Trial Outcomes & Findings for Study of Ruxolitinib in Solid Organ Transplant Recipients With Advanced Cutaneous Squamous Cell Carcinoma (NCT NCT04807777)

Last Updated: 2024-12-27

Results Overview

The primary endpoint is the overall response rate as defined as the best response, confirmed at ≥4 weeks using RECIST v1.1 criteria. Responses defined as: Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes must have reduction in short axis to \<10 mm. Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Progressive Disease (PD): At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. (Note: the appearance of one or more new lesions is also considered progressions). Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

within the first 24 weeks of the start of study therapy

Results posted on

2024-12-27

Participant Flow

Participant milestones

Participant milestones
Measure	Ruxolitinib 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: Ruxolitinib will be administered orally twice daily during the entirety of each 28-day cycle.
Safety Lead-in: Ruxolitinib 15mg STARTED	3
Safety Lead-in: Ruxolitinib 15mg COMPLETED	3
Safety Lead-in: Ruxolitinib 15mg NOT COMPLETED	0
Stage 1 STARTED	0
Stage 1 COMPLETED	0
Stage 1 NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Ruxolitinib in Solid Organ Transplant Recipients With Advanced Cutaneous Squamous Cell Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ruxolitinib n=3 Participants 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: Ruxolitinib will be administered orally twice daily during the entirety of each 28-day cycle.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: within the first 24 weeks of the start of study therapy

Outcome measures

Outcome measures
Measure	Ruxolitinib n=3 Participants 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: Ruxolitinib will be administered orally twice daily during the entirety of each 28-day cycle.
Overall Response Rate (ORR) Complete Response	0 Participants
Overall Response Rate (ORR) Partial Response	0 Participants
Overall Response Rate (ORR) Stable Disease	1 Participants
Overall Response Rate (ORR) Progressive Disease	2 Participants

SECONDARY outcome

Timeframe: Up to 14 months

Progression-free survival (PFS) (time alive without advanced cutaneous squamous cell carcinoma (cSCC) probabilities will be estimated

Outcome measures

Outcome measures
Measure	Ruxolitinib n=3 Participants 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: Ruxolitinib will be administered orally twice daily during the entirety of each 28-day cycle.
Progression-Free Survival (PFS)	46.67 Days Interval 28.0 to 56.0

SECONDARY outcome

Timeframe: Up to17 months

The length of time from the start of treatment that subjects with the disease are still alive.

Outcome measures

Outcome measures
Measure	Ruxolitinib n=3 Participants 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: Ruxolitinib will be administered orally twice daily during the entirety of each 28-day cycle.
Overall Survival (OS)	290.67 Days Interval 160.0 to 506.0

Adverse Events

Ruxolitinib

Serious events: 0 serious events

Other events: 2 other events

Deaths: 3 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Ruxolitinib n=3 participants at risk 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: Ruxolitinib will be administered orally twice daily during the entirety of each 28-day cycle.
Blood and lymphatic system disorders Anemia	33.3% 1/3 • Up to 17 months Systematic collection of adverse events on Day 1 and Day 15 (± 3 days) of the first cycle, then on Day 1 (± 3 days) of every subsequent cycle (starting cycle 2).
Infections and infestations Lung Infection	33.3% 1/3 • Up to 17 months Systematic collection of adverse events on Day 1 and Day 15 (± 3 days) of the first cycle, then on Day 1 (± 3 days) of every subsequent cycle (starting cycle 2).
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumor Pain	33.3% 1/3 • Up to 17 months Systematic collection of adverse events on Day 1 and Day 15 (± 3 days) of the first cycle, then on Day 1 (± 3 days) of every subsequent cycle (starting cycle 2).
General disorders Fatigue	33.3% 1/3 • Up to 17 months Systematic collection of adverse events on Day 1 and Day 15 (± 3 days) of the first cycle, then on Day 1 (± 3 days) of every subsequent cycle (starting cycle 2).
General disorders Flu like symptoms	33.3% 1/3 • Up to 17 months Systematic collection of adverse events on Day 1 and Day 15 (± 3 days) of the first cycle, then on Day 1 (± 3 days) of every subsequent cycle (starting cycle 2).

Additional Information

Alexander Wei, MD

Columbia University

Phone: 212-305-7115

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Neither the complete nor any part of the results of the study carried out under this protocol, nor any of the information provided by the sponsor for the purposes of performing the study, will be published or passed on to any third party without the consent of the study sponsor-investigator. Any investigator involved with this study is obligated to provide the sponsor-investigator with complete test results and all data derived from the study.
Publication restrictions are in place

Restriction type: OTHER