Trial Outcomes & Findings for Evaluation of Tofacitinib in Prevention of Photosensitivity in Lupus (NCT NCT05048238)

NCT ID: NCT05048238

Last Updated: 2025-08-22

Results Overview

At Day 0 and Day 25, skin was exposed to 10-80 mJ/cm2 UVB irradiation. At Day 1 and Day 26, erythema levels were measured with a Chroma Meter at each dose site and in an unexposed section. Skin biopsies at Day 1 and Day 26 were taken at the unexposed site and at the minimal erythema dose (MED) at Day 1. The percentage of apoptotic epidermal cells were determined in each skin biopsy sample by TUNEL staining. The percentage of UVB-induced apoptotic cells is defined as the difference between the percentage of apoptotic epidermal cells in the UVB-exposed biopsy at the MED and percentage of apoptotic epidermal cells in the unexposed biopsy at the same visit. Change in percentage of UVB-induced apoptotic epidermal cells is calculated as the difference in the percentage of UVB-induced apoptotic cells at Day 26 and at Day 1.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

7 participants

Primary outcome timeframe

Day 1 (pre-treatment) to Day 26

Results posted on

2025-08-22

Participant Flow

Two study sites were activated in the United States, beginning in March 2022. A total of eight participants were screened from September 2022 to January 2024 at both sites. Of those screened seven participants enrolled in the study.

Participant milestones

Participant milestones
Measure
Tofacitinib
25-day regimen of tofacitinib 11 mg daily
Overall Study
STARTED
7
Overall Study
COMPLETED
6
Overall Study
NOT COMPLETED
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Tofacitinib
25-day regimen of tofacitinib 11 mg daily
Overall Study
Withdrawal by Subject
1

Baseline Characteristics

Evaluation of Tofacitinib in Prevention of Photosensitivity in Lupus

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Tofacitinib
n=7 Participants
25-day regimen of tofacitinib 11 mg daily
Age, Continuous
42.7 years
STANDARD_DEVIATION 6.26 • n=5 Participants
Sex: Female, Male
Female
6 Participants
n=5 Participants
Sex: Female, Male
Male
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
2 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
5 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
1 Participants
n=5 Participants
Race (NIH/OMB)
White
6 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
7 participants
n=5 Participants
Diagnosed with Systemic Lupus Erythematosus (SLE)
Yes
3 Participants
n=5 Participants
Diagnosed with Systemic Lupus Erythematosus (SLE)
No
4 Participants
n=5 Participants
Fitzpatrick Scale
Type I
1 Participants
n=5 Participants
Fitzpatrick Scale
Type II
2 Participants
n=5 Participants
Fitzpatrick Scale
Type III
1 Participants
n=5 Participants
Fitzpatrick Scale
Type IV
2 Participants
n=5 Participants
Fitzpatrick Scale
Type V
1 Participants
n=5 Participants
Fitzpatrick Scale
Type VI
0 Participants
n=5 Participants
SLEDAI-2K Score (Clinical Criteria Only)
0.9 SLEDAI-2K Score
STANDARD_DEVIATION 1.07 • n=5 Participants
CLASI Total Activity (CLASI-A) Score
2.4 CLASI-A Score
STANDARD_DEVIATION 3.87 • n=5 Participants
CLASI Total Damage (CLASI-D) Score
2.9 CLASI-D Score
STANDARD_DEVIATION 5.40 • n=5 Participants

PRIMARY outcome

Timeframe: Day 1 (pre-treatment) to Day 26

Population: The Per Protocol 1 (PP1) population includes all participants who received any amount of tofacitinib and who completed phototests and skin biopsies at both baseline (Visits 1 and 2) and post-treatment (Visits 4 and 5) time points. Only individuals who completed at least 21 days of IP dosing had the post-treatment phototest.

At Day 0 and Day 25, skin was exposed to 10-80 mJ/cm2 UVB irradiation. At Day 1 and Day 26, erythema levels were measured with a Chroma Meter at each dose site and in an unexposed section. Skin biopsies at Day 1 and Day 26 were taken at the unexposed site and at the minimal erythema dose (MED) at Day 1. The percentage of apoptotic epidermal cells were determined in each skin biopsy sample by TUNEL staining. The percentage of UVB-induced apoptotic cells is defined as the difference between the percentage of apoptotic epidermal cells in the UVB-exposed biopsy at the MED and percentage of apoptotic epidermal cells in the unexposed biopsy at the same visit. Change in percentage of UVB-induced apoptotic epidermal cells is calculated as the difference in the percentage of UVB-induced apoptotic cells at Day 26 and at Day 1.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=6 Participants
25-day regimen of tofacitinib 11 mg daily
Change in Percentage of UVB-induced Apoptotic Epidermal Cells
Day 1
20.06 percentage of epidermal cells
Standard Deviation 9.587
Change in Percentage of UVB-induced Apoptotic Epidermal Cells
Day 26
3.03 percentage of epidermal cells
Standard Deviation 2.349
Change in Percentage of UVB-induced Apoptotic Epidermal Cells
Change from Day 1 to Day 26
-17.03 percentage of epidermal cells
Standard Deviation 8.355

PRIMARY outcome

Timeframe: Day 1 (pre-treatment) to Day 26

Population: The Per Protocol 2 (PP2) population includes all PP1 participants with no protocol deviations that would impact photo sensitivity assessments.

