolitinib is safe with radiation and temozolomide. Preliminary survival data appears promising compared to the historical ...

10. acsjournals.onlinelibrary.wiley.com acsjournals.onlinelibrary.wiley.com/doi/10.1002/cncr.35661

Ten years of experience with ruxolitinib since approval for ... This review covers ruxolitinib efficacy and safety data from clinical trials and real-world settings in the decade since ruxolitinib was ...