Ruxolitinib Continuation for Cancer

Recruiting at 11 trial locations

Overseen By Incyte Corporation Call Center

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Incyte Corporation

Must be taking: Ruxolitinib

Disqualifiers: Discontinued treatment, Access outside study

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 24 months

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on continuing treatment with the drug ruxolitinib for cancer patients. Researchers aim to evaluate patient outcomes when using ruxolitinib alone, in combination with other cancer treatments, or those treatments alone. The trial also monitors any side effects. It suits individuals currently benefiting from ruxolitinib in a previous study and experiencing stable disease results. Participants must still be part of an earlier Incyte-sponsored study and unable to access the drug outside the trial. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, it seems you can continue with your background cancer therapy if you are benefiting from it.

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

Research has shown that ruxolitinib is generally well-tolerated by patients. Studies indicate that minor side effects occur less frequently with long-term use of ruxolitinib compared to other treatments, and serious blood clots are also less common. Over ten years of use, no new safety concerns have emerged. One study found that cases of skin cancer are rare, occurring at a rate of about 0.46 per 100 patient-years.

When combined with other cancer treatments, ruxolitinib maintains consistent safety, causing no unexpected side effects. These findings suggest that ruxolitinib, whether used alone or with other treatments, has a strong safety record, making it a promising option for ongoing cancer treatment. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about ruxolitinib for cancer treatment because it targets the Janus kinase (JAK) pathway, which is different from traditional chemotherapy that primarily targets rapidly dividing cells. This unique mechanism can potentially reduce the side effects typically associated with chemotherapy. Additionally, combining ruxolitinib with existing cancer therapies could enhance their effectiveness, offering a more comprehensive approach to treatment. These features make ruxolitinib a promising option for improving outcomes in cancer care.

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

Research has shown that ruxolitinib may help treat certain cancers. In this trial, participants may receive ruxolitinib alone or with background cancer therapy. Studies have found that ruxolitinib can improve survival rates and manage symptoms like an enlarged spleen. Specifically, patients taking ruxolitinib had a better chance of living for at least one year compared to those who did not take it. When combined with other cancer treatments, ruxolitinib effectively manages blood-related symptoms and reduces spleen size. The treatment has consistently proven safe and effective, offering hope for long-term benefits in managing cancer symptoms. ^{1 2 3 6 7}

Who Is on the Research Team?

Peter Langmuir, MD

Principal Investigator

Incyte Corporation

Are You a Good Fit for This Trial?

This trial is for patients already in an Incyte-sponsored ruxolitinib study that's ended or stopped. They should have stable disease, be tolerating and benefiting from their current treatment (ruxolitinib alone, with other cancer drugs, or just the cancer drugs), and must have followed the original study rules.

Inclusion Criteria

Has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements

I am currently seeing positive effects from my cancer treatment, as confirmed by my doctor.

My condition is not worsening according to my doctor.

Exclusion Criteria

I can get ruxolitinib or other cancer treatments not provided by the study.

Has been permanently discontinued from study treatment in the parent study for any reason

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants continue receiving ruxolitinib, ruxolitinib plus background cancer therapy, or background cancer therapy alone as per the parent study

Up to 24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

30-37 days

What Are the Treatments Tested in This Trial?

Interventions

• Ruxolitinib

Trial Overview The trial provides ongoing access to ruxolitinib for those who were part of previous studies. It examines continued use of ruxolitinib by itself, combined with standard cancer treatments like Capecitabine and Regorafenib, or these treatments on their own.

How Is the Trial Designed?

Group I: Ruxolitinib plus background cancer therapy Experimental Treatment 3 Interventions

Study treatment for participants should be the same as the dosage from the parent study at the time the roll over protocol is initiated. Dose modifications are permitted.

Group II: Ruxolitinib Experimental Treatment 1 Intervention

Study treatment for participants should be the same as the dosage from the parent study at the time the roll over protocol is initiated. Dose modifications are permitted.

Group III: Background cancer therapy alone Experimental Treatment 2 Interventions

Capecitabine and Regorafenib at the same dose provided in the parent study at the time of the rollover.

Who Is Running the Clinical Trial?

Incyte Corporation

Lead 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

Published Research Related to This Trial

Ruxolitinib, a JAK-2 inhibitor used for treating myeloproliferative neoplasms, is generally associated with very rare skin toxicity, but an unusual case of erythematous skin eruption with necrotic centers was observed in a patient with primary myelofibrosis.

