Trial Outcomes & Findings for Topical Ruxolitinib for Cutaneous Chronic Graft Versus Host Disease (cGVHD) (NCT NCT03395340)

Last Updated: 2022-07-12

Results Overview

The surface area will be measured by tracing the lesion on transparency paper and measuring the area from the transparency. Differences in remaining active surface area at 6 weeks from baseline between the treated and placebo sites were compared to calculate efficacy. A decrease in active surface area would signify improvement in skin disease.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

Baseline to week 6

Results posted on

2022-07-12

Participant Flow

Participant milestones

Participant milestones
Measure	Ruxolitinib Cream Investigational cream to 1 location; vehicle cream to 2nd location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Topical Ruxolitinib for Cutaneous Chronic Graft Versus Host Disease (cGVHD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ruxolitinib Cream n=1 Participants Investigational cream to 1 location; vehicle cream to 2nd location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	51.9 years STANDARD_DEVIATION 0 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 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	1 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	1 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to week 6

Outcome measures

Outcome measures
Measure	Ruxolitinib Treated Lesions n=1 Participants Investigational cream to 1 location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.	Placebo Treated Lesions n=1 Participants Vehicle cream to 2nd location
Percent Change in the Surface Area Measurement of the Target Lesions for Ruxolitinib Treated Versus Placebo Treated Lesions	-6.35 percent change	-3.81 percent change

PRIMARY outcome

Timeframe: date on study, up to approximately 2 months and 12 days.

Adverse events were measured by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Grade 3 is severe. Grade 4 is life-threatening.

Outcome measures

Outcome measures
Measure	Ruxolitinib Treated Lesions n=1 Participants Investigational cream to 1 location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.	Placebo Treated Lesions Vehicle cream to 2nd location
Number of Participants With Grade 3 and Grade 4 Adverse Events	0 Participants	—

SECONDARY outcome

Timeframe: Baseline and week 6

The Overall Severity VAS is a psychometric response questionnaire to measure subjective characteristics or attitudes that cannot be directly measured on a scale of 0-10; a participants assessment of degree of disease activity on the skin. 0=none and 10 = worst imaginable.

Outcome measures

Outcome measures
Measure	Ruxolitinib Treated Lesions n=1 Participants Investigational cream to 1 location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.	Placebo Treated Lesions n=1 Participants Vehicle cream to 2nd location
Change in Overall Severity Visual Analog Scale (VAS) Week 6	3.2 score on a scale	5.1 score on a scale
Change in Overall Severity Visual Analog Scale (VAS) Baseline	7.1 score on a scale	7.7 score on a scale

SECONDARY outcome

Timeframe: Baseline and week 6

The Pain VAS is a psychometric response questionnaire to measure subjective characteristics or attitudes that cannot be directly measured on a scale of 0 (no pain) -10 (worst imaginable pain). The participant draws a line on the scale representing how they feel between 0 (none) and 10 (worst). That line is measured and assigned a value. Scores from Baseline and week 6 will be reported. A change is considered a higher number (worsening of disease) or a lower number (improvement of disease) from baseline.

Outcome measures

Outcome measures
Measure	Ruxolitinib Treated Lesions n=1 Participants Investigational cream to 1 location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.	Placebo Treated Lesions n=1 Participants Vehicle cream to 2nd location
Change in Pain Visual Analog Scale (VAS) Baseline	0.3 score on a scale	0.3 score on a scale
Change in Pain Visual Analog Scale (VAS) Week 6	0.1 score on a scale	0.1 score on a scale

SECONDARY outcome

Timeframe: Baseline and week 6

The pruritus VAS is a psychometric response questionnaire to measure subjective characteristics or attitudes that cannot be directly measured on a scale of 0 (no itching) -10 (worst imaginable itching). Scores from Baseline and week 6 will be reported. A change is considered a higher number (worsening of disease) or a lower number (improvement of disease) from baseline.

Outcome measures

Outcome measures
Measure	Ruxolitinib Treated Lesions n=1 Participants Investigational cream to 1 location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.	Placebo Treated Lesions n=1 Participants Vehicle cream to 2nd location
Change in Pruritus Visual Analog Scale (VAS) Baseline	0.5 score on a scale	0.6 score on a scale
Change in Pruritus Visual Analog Scale (VAS) Week 6	0.1 score on a scale	0.1 score on a scale

SECONDARY outcome

Timeframe: A pre-dose trough sample will be collected prior to application of the treatment on the day of the follow up visit. After application in clinic, blood samples will be collected at 1 hour, 2 hour and 4 hours.

Population: This outcome measure was not done. Blood was collected for purposes of eventual pharmacokinetic (PK) and pharmacodynamic studies, including AUC but the assay was not developed because the study was not completed and samples were not run. There are no plans to develop the assay, no data was generated for this Outcome Measure.

The AUC is a measure of the serum concentration of the drug over time. It is used to characterize drug absorption.

Outcome measures

Outcome data not reported

SECONDARY outcome

Population: This outcome measure was not done. Blood was collected for purposes of eventual pharmacokinetic (PK) and pharmacodynamic studies, including total clearance but the assay was not developed because the study was not completed and samples were not run. There are no plans to develop the assay, no data was generated for this Outcome Measure.

The CL is a quantitative measure of the rate at which a drug substance is removed from the body.

Outcome measures

Outcome data not reported

SECONDARY outcome

Population: This outcome measure was not done. Blood was collected for purposes of eventual pharmacokinetic (PK) and pharmacodynamic studies, including half-life but the assay was not developed because the study was not completed and samples were not run. There are no plans to develop the assay, no data was generated for this Outcome Measure.

Plasma decay half-life is the time measured for the plasma concentration of the drug to decrease by one half.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Date treatment consent signed to date off study, approximately 2 months and 12 days.

Here is the number of participants with non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence.

Outcome measures

Outcome measures
Measure	Ruxolitinib Treated Lesions n=1 Participants Investigational cream to 1 location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.	Placebo Treated Lesions Vehicle cream to 2nd location
Number of Participants With Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)	1 Participants	—

Adverse Events

Ruxolitinib Cream

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Ruxolitinib Cream n=1 participants at risk Investigational cream to 1 location; vehicle cream to 2nd location Ruxolitinib 1.5% cream: Topical formulation of ruxolitinib, a Janus kinases (JAK) 1/2 inhibitor.
Respiratory, thoracic and mediastinal disorders Dyspnea	100.0% 1/1 • Number of events 1 • Date treatment consent signed to date off study, approximately 2 months and 12 days.
Renal and urinary disorders Urine discoloration	100.0% 1/1 • Number of events 1 • Date treatment consent signed to date off study, approximately 2 months and 12 days.

Additional Information

Dr. Edward W. Cowen

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Phone: 301-827-2328

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place