At Day 0 and Day 25, skin was exposed to 10-80 mJ/cm2 UVB irradiation. At Day 1 and Day 26, erythema levels were measured with a Chroma Meter at each dose site and in an unexposed section. Skin biopsies at Day 1 and Day 26 were taken at the unexposed site and at the minimal erythema dose (MED) at Day 1. The percentage of apoptotic epidermal cells were determined in each skin biopsy sample by TUNEL staining. The percentage of UVB-induced apoptotic cells is defined as the difference between the percentage of apoptotic epidermal cells in the UVB-exposed biopsy at the MED and percentage of apoptotic epidermal cells in the unexposed biopsy at the same visit. Change in percentage of UVB-induced apoptotic epidermal cells is calculated as the difference in the percentage of UVB-induced apoptotic cells at Day 26 and at Day 1.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=3 Participants
25-day regimen of tofacitinib 11 mg daily
Change in Percentage of UVB-induced Apoptotic Epidermal Cells
Day 1
21.51 percentage of epidermal cells
Standard Deviation 12.334
Change in Percentage of UVB-induced Apoptotic Epidermal Cells
Day 26
3.45 percentage of epidermal cells
Standard Deviation 2.976
Change in Percentage of UVB-induced Apoptotic Epidermal Cells
Change from Day 1 to Day 26
-18.06 percentage of epidermal cells
Standard Deviation 11.182

SECONDARY outcome

Timeframe: Day 1 (pre-treatment) to Day 26

Population: The Per Protocol 1 (PP1) population includes all participants who received any amount of tofacitinib and who completed phototests and skin biopsies at both baseline (Visits 1 and 2) and post-treatment (Visits 4 and 5) time points. Only individuals who completed at least 21 days of IP dosing had the post-treatment phototest.

At Day 0 and Day 25, skin was exposed to 10-80 mJ/cm2 UVB irradiation. At Day 1 and Day 26, erythema levels were measured with a Chroma Meter at each dose site and in an unexposed section. The MED was defined as the dose that contained the lowest UVB exposure and the average redness reading that was at least 2.5 higher than the average redness reading in the unexposed skin. The possible values for MED ranged from 10 to 80 mJ/cm2.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=6 Participants
25-day regimen of tofacitinib 11 mg daily
Change in Minimal Erythema Dose (MED) Due to UVB
+30 mJ/cm2
0 Participants
Change in Minimal Erythema Dose (MED) Due to UVB
+20 mJ/cm2
0 Participants
Change in Minimal Erythema Dose (MED) Due to UVB
+10 mJ/cm2
1 Participants
Change in Minimal Erythema Dose (MED) Due to UVB
Stayed the same
3 Participants
Change in Minimal Erythema Dose (MED) Due to UVB
-10 mJ/cm2
0 Participants
Change in Minimal Erythema Dose (MED) Due to UVB
-20 mJ/cm2
1 Participants
Change in Minimal Erythema Dose (MED) Due to UVB
-30 mJ/cm2
1 Participants

SECONDARY outcome

Timeframe: Day 1 (pre-treatment) to Day 26

Population: The Per Protocol 1 (PP1) population includes all participants who received any amount of tofacitinib and who completed phototests and skin biopsies at both baseline (Visits 1 and 2) and post-treatment (Visits 4 and 5) time points. Only individuals who completed at least 21 days of IP dosing had the post-treatment phototest.

