Ruxolitinib for Skin Cancer

Recruiting at 2 trial locations

Overseen By Research Nurse Navigator

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Columbia University

Must not be taking: Strong CYP3A4 drugs

Disqualifiers: Brain metastases, HIV, Hepatitis, 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

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

Compensation: Varies

Number of Visits: 3

Duration: 24 weeks

3 Participants Needed

Trial Summary

What is the purpose of this trial?

In this open-label, multicenter, Phase II study, the investigators propose to evaluate the efficacy of ruxolitinib, an orally administered inhibitor of JAK1/2, in solid organ transplant recipients with advanced cSCC. In a safety lead-in of 6 patients, subjects will receive ruxolitinib 15mg twice daily (BID). After 4 weeks, if dose-limiting toxicities (DLT) are observed in 1 or fewer patients, the study will enter stage 1 of the Simon two-stage design where all subsequent patients will receive a starting dose of ruxolitinib 15mg BID. If more than 1 DLTs are observed, another cohort of 6 patients will be treated at a dose of 10mg BID. If less than 2 DLTs are observed at the new dose of 10mg, then the study will proceed to stage I using this dose; otherwise the study will stop.

Research Team

Alexander Wei, MD

Principal Investigator

Associate Professor of Medicine at the Columbia University Medical Center

Eligibility Criteria

This trial is for adults over 18 with advanced skin cancer (cSCC) after a solid organ transplant. They must have measurable disease, be in fair health (KPS ≥60%, ECOG ≤2), and have recovered from previous treatments. A biopsy may be required, they can't be pregnant, must take oral meds well, haven't used JAK inhibitors before, and their organs must work properly.

Inclusion Criteria

I have had an organ transplant and am on immunosuppressants.

Negative pregnancy test for women of child bearing potential

I can take pills by mouth.

Exclusion Criteria

I have been treated with a JAK inhibitor before.

I am not HIV-positive or on antiretroviral therapy.

I haven't had major surgery in the last 4 weeks, except for procedures to place a vascular access device.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Safety Lead-in

In a safety lead-in of 6 patients, subjects will receive 15mg of ruxolitinib twice daily (BID) for 4 weeks to assess dose-limiting toxicities.

4 weeks

2 visits (in-person) on Day 1 and Day 15

Treatment

Participants receive ruxolitinib 15mg BID in a Simon two-stage design, with regular safety assessments.

24 weeks

1 visit (in-person) every cycle starting from cycle 3

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term Follow-up

Participants are monitored for progression-free survival and overall survival.

Up to 17 months

Treatment Details

Interventions

• Ruxolitinib

Trial Overview The study tests Ruxolitinib's effectiveness on cSCC in transplant recipients. Initially, patients get 15mg twice daily; if side effects are manageable, the dose continues or reduces to 10mg based on early results. It's an open-label Phase II trial using Simon two-stage design to adjust dosing.

Participant Groups

Group I: Ruxolitinib Experimental Treatment 1 Intervention

In a safety lead-in of 6 patients, subjects will receive 15mg of ruxolitinib twice daily (BID). After 4 weeks, if dose-limiting toxicities (DLT) are observed in 1 or fewer patients, the study will enter stage 1 of the Simon two-stage design where all subsequent patients will receive a starting dose of ruxolitinib 15mg BID. Subjects will have regularly scheduled study visits at the clinical site on Day 1 and Day 15 (± 3 days) of the first 2 cycles, then on Day 1 (± 3 days) of every subsequent cycle (starting cycle 3), where safety assessments, including laboratory assessments, vital signs, and physical examinations will be performed.

Ruxolitinib is already approved in United States, European Union for the following indications:

Approved in United States as Jakafi for:

• Intermediate or high-risk myelofibrosis
• Polycythemia vera
• Steroid-refractory acute graft-versus-host disease
• Chronic graft-versus-host disease
• Vitiligo

Approved in European Union as Jakavi for:

• Intermediate or high-risk myelofibrosis
• Polycythemia vera
• Steroid-refractory acute graft-versus-host disease
• Chronic graft-versus-host disease
• Non-segmental vitiligo

Who Is Running the Clinical Trial?

Columbia University

Lead Sponsor

Trials

1,529

Recruited

2,832,000+

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