Ruxolitinib Continuation for Cancer

Recruiting at 10 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

Trial Summary

What is the purpose of this trial?

The purpose of this study is to provide continued supply of ruxolitinib alone, ruxolitinib plus background cancer therapy, or background cancer therapy alone to subjects from an Incyte-sponsored study of ruxolitinib that has reached its study objectives or has been terminated. This study will also provide another mechanism for reporting adverse events related to study drug safety.

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 ruxolitinib generally safe for humans?

Ruxolitinib has been generally well tolerated in long-term studies for myelofibrosis, with manageable side effects like anemia (low red blood cell count) and thrombocytopenia (low platelet count). However, it has been associated with some rare skin reactions and an increased risk of infections, blood clots, and certain cancers. ^{1 2 3 4 5}

How does the drug ruxolitinib differ from other treatments for cancer?

Ruxolitinib is unique because it is a Janus kinase (JAK) 1 and 2 inhibitor, which means it works by blocking specific proteins involved in the growth of cancer cells. Unlike some other cancer treatments, it is taken orally and has shown benefits in conditions like myelofibrosis and polycythemia vera, where it helps reduce symptoms and improve survival rates. ^{4 6 7 8 9}

Research Team

Peter Langmuir, MD

Principal Investigator

Incyte Corporation

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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]

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]

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]

References

1. England pubmed.ncbi.nlm.nih.gov

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

2. United States 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. [2022]

3. Italy pubmed.ncbi.nlm.nih.gov

Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis: results of a median 3-year follow-up of COMFORT-I. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Off-label Studies on the Use of Ruxolitinib in Dermatology. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Adverse events associated with JAK inhibitors in 126,815 reports from the WHO pharmacovigilance database. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Mycobacterial Infections With Ruxolitinib: A Retrospective Pharmacovigilance Review. [2021]

8. New Zealand pubmed.ncbi.nlm.nih.gov

Ruxolitinib: A Review in Polycythaemia Vera. [2021]

9. England pubmed.ncbi.nlm.nih.gov

A case report of disseminated histoplasmosis and concurrent cryptococcal meningitis in a patient treated with ruxolitinib. [2022]