Skin biopsies at Day 1 and Day 26 were taken at the unexposed site and at the site corresponding to the next higher UVB dose than the Day 26 MED. RNA was isolated and expression values were analyzed by RNA sequencing. UVB-induced expression is defined as the difference in expression measured in the biopsy exposed at the Visit 2 (Day 1) 1x minimal erythema dose (MED) and that measured in the unexposed biopsy.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=6 Participants
25-day regimen of tofacitinib 11 mg daily
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
TNF at Day 1
-5.5 Count of RNA transcripts
Standard Deviation 10.13
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
TNF at Day 26
-0.8 Count of RNA transcripts
Standard Deviation 5.85
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
TNF Change from Day 1 to Day 26
4.7 Count of RNA transcripts
Standard Deviation 6.50
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IL1B at Day 1
38.5 Count of RNA transcripts
Standard Deviation 42.57
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IL1B at Day 26
4.8 Count of RNA transcripts
Standard Deviation 20.82
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IL1B Change from Day 1 to Day 26
-33.7 Count of RNA transcripts
Standard Deviation 44.85
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
CXCL9 at Day 1
53.8 Count of RNA transcripts
Standard Deviation 63.35
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
CXCL9 at Day 26
133.7 Count of RNA transcripts
Standard Deviation 246.27
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
CXCL9 Change from Day 1 to Day 26
79.8 Count of RNA transcripts
Standard Deviation 289.29
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IL6 at Day 1
-0.7 Count of RNA transcripts
Standard Deviation 5.24
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IL6 at Day 26
10.5 Count of RNA transcripts
Standard Deviation 25.74
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IL6 Change from Day 1 to Day 26
11.2 Count of RNA transcripts
Standard Deviation 25.33
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IFNB1 at Day 1
0.0 Count of RNA transcripts
Standard Deviation 0.00
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IFNB1 at Day 26
0.00 Count of RNA transcripts
Standard Deviation 0.00
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IFNB1 Change from Day 1 to Day 26
0.0 Count of RNA transcripts
Standard Deviation 0.00
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IFI44 at Day 1
10.2 Count of RNA transcripts
Standard Deviation 54.62
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IFI44 at Day 26
109.5 Count of RNA transcripts
Standard Deviation 137.50
Change in UVB-induced Expression of Inflammatory Genes in Skin Based on Enumeration of RNA Transcripts.
IFI44 Change from Day 1 to Day 26
99.3 Count of RNA transcripts
Standard Deviation 107.08

SECONDARY outcome

Timeframe: Day 0 (pre-treatment) to Day 25

Population: The Per Protocol 1 (PP1) population includes all participants who received any amount of tofacitinib and who completed phototests and skin biopsies at both baseline (Visits 1 and 2) and post-treatment (Visits 4 and 5) time points. Only individuals who complete at least 21 days of IP dosing had the post-treatment phototest.

CLASI is a validated, physician-based assessment tool used to measure cutaneous lupus severity. Severity is calculated based on disease activity (erythema and scale) and damage (dyspigmentation and scarring) for the cumulative areas of involved skin. CLASI activity score ranges from 0 to 70, with higher scores indicating increased disease severity.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=6 Participants
25-day regimen of tofacitinib 11 mg daily
Change in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Day 0
2.8 CLASI-A Score
Standard Deviation 4.07
Change in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Day 25
2.3 CLASI-A Score
Standard Deviation 3.27
Change in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Change from Day 0 to Day 25
-0.5 CLASI-A Score
Standard Deviation 1.22

SECONDARY outcome

Timeframe: Day 0 (pre-treatment) to Day 25

Population: The Per Protocol 1 (PP1) population includes all participants who received any amount of tofacitinib and who completed phototests and skin biopsies at both baseline (Visits 1 and 2) and post-treatment (Visits 4 and 5) time points. Only individuals who completed at least 21 days of IP dosing had the post-treatment phototest.

CLASI is a validated, physician-based assessment tool used to measure cutaneous lupus severity. Damage is calculated based on dyspigmentation and scarring for the cumulative areas of involved skin. The CLASI damage score ranges from 0 to 56, with higher scores indicating worse damage to the skin due to lupus.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=6 Participants
25-day regimen of tofacitinib 11 mg daily
Change in CLASI Damage Score
Day 0
3.3 CLASI-D Score
Standard Deviation 5.75
Change in CLASI Damage Score
Day 25
2.8 CLASI-D Score
Standard Deviation 4.67
Change in CLASI Damage Score
Change to Day 25
-0.5 CLASI-D Score
Standard Deviation 1.22

SECONDARY outcome

Timeframe: Day 0 (pre-treatment) to Day 25

Population: The Per Protocol 1 (PP1) population includes all participants who received any amount of tofacitinib and who completed phototests and skin biopsies at both baseline (Visits 1 and 2) and post-treatment (Visits 4 and 5) time points. Only individuals who completed at least 21 days of IP dosing had the post-treatment phototest.

Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) is a validated, physician-based assessment tool used to measure the extent of lupus disease activity within the past 30 days. SLEDAI-2K total score is a weighted sum of the presence of 24 lupus disease symptoms and ranges from 0 to 105, with higher scores indicating more lupus disease activity present.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=6 Participants
25-day regimen of tofacitinib 11 mg daily
Change in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) Total Score
Day 0
1.3 SLEDAI-2K Score
Standard Deviation 1.63
Change in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) Total Score
Day 25
1.7 SLEDAI-2K Score
Standard Deviation 1.97
Change in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) Total Score
Change to Day 25
0.3 SLEDAI-2K Score
Standard Deviation 0.82

SECONDARY outcome

Timeframe: Screening Visit through Day 40

Population: The Safety Sample (SS) population includes all participants who received any amount of tofacitinib.

The study sites graded the severity of laboratory results for the study participants according to the criteria set forth in the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) 5.0. The NCI-CTCAE manual provides a common language to describe levels of severity, to analyze and interpret data, and to articulate the clinical significance of adverse events. Select laboratory results are incorporated into relevant adverse event criteria.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=7 Participants
25-day regimen of tofacitinib 11 mg daily
Change in Severity Grade for Laboratory Tests From Grade 0
Alanine Aminotransferase: Increase by 1 grade
1 participants
Change in Severity Grade for Laboratory Tests From Grade 0
Alanine Aminotransferase: Increase by 2+ grades
0 participants
Change in Severity Grade for Laboratory Tests From Grade 0
Aspartate Aminotransferase: Increase by 1 grade
1 participants
Change in Severity Grade for Laboratory Tests From Grade 0
Aspartate Aminotransferase: Increase by 2+ grades
0 participants
Change in Severity Grade for Laboratory Tests From Grade 0
Total Cholesterol: Increase by 1 grade
2 participants
Change in Severity Grade for Laboratory Tests From Grade 0
Total Cholesterol: Increase by 2+ grades
0 participants

SECONDARY outcome

Timeframe: Day 2 to Day 40

Population: The Safety Sample (SS) population includes all participants who received any amount of tofacitinib.

The study sites graded the severity of AEs experienced by the study participants according to the criteria set forth in the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) 5.0. The NCI-CTCAE manual provides a common language to describe levels of severity, to analyze and interpret data, and to articulate the clinical significance of all AEs. A treatment-emergent AE is defined as an increase in grade from the last assessment prior to a participant starting treatment at Day 2, or from the last post-baseline value that doesn't meet grading criteria. AEs were classified by system organ class and preferred term according to the Medical Dictionary for Regulatory Activities (MedDRA) version 25.1.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=7 Participants
25-day regimen of tofacitinib 11 mg daily
Treatment-emergent Adverse Events by Severity
Grade 2
1 Adverse Events
Treatment-emergent Adverse Events by Severity
Grade 3
0 Adverse Events
Treatment-emergent Adverse Events by Severity
Grade 4
0 Adverse Events
Treatment-emergent Adverse Events by Severity
Grade 5
0 Adverse Events

SECONDARY outcome

Timeframe: Day 2 to Day 40

Population: The Safety Sample (SS) population includes all participants who received any amount of tofacitinib.

The relationship, or attribution, of an adverse event to the study therapy regimen or study procedure(s) was initially determined by the site investigator and recorded on the appropriate AE/SAE eCRF as not related, possibly related or related. Final determination of attribution for safety reporting was determined by sponsor. An AE assessed as possibly related or related was classified as related to the study therapy or procedure. A treatment-emergent AE is defined as an increase in grade from the last assessment prior to a participant starting treatment at Day 2, or from the last post-baseline value that doesn't meet grading criteria. AEs were classified by system organ class and preferred term according to the Medical Dictionary for Regulatory Activities (MedDRA) version 25.1.

Outcome measures

Outcome measures
Measure
Tofacitinib
n=7 Participants
25-day regimen of tofacitinib 11 mg daily
Treatment-emergent Adverse Events by Relationship to Tofacitinib
Related to Tofacitinib
0 Adverse Events
Treatment-emergent Adverse Events by Relationship to Tofacitinib
Not Related to Tofacitinib
1 Adverse Events

Adverse Events

Tofacitinib

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
Tofacitinib
n=7 participants at risk
25-day regimen of tofacitinib 11 mg daily
Gastrointestinal disorders
Dental caries
14.3%
1/7 • Number of events 1 • Treatment-emergent adverse events were collected in EDC from Day 2 until Day 40.
Adverse events (AEs) of Grade 2 or higher and all Grade 1 or higher COVID-19 events were recorded in EDC. A treatment-emergent AE is defined as an increase in grade from the last assessment prior to a participant starting treatment at Day 2, or from the last post-baseline value that doesn't meet grading criteria. AEs were classified by system organ class and preferred term according to the Medical Dictionary for Regulatory Activities (MedDRA) version 25.1.

Additional Information

Director, Clinical Research Operations Program

DAIT/NIAID

Phone: 301-594-7669

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place