This case highlights the need for increased awareness of potential skin side effects from ruxolitinib, especially as JAK-2 inhibitors may be used more widely in dermatological treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Erythematous skin lesions with necrotic centers on lower extremities due to the use of ruxolitinib for primary myelofibrosis. Dasanu, CA.[2021]

A study analyzing 126,815 safety reports found that the JAK inhibitors ruxolitinib, tofacitinib, and baricitinib are associated with increased risks of infectious adverse events, particularly viral, fungal, and mycobacterial infections.

Tofacitinib specifically showed a significant association with gastrointestinal perforation events, while no significant increase in major cardiovascular events was observed, indicating a need for careful monitoring of these risks in patients using JAK inhibitors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events associated with JAK inhibitors in 126,815 reports from the WHO pharmacovigilance database. Hoisnard, L., Lebrun-Vignes, B., Maury, S., et al.[2023]

Ruxolitinib, a Janus kinase inhibitor approved for myelofibrosis and polycythemia vera, shows promising efficacy in treating dermatologic conditions like atopic dermatitis, psoriasis, and vitiligo, based on data from clinical trials.

The evidence for ruxolitinib's effectiveness in alopecia areata is mixed, varying between topical and oral formulations, indicating the need for further research to clarify its role in treating various skin disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Off-label Studies on the Use of Ruxolitinib in Dermatology. Tegtmeyer, K., Ravi, M., Zhao, J., et al.[2021]

Patients treated with ruxolitinib, a JAK inhibitor for myelofibrosis and polycythemia vera, have a significantly increased risk of developing Mycobacterium tuberculosis (MTB) and atypical mycobacterial infections (AMI), with reporting odds ratios of 9.2 and 8.3, respectively.

Out of the reported cases, 13.2% of patients with MTB and 34.8% with AMI died, highlighting the importance of screening for latent MTB before starting ruxolitinib treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mycobacterial Infections With Ruxolitinib: A Retrospective Pharmacovigilance Review. Anand, K., Burns, EA., Ensor, J., et al.[2021]

Ruxolitinib is an effective treatment for patients with polycythaemia vera (PV) who do not respond well to hydroxyurea, showing superior results in reducing hematocrit and spleen size after 32 weeks in the phase III RESPONSE trial.

The drug is generally well tolerated, with manageable side effects like mild to moderate anemia and thrombocytopenia, making it a valuable option for PV patients with limited treatment alternatives.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ruxolitinib: A Review in Polycythaemia Vera. McKeage, K.[2021]

In a 3-year follow-up of the COMFORT-II Trial involving 219 patients with myelofibrosis, ruxolitinib demonstrated sustained reductions in spleen size and improved quality of life, with 45% of patients remaining on treatment after 3 years.

Ruxolitinib was well tolerated, with manageable side effects like anemia and thrombocytopenia, and it was associated with longer overall survival compared to the best available therapy, indicating its efficacy and safety as a treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Cervantes, F., Vannucchi, AM., Kiladjian, JJ., et al.[2022]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4380721/

Efficacy, safety, and survival with ruxolitinib in patients with ... This planned analysis assessed the long-term efficacy and safety of ruxolitinib at a median follow-up of 149 weeks. At data cutoff, approximately 50% of ...

2. 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 ...

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.22.01935

Ruxolitinib Versus Best Available Therapy for Polycythemia ... Thromboembolic event-free, but not hemorrhage-free, survival was significantly improved with ruxolitinib (hazard ratio [HR], 0.56; 95% CI, 0.32 ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8938906/

Long-term efficacy and safety of ruxolitinib versus best ... The long-term follow-up in RESPONSE showed that ruxolitinib provided durable haematocrit control, spleen volume reduction, complete haematological remission, ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S2152265024024650

A Review of Real-World Experience With Ruxolitinib for ... Consistent with these findings, 1-year survival rates in the ruxolitinib-exposed group (82.3%) were higher than in the unexposed group (72.5%). Another ...

6. hcp.jakafi.com hcp.jakafi.com/acute-graft-versus-host-disease/safety-adverse-events

Safety Profile of Jakafi in aGVHD Safety results for Jakafi ® (ruxolitinib) in REACH1 · Nonhematologic Adverse Reactions Occurring in ≥15% of Patients · Selected Laboratory Abnormalities Worsening ...

7. jhoonline.biomedcentral.com jhoonline.biomedcentral.com/articles/10.1186/s13045-023-01471-z

Ten years of treatment with ruxolitinib for myelofibrosis Ruxolitinib has been shown to not only improve splenomegaly and the burdensome symptoms associated with MF but also to improve overall survival (